acute myelogenous leukemia

(uh-KYOOT my-uh-LAH-juh-nuss loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become AML. But most do not. Also called AML, acute myeloblastic leukemia, acute myelocytic leukemia, acute myeloid leukemia.

A procedure where bone marrow stem cells are taken from a genetically matched donor (a brother, sister, or unrelated donor) and given to the patient through an intravenous (IV) line. In time, donated stem cells start making new, healthy blood cells.

See complementary and alternative medicine.

(an-uh-fuh-LAK-suss) A very severe allergic reaction to a foreign protein, as in a bee sting, or to a medicine. This reaction causes the blood pressure to drop and trouble breathing. Before a patient receives ATG, a treatment for aplastic anemia, a skin test is given to find out if they are likely to develop anaphylaxis. Also known as anaphylactic shock.

An approach to treating bone marrow failure using natural male hormones. Androgen therapy can help the bone marrow make more blood cells. This is an older treatment for bone marrow failure that is rarely used because of the side effects. Scientists are studying these medicines to try to better understand why they work in some cases of acquired and genetic bone marrow failure.

(uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness.

(an-tee-by-AH-tik) A medicine that fights bacterial infections. When a person with bone marrow failure does not have enough neutrophils, the white blood cells that fight infection, antibiotics may help to prevent and fight infection.

(ant-i-ko-AG-yuh-lunt) See blood thinner.

(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells: red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most scientists believe that aplastic anemia happens when the immune system attacks the bone marrow stem cells. Aplastic anemia can be acquired (begin any time in life) or can be hereditary (less common, passed down from parent to child).

Programmed cell death.

(uh-SITE-eez) Extra fluid and swelling in the belly area (abdomen). Also called hydroperitoneum.

Any condition that happens when the immune system attacks the body's own normal tissues by mistake.

A procedure in which some of the patient's own bone marrow stem cells are removed, frozen, and then returned to the through an intravenous (IV) line. In time, the stem cells start making new, healthy blood cells.

Describes one of several ways that a trait or disorder can be inherited, or passed down through families. "Autosomal" means that the mutated, or abnormal, gene is located on one of the numbered, or non-sex, chromosomes. "Dominant" means that only one copy of the mutated gene is enough to cause the disease. Dyskeratosis congenita is a rare cause of bone marrow failure disease. It may have an autosomal dominant, autosomal recessive or x-linked pattern of inheritance.

Describes one of several ways that a trait or disorder can be inherited, or passed down through families. "Autosomal" means that the mutated, or abnormal, gene is located on one of the numbered, or non-sex, chromosomes. "Recessive" means that two copies of a mutated gene must be present to cause the disease. Dyskeratosis congenita is a rare cause of bone marrow failure. It may have an autosomal dominant, autosomal recessive or x-linked pattern of inheritance.

The study of a subject to increase knowledge and understanding about it. The goal of basic research in medicine is to better understand disease. In the laboratory, basic research scientists study changes in cells and molecules linked to disease. Basic research helps lead to better ways of diagnosing, treating, and preventing disease. Also called basic science research.

A type of white blood cell that plays a role in allergic reactions.

A chemical that is widely used by the chemical industry in the United States to make plastics, resins, nylon and synthetic fibers. Benzene is found in tobacco smoke, vehicle emissions, and gasoline fumes. Exposure to benzene may increase the risk of developing a bone marrow failure disease. Benzene can affect human health by causing bone marrow stem cells not to work correctly.

(bil-i-ROO-bun) A reddish yellow substance formed when red blood cells break apart. It is found in the bile and in the blood. Yellowing of the skin and eyes can occur with high levels of bilirubin. Also called total bilirubin.

A substance made from a living system, such as a virus, and used to prevent or treat disease. Biological drugs include antibodies, globulin, interleukins, serum, and vaccines. Also called a biologic or biological drug.

A young white blood cell. The number of blast cells in the bone marrow helps define how severe MDS is in a person. When 20 out of 100 cells in the bone marrow are blasts, this is considered acute myeloid leukemia.

See Blast Cells.

A mass of blood that forms when platelets stick together. Harmful blood clots are more likely to happen in PNH. The term thrombus describes a blood clot that develops and attaches to a blood vessel. The term embolus describes a blood clot or other foreign matter that gets into the bloodstream and gets stuck in a blood vessel.

A medicine used to stop blood clots from forming. Blood thinners can be used to treat or prevent clots. Some common blood thinners are enoxaprin (Lovenox), heparin (Calciparine or Liquaemin), and warfarin (Coumadin). Also called and anticoagulant or thrombopoiesis inhibitor.

A procedure in which whole blood or one of its components is given to a person through an intravenous (IV) line into the bloodstream. A red blood cell transfusion or a platelet transfuson can help some patients with low blood counts.

The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.

A medical procedure to remove of a small amount of liquid bone marrow through a needle inserted into the back of the hip. The liquid bone marrow is examined for abnormalities in cell size, shape, or look. Tests may also be run on the bone marrow cells to look for any genetic abnormalities.

A medical procedure to remove a small piece of solid bone marrow using a needle that goes into the marrow of the hip bone. The solid bone marrow is examined for cell abnormalities, the number of different cells and checked for scaring of the bone marrow.

A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be hereditary (less common, passed down from parent to child).

A procedure where bone marrow stem cells are collected from marrow inside the donor's hipbone and given to the patient through an intravenous (IV) line. In time, donated stem cells start making new, healthy blood cells.

(bud-kee-AR-ee SIN-drome) A blood clot in the major vein that leaves the liver (hepatic vein). The liver and the spleen may become enlarged. Budd-Chiari syndrome can occur in PNH.

How much of the bone marrow volume is occupied by various types of blood cells.

(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be given chemotherapy to kill bone marrow cells that have an abnormal size, shape, or look. Chemotherapy hurts healthy cells along with abnormal cells. If chemotherapy works in controlling abnormal cells, then relatively normal blood cells will start to grow again. Low-dose chemotherapy agents include: cytarabine (Ara-C) and hydroxyurea (Hydrea). High-dose chemotherapy agents include: daunorubicin (Cerubidine), idarubicin (Idamycin), and mitoxanrone (Novantrone).

The part of the cell that contains our DNA or genetic code.

A medical condition that lasts a long time. A chronic illness can affect a person's lifestyle, ability to work, physical abilities and independence.

A person who gives advice, or counsel, to people who are coping with long-term illness. A chronic illness counselor helps people understand their abilities and limitations, cope with the stress, pain, and fatigue associated with long-term illness. A chronic illness counselor can often be located by contacting a local hospital.

A type of research that involves individual persons or a group of people. There are three types of clinical research. Patient-oriented research includes clinical trials which test how a drug, medical device, or treatment approach works in people. Epidemiology or behavioral studies look at the patterns and causes of disease in groups of people. Outcomes and health services research seeks to find the most effective treatments and health services.

A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a disease or health problem. Quality of life (supportive care) trials study ways to improve the comfort of people with chronic illness. Prevention trials look for better ways to prevent disease in people who have never had the disease.

Trials are in four phases: Phase I tests a new drug or treatment in a small group to see if it is safe. Phase II expands the study to a larger group of people to find out if it works. Phase III expands the study to an even larger group of people to compare it to the standard treatment for the disease; and Phase IV takes place after the drug or treatment has been licensed and marketed to find out the long-term impact of the new treatment.

To make copies. Bone marrow stem cells clone themselves all the time. The cloned stem cells eventually become mature blood cells that leave the bone marrow and enter the bloodstream.

To thicken. Normal blood platelets cause the blood to coagulate and stop bleeding.

A group of proteins that move freely in the bloodstream. These proteins support (complement) the work of white blood cells by fighting infections.

A medical approach that is not currently part of standard practice. Complementary medicine is used along with standard medicine. Alternative medicine is used in place of standard medicine. Example of CAM therapies are acupuncture, chiropractic, homeopathic, and herbal medicines. There is no complementary or alternative therapy that effectively treats bone marrow failure. Some CAM therapies may even hinder the effectiveness of standard medical care. Patients should talk with their doctor if they are currently using or considering using a complementary or alternative therapy.

A group of tests performed on a small amount of blood. The CBC measures the number of each blood cell type, the size of the red blood cells, the total amount of hemoglobin, and the fraction of the blood made up of red blood cells. Also called a CBC.

A procedure where umbillical cord stem cells are given to the patient through an intravenous (IV) line. Stem cells are collected from an umbilical cord right after the birth of a baby. They are kept frozen until needed. In time, donated stem cells given to the patient begin making new, healthy blood cells.

An imaging technique using x-ray technology and computerization to create a three-dimentional image of a body part. Also called a CT scan, it can be used to locate a blood clot in the body.

A response to treatment indicating that no sign of abnormal chromosomes are found. When a test is done on a patient with 5q deletion MDS, and there are no signs of an abnormal chromosome 5, then that patient has achieved a cytogenetic remission. Also called cytegenetic response.

(sie-toe-juh-NEH-tiks) The study of chromosomes (DNA), the part of the cell that contains genetic information. Some cytogenetic abnormalities are linked to different forms of myelodysplastic syndromes (MDS).

(sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count.

(sie-tuh-TOK-sik) A medicine that kills certain cells. Chemotherapy for MDS patients often involves the use of cytotoxic agents.

A test that helps doctors find out if a person has a problem with blood clotting.

(di-NO-vo) Brand new, referring to the first time something occurs. MDS that is untreated or that has no known cause is called de novo MDS.

The death of part of the intestine. This can happen if the blood supply in the intestine is cut off, for example, from a blood clot in the abdomen. Also called intestinal necrosis, ischemic bowel, dead gut.

A rare form of pure red cell aplasia that can be passed down from parent to child. Diamond-Blackfan anemia (DBA) is characterized by low red blood cell counts detected in the first year of life. Some people with DBA have physical abnormalities such as small head size, low frontal hairline, wide-set eyes, low-set ears. Genetic testing is used to diagnose DBA.

Vitamins, minerals, herbs and other substances meant to improve your nutritional intake. Dietary supplements are taken by mouth in the form of a pill, capsule, tablet or liquid.

To become distinct or specialized. In the bone marrow, young parent cells (stem cells) develop, or differentiate, into specific types of blood cells (red cells, white cells, platelets).

The gene that always expresses itself over a recessive gene. A person with a dominant gene for a disease has the symptoms of the disease. They can pass the disease on to children.

An inherited disease that may lead to bone marrow failure.

Refers to how well a graft (donor cells) is accepted by the host (the patient) after a bone marrow or stem cell transplant. Several factors contribute to better engraftment: physical condition of the patient, how severe the disease is, type of donor available, age of patient. Successful engraftment results in new bone marrow that produces healthy blood cells.

A type of white blood cell that kills parasites and plays a role in allergic reactions.

The study of patterns and causes of disease in groups of people. Epidemiology researchers study how many people have a disease, how many new cases are diagnosed each year, where patients are located, and environmental or other factors that influence disease.

(i-RITH-ruh-site) See red blood cell.

(i-rith-row-POY-uh-tun) A protein made by the kidneys. Erythropoietin, also called EPO, is created in response to low oxygen levels in the body (anemia). EPO causes the bone marrow to make more red blood cells. A shortage of EPO can also cause anemia.

A medicine used to help the bone marrow make more red blood cells. Epoetin alfa (Epogen, Procrit) and darbepoetin alfa (Aranesp) are erythropoietin-stimulating agents that can help boost the red blood cell count of some bone marrow failure patients. Also called red blood cell growth factor.

A form of estrogen, it is the most potent female hormone. It is also present in males. Estradiol is involved in many body functions beyond the reproductive system. Researchers are investigating the role of estradiol in the treatment of genetic bone marrow failure.

The cause or origin of a disease.

A criteria used for classifying different types of myelodysplastic syndromes (MDS). The FAB (French, American, British) Classification System was developed by a group of French, American and British scientists. This system is based on 2 main factors: the percentage of blast cells in bone marrow, and the percentage of blast cells in the bloodstream. The FAB system is somewhat outdated, but is still used by some doctors today. The World Health Organization (WHO) Classification System has largely replaced the FAB Classification System.

A rare inherited disorder that happens when the bone marrow does not make enough blood cells: red cells, white cells, and platelets. Fanconi anemia is diagnosed early in life. People with Fanconi anemia have a high likelihood of developing cancer. Genetic testing is used to diagnose Fanconi anemia.

(FER-i-tin) A protein inside of cells that stores iron for later use by your body. Sometimes ferritin is released into the blood. The ferritin level in the blood is called serum ferritin.

(FER-i-tin) A blood test used to monitor how much iron the body is storing for later use.

(fie-BRO-suss) Scarring of tissue. Fibrosis of the bone marrow is an feature seen in some types of unclassified myeldysplastic syndrome (MDS).

See fluorescence in situ hybridization.

(sy-TOM-uh-tree) A laboratory test that gives information about cells, such as size, shape, and percentage of live cells. Flow cytometry is the test doctors use to see if there are any proteins missing from the surface of blood cells. It is the standard test for confirming a diagnosis of paroxysmal nocturnal hemoglobinuria (PNH).

(flor-EH-sense in SIT-tyoo hy-bru-duh-ZAY-shun) An important laboratory test used to help doctors look for chromosomal abnormalities and other genetic mutations. Fluorescence in situ hybridization, also called FISH, directs colored light under a microscope at parts of chromosomes or genes. Missing or rearranged chromosomes are identified using FISH.

(FOE-late) A B-vitamin that is found in fresh or lightly cooked green vegetables. It helps the bone marrow make normal blood cells. Most people get enough folate in their diet. Doctors may have people with paroxysmal nocturnal hemaglobinuria (PNH) take a man-made form of folate called folic acid.

See folate.

A laboratory test that looks at the whether red blood cells break apart too easily when they are placed in mild acid. This test has been used in the past to diagnose paroxysmal nocturnal hemoglobinuria (PNH). Most doctors now use flow cytometry, a more accurate method of testing for PNH. Ham Test is also called acid hemolysin test.

(hi-MA-tuh-crit) A blood test that measures the percentage of the blood made up of red blood cells. This measurement depends on the number of red blood cells and their size. Hematocrit is part of a complete blood count. Also called HCT, packed cell volume, PCV.

(hee-muh-TOL-uh-jist) A doctor who specializes in treating blood diseases and disorders of blood producing organs.

(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.

A condition that occurs when the body absorbs and stores too much iron. This leads to a condition called iron overload. In the United States, hemochromatosis is usually caused by a genetic disorder. Organ damage can occur if iron overload is not treated.

A protein in the red blood cells. Hemoglobin picks up oxygen in the lungs and brings it to cells in all parts of the body.

(hee-muh-gloe-buh-NYOOR-ee-uh) The presence of hemoglobin in the urine.

(hi-MOL-uh-suss) The destruction of red blood cells.

See human leukocyte antigen.

A part of the endocrine system that serves as the body's chemical messengers. Hormones move through the bloodstream to transfer information and instruction from one set of cells to another.

(LEW-kuh-site ANT-i-jun) One of a group of proteins found on the surface of white blood cells and other cells. These antigens differ from person to person. A human leukocyte antigen test is done before a stem cell transplant to closely match a donor and a recipient. Also called HLA.

A condition in which there are too many cells, for example, within the bone marrow. Patients with leukemia have hypercellular bone marrow filled with to many immature white blood cells.

A condition in which there are too few cells, for example, within the bone marrow. Patients with aplastic anemia have hypocellular bone marrow.

Usually refers to any condition with no known cause.

(i-myoo-no-KOM-pruh-mized) Occurs when the immune system is not functioning properly, leaving the patient open to infection. A person can be immunocompromised due to low white blood cell count or due to some medicines. Also called immune compromised.

(i-myoo-no-suh-PREH-siv) Drugs that lower the body's immune response and allow the bone marrow stem cells to grow and make new blood cells. ATG (antithymocyte globulin) or ALG (antilymphocyte globulin) with cyclosporine are used to treat bone marrow failure in aplastic anemia. Immunosuppressive drugs may help some patients with myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH).

A committee that makes sure a clinical trial is safe for patients in the study. Each medical center, hospital, or research facility doing clinical trials must have an active Institutional Review Board (IRB). Each IRB is made up of a diverse group of doctors, faculty, staff and students at a specific institution.

A system that turns patient data into a score. The score tells how quickly a myelodysplastic syndrome (MDS) case is progressing and helps predict what may happen with the patient's MDS in the future. Also called IPSS.

A method of getting fluids or medicines directly into the bloodstream over a period of time. Also called IV infusion.

A new drug, antibiotic drug, or biological drug that is used in a clinical trial. It also includes a biological product used in the laboratory for diagnostic purposes. Also called IND.

(kee-LAY-shun) A drug therapy to remove extra iron from the body. Patients with high blood iron (ferritin) levels may receive iron chelation therapy. The U.S. Food and Drug Administratin (FDA) has approved two iron chelators to treat iron overload in the U.S.: deferasirox, an oral iron chelator, and deferoxamine, a liquid given by injection.

A condition that occurs when too much iron accumulates in the body. Bone marrow failure disease patients who need regular red blood cell transfusions are at risk for iron overload. Organ damage can occur if iron overload is not treated.

(iss-KEE-mee-uh) Occurs when the blood supply to specific organ or part of the body is cut off, causing a localized lack of oxygen.

Go here to see the original:
Glossary of Terms | Aplastic Anemia and MDS International ...

Skin Stem Cells Comments Off on Glossary of Terms | Aplastic Anemia and MDS International … | November 22nd, 2017

T

he complications of the little boys genetic skin disease grew as he did. Tiny blisters had covered his back as a newborn. Then came the chronic skin wounds that extended from his buttocks down to his legs.

By June 2015, at age 7, the boy had lost nearly two-thirds of his skin due to an infection related to the genetic disorder junctional epidermolysis bullosa, which causes the skin to become extremely fragile. Theres no cure for the disease, and it is often fatal for kids. At the burn unit at Childrens Hospital in Bochum, Germany, doctors offered him constant morphine and bandaged much of his body, but nothing not even his fathers offer to donate his skin worked to heal his wounds.

We were absolutely sure we could do nothing for this kid, Dr. Tobias Rothoeft, a pediatrician with Childrens Hospital in Bochum, which is affiliated with Ruhr University. [We thought] that he would die.

advertisement

As a last-ditch effort, the boys father asked if there were any available experimental treatments. The German doctors reached out to Dr. Michele De Luca, an Italian stem cell expert who heads the Center for Regenerative Medicine at the University of Modena and Reggio Emilia, to see if a transplant of genetically modified skin cells might be possible. De Luca knew the odds were against them such a transplant had only been performed twice in the past, and never on such a large portion of the body. But he said yes.

The doctors were ultimately able to reconstruct fully functional skin for 80 percent of the boys body by grafting on genetically modified cells taken from the boys healthy skin. The researchers say the results of this single-person clinical trial, published on Wednesday in Nature, show that transgenic stem cells can regenerate an entire tissue. De Luca told reporters the procedure not only offers hope to the 500,000 epidermolysis bullosa patients worldwide but also could offer a blueprint for using genetically modified stem cells to treat a variety of other diseases.

To cultivate replacement skin, the medical team took a biopsy the size of a matchbook from the boys healthy skin and sent it to De Lucas team in Italy. There, researchers cloned the skin cells and genetically modified them to have a healthy version of the gene LAMB3, responsible for making the protein laminin-332. They grew the corrected cultures into sheets, which they sent back to Germany. Then, over a series of three operations between October 2015 and January 2016, the surgical team attached the sheets on different parts of the boys body.

The gene-repaired skin took, and spread. Within just a month the wounds were islands within intact skin. The boy was sent home from the hospital in February 2016, and over the next 21 months, researchers said his skin healed normally. Unlike burn patients whose skin grafts arent created from genetically modified cells the boy wont need ointment for his skin and can regrow his hair.

And unlike simple grafts of skin from one body part to another, we had the opportunity to reproduce as much as those cells as we want, said plastic surgeon Dr. Tobias Hirsch, one of the studys authors. You can have double the whole body surface or even more. Thats a fantastic option for a surgeon to treat this child.

Dr. John Wagner, the director of the University of Minnesota Masonic Childrens Hospitals blood and marrow transplant program, told STAT the findings have extraordinary potential because, until now, the only stem cell transplants proven to work in humans was of hematopoietic stem cells those in blood and bone marrow.

Theyve proven that a stem cell is engraftable, Wagner said. In humans, what we have to demonstrate is that a parent cell is able to reproduce or self-renew, and differentiate into certain cell populations for that particular organ. This is the first indication that theres another stem cell population [beyond hematopoietic stem cells] thats able to do that.

The researchers said the aggressive treatment outlined in the study necessary in the case of the 7-year-old patient could eventually help other patients in less critical condition. One possibility, they noted in the paper, was to bank skin samples from infants with JEB before they develop symptoms. These could then be used to treat skin lesions as they develop rather than after they become life-threatening.

The treatment might be more effective in children, whose stem cells have higher renewal potential and who have less total skin to replace, than in adults, Mariaceleste Aragona and Cdric Blanpain, stem cell researchers with the Free University of Brussels, wrote in an accompanying commentary for Nature.

But De Luca said more research must be conducted to see if the methods could be applied beyond this specific genetic disease. His group is currently running a pair of clinical trials in Austria using genetically modified skin stem cells to treat another 12 patients with two different kinds of epidermolysis bullosa, including JEB.

For the 7-year-old boy, life has become more normal now that it ever was before, the researchers said. Hes off pain meds. While he has some small blisters in areas that didnt receive a transplant, they havent stopped him from going to school, playing soccer, or behaving like a healthy child.

The kid is doing quite well. If he gets bruises like small kids [do], they just heal as normal skin heals, Rothoeft said. Hes quite healthy.

Southern Correspondent

Max covers hospitals and health care.

More:
'Extraordinary' tale: Stem cells heal a young boy's lethal ...

Skin Stem Cells Comments Off on ‘Extraordinary’ tale: Stem cells heal a young boy’s lethal … | November 14th, 2017

Scientists reported Wednesday that they genetically modified stem cells to grow skin that they successfully grafted over nearly all of a child's body - a remarkable achievement that could revolutionize treatment of burn victims and people with skin diseases.

The research, published in the journal Nature, involved a 7-year-old boy who suffers from a genetic disease known as junctional epidermolysis bullosa (JEB) that makes skin so fragile that minor friction such as rubbing causes the skin to blister or come apart.

By the time the boy arrived at Children's Hospital of Ruhr-University in Germany in 2015, he was gravely ill. Doctors noted that he had "complete epidural loss" on about 60 percent of his body surface area, was in so much pain that he was on morphine, and fighting off a systemic staph infection. The doctors tried everything they could think of: Antibiotics, changing dressings, grafting skin donated by his father. But nothing worked, and they told his parents to prepare for the worst.

"We had a lot of problems in the first days keeping this kid alive," Tobias Hirsch, one of the treating physicians, recalled in a conference call with reporters this week.

Hirsch and his colleague Tobias Rothoeft began to scour the medical literature for anything that might help and came across an article describing a highly experimental procedure to genetically engineer skin cells. They contacted the author, Michele De Luca, of the Center for Regenerative Medicine University of Modena and Reggio Emilia in Italy. De Luca flew out right away.

Using a technique he had used only twice before and even then only on small parts of the body, De Luca harvested cells from a four-square-centimeter patch of skin on an unaffected part of the boy's body and brought them into the lab. There, he genetically modified them so that they no longer contained the mutated form of a gene known to cause the disease and grew the cells into patches of genetically modified epidermis. They discovered, the researchers reported, that "the human epidermis is sustained by a limited number of long-lived stem cells which are able to extensively self-renew."

In three surgeries, the child's doctors took that lab-grown skin and used it to cover nearly 80 percent of the boy's body - mostly on the limbs and on his back, which had suffered the most damage. The procedure was permitted under a "compassionate use" exception that allows researchers under certain dire circumstances to make a treatment available even though it is not approved by regulators for general use. Then, over the course of the next eight months while the child was in the intensive care unit, they watched and waited.

The boy's recovery was stunning.

The regenerated epidermis "firmly adhered to the underlying dermis," the researchers reported. Hair follicles grew out of some areas. And even bumps and bruises healed normally. Unlike traditional skin grafts that require ointment once or twice a day to remain functional, the boy's new skin was fine with the normal amount of washing and moisturizing.

"The epidermis looks basically normal. There is no big difference," De Luca said. He said he expects the skin to last "basically the life of the patient."

In an analysis accompanying the main article in Nature, Mariacelest Aragona and Cedric Blanpain wrote that this therapy appears to be one of the few examples of truly effective stem-cell therapies. The study "demonstrates the feasibility and safety of replacing the entire epidermis using combined stem-cell and gene therapy," and also provides important insights into how different types of cells work together to help our skin renews itself.

They said there are still many other lingering questions, including whether such procedures might work better in children than adults and whether there would be longer-term adverse consequences, such as the development of cancer.

There are also many challenges to translating this research to treating wounds sustained in fires or other violent ways. In the skin disease that was treated in the boy, the epidermis is damaged but the layer beneath it, the dermis, is intact. The dermis is what the researchers called an ideal receiving bed for the lab-grown skin. But if deeper layers of the skin are burned or torn off, it's possible that the artificial skin would not adhere as well.

"No matter how you prepare, it's a bad situation," De Luca said. For the time being, he says he's continuing to study the procedure in two clinical trials that involve genetic diseases.

Meanwhile, Hirsch and Rothoeft report that the boy is continuing to do well and is not on any medication for the first time in many years. Doctors are carefully monitoring the child for any signs that there may be some cells that were not corrected and that the disease may re-emerge, but right now that does not appear to be happening in the transplanted areas. However, the child does have some blistering in about 2 to 3 percent of his body in non-grafted areas and they are considering whether to replace that skin as well.

But for now, they are giving the boy time to be a boy, Rothoeft said: "The kid is now back to school and plays soccer and spends other days with the children."

Read more:
Genetically modified skin grown from stem cells saved a 7 ...

Skin Stem Cells Comments Off on Genetically modified skin grown from stem cells saved a 7 … | November 14th, 2017

A new therapy could restore healthy and protective skin to patients with a rare genetic disease.

iStock.com/Andrey Prokhorov

By Kelly ServickNov. 8, 2017 , 1:00 PM

A 7-year-old who lost most of his skin to a rare genetic disease has made a dramatic recovery after receiving an experimental gene therapy, researchers announced today. The treatmenta whole-body graft of genetically modified stem cellsis the most ambitious attempt yet to treat a severe form of epidermolysis bullosa (EB), an often-fatal group of conditions that cause skin to blister and tear off at the slightest touch.

The new approach can address only a subset of the genetic mutations that cause EB. But the boys impressive recoveryhes now back inschool and is even playing soccercould yield insights that help researchers use stem cells to treat other genetic skin conditions.

It is very unusual that we would see a publication with a single case study anymore, but this one is a little different, says Jakub Tolar, a bone marrow transplant physician at the Masonic Cancer Center, University of Minnesotain Minneapolis who is developing therapies for EB. This is one of these [studies] that can determine where the future of the field is going to go.

EB results from mutations to any of several genes that encode proteins crucial for anchoring the outer layer of skin, the epidermis, to the tissue below. The missing or defective protein can cause skin to slough off from minor damage, creating chronic injuries prone to infection. Some forms of EB can be lethal in infancy, and some predispose patients to an aggressive and deadly skin cancer. The only treatment involves painfully dressing and redressing wounds daily. Bandage costs can approach $100,000 a year, says Peter Marinkovich, a dermatologist at Stanford University in Palo Alto, California, who treats EB patients. Theyre like walking burn victims, he says.

In fact, the new approach is similar to an established treatment for severe burns, in which sheets of healthy skin are grown from a patients own cells and grafted over wounds. But stem cell biologist and physician Michele De Luca of the University of Modena and Reggio Emilia in Italy and his colleagues have been developing a way to counteract an EB-causing mutation by inserting a new gene into the cells used for grafts. His group has already treated two EB patients with this approach. They publishedencouraging resultsfrom their first attemptwith small patches of gene-corrected skin on a patients legsin 2006.

In 2015, De Lucas team got a desperate request from doctors in Germany. Their young patient had a severe form of the disease known as junctional EB, caused by a mutation in a gene encoding part of the protein laminin 332, which makes up a thin membrane just below the epidermis. It was the same gene De Lucas team was targeting in an ongoing clinical trial, but this case was especially dire: Lacking most of his skin, the boy had contracted multiple infections and was in a life-threatening septic state. The emergency treatment would be the first test of their gene therapy approach over such a large and severely damaged area.

De Lucas team used a patch of skin a little bigger than a U.S. postage stamp from an unblistered part of the boys groin to culture epidermal cells, which include stem cells that periodically regenerate the skin. They infected those cells with a retrovirus bearing healthy copies of the needed gene,LAMB3, and grew them into sheets ranging from 50 to 150 square centimeters. In two surgeries, a team at Ruhr University in Bochum, Germany, covered the boys arms, legs, back, and some of his chest in the new skin.

After a month,most of the new skin had begun to regenerate, covering 80% of the boys body in strong and elastic epidermis, the researchers report online today inNature. Whats more, hes developed no blisters in the grafted areas in the 2 years since the surgery.

Other researchers have long been concerned that using a retrovirus to insert genes at random points in cells genomes might cause cancer. (In the early 2000s, five children who participated in a retrovirus-based gene therapy trial for severe combined immunodeficiencydeveloped leukemia.) But the current study found no evidence that the insertion affected cancer genes.

De Luca and colleagues were also able to track which grafted cells regenerated the skin over time by using the different locations of the genetic insert as markers for individual cells and their progeny. They found that most cells from the graft disappeared after a few months, but a small population of long-lived cells called holoclones formed colonies that renewed the epidermis.

Epidermal stem cells known as holoclones (shown in pink) were responsible for regenerating the young epidermolysis bullosapatients skin, whileother cell types disappeared over time.

News & Views/Nature; adapted by E. Petersen/Science

Thats an important lesson, Tolar says; it suggests that future attempts to correct genetic skin diseases should focus on culture conditions that nourish these stem cells, and potentially even target them for modification. If you have a gene correction strategy, he says, youd better have these primitive epidermal stem cells in mind.

The current results could benefit several thousand EB patients across the world, Marinkovich says, but it wont work for all of them. More than half have a form of the disease called EB simplex, which is causednot by a missing protein, but by mutations that produce an active but dysfunctional protein. For these errors, correction with a gene-editing tool like CRISPR makes more sense, De Luca says.

The grafts also cant repair damage to internal surfaces such as the esophagus, Tolar notes, which occurs in some EB cases. Fortunately, that wasnt an issue for the boy in this study. The treatment is a good step in the right direction, he says, but its not curative.

Both De Luca and Marinkovichs teams are exploring a similar gene therapy for another major form of the disease, called dystrophic EB, caused by a different genetic error affecting a larger protein. Biotech companies are working with each group to test the approach in larger clinical trials.

Here is the original post:
A boy with a rare disease gets new skin, thanks to gene ...

Skin Stem Cells Comments Off on A boy with a rare disease gets new skin, thanks to gene … | November 14th, 2017

In a breakthrough treatment, researchers at a burn unit in Europe found a way to replace 80 percent of a boys skin using a combination of gene therapy and stem cells. The grafted skin attached to his body has continued to replace itself, even months later.

The patient - a boy who was 7 years old at the time of the treatment - was born with a rare skin condition called junctional epidermolysis bullosa. The condition causes the outer layer of the skin to peel away easily from the lower skin layers, making it incredibly fragile and prone to injury.

This is a very severe, devastating disease, where kids suffer a lot, said Dr. Michele De Luca, one of the authors of the research.

Experts not involved in the research have said this successful grafting treatment is a big step for those suffering from genetic skin conditions like this one.

This is really quite exciting, to have this translation for these patients, said Dr. Dennis Orgill, medical director of the Brigham and Womens Hospital Wound Center in Boston, who was not involved with the study. "That they can do these genetic manipulations and then have a long term result, which theyve demonstrated here, is a major breakthrough."

In this case, the treatment may have been lifesaving. The patient arrived at the hospital with a life-threatening bacterial skin infection spread over much of his body. Over the following weeks, his doctors tried everything they could to treat him without success.

Out of options, his treatment team was preparing to start end-of-life care when his parents pleaded with them to try an experimental therapy.

Surgeons in Germany took a sample of the boys skin, less than one square inch in size, that was unharmed by the bacterial infection. In a lab, researchers infected the skin biopsy with a virus specially designed to alter the genetic code within the skin cells, correcting the mutation responsible for his fragile skin. The researchers "grew" the skin and used it to surgically replace the patients blistered and destroyed skin.

After 21 months, the new skin is regenerating itself without problems and has been resilient; it can hold up to normal wear much better than his original skin.

While this result only applies to one rare skin disorder right now, experts said the approach could be used more widely for other diseases in the future.

We are running other clinical trials on other kinds of junctional epidermolysis bullosa," De Luca said. "In the future, it could be applied to other genetic diseases of the skin.

Researchers hope that it could help other people with seriously damaged skin in the future, too.

This technology could be extended into other patients with genetic conditions, or patients with extensive burns, Orgill said.

Read more:
Doctors replace boys skin using breakthrough gene therapy ...

Skin Stem Cells Comments Off on Doctors replace boys skin using breakthrough gene therapy … | November 14th, 2017

Related content

(CNN) - For the first time, doctors were able to treat a child who had a life-threatening rare genetic skin disease through a transplant of skin grown using genetically modified stem cells.

The grafts replaced 80% of the boy's skin.

The skin of his arms, legs, back and flanks, and some of the skin on his stomach, neck and face was missing or severely affected due to epidermolysis bullosa.

The compassionate-use experimental treatment is detailed in a case study published in the journal Nature on Wednesday.

Skin as fragile as a butterfly's wings -- that's how children with epidermolysis bullosa are described and why they're often called butterfly children.

The disease, of which there are five major types and at least 31 subtypes, is incurable. People with the condition have a defect in the protein-forming genes necessary for skin regeneration.

About 500,000 people worldwide are affected by forms of the disease. More than 40% of patients die before reaching adolescence.

Their skin can blister and erode due to something as simple as bumping into something or even the light friction of clothing, according to an email from Dr. Jouni Uitto, a professor and chairman of the Department of Dermatology and Cutaneous Biology at the Sidney Kimmel Medical College in Philadelphia. Uitto was not involved with this study.

Epidermolysis bullosa makes the skin incredibly susceptible to infections, and in the case of 7-year-old Hassan, whose treatment was detailed in Nature, those infections can be life-threatening.

A week after he was born in Syria, Hassan had a blister on his back, his father said through an interpreter in an interview provided by the hospital in Germany where the boy was treated.

Hassan's last name, as well as the first names of his family members, are not being disclosed to protect the privacy of the family.

In his first few weeks of life, Hassan was immediately diagnosed with epidermolysis bullosa, and their doctor in Syria told Hassan's family that there was no cure or therapy.

Over the years, their efforts to find help for their son's disease led the family to the Muenster University hospital in Germany in 2015, when Hassan was 7. His condition worsened, and he struggled with severe sepsis and a high fever. He weighed just over 37 pounds.

They didn't think he would make it, and doctors at Muenster decided in summer 2015 to transfer Hassan to the Ruhr-Universitt Bochum's University Hospitals, including the burn center -- one of the oldest in the country.

By the time Hassan arrived at Bochum, he had lost two-thirds of his surface skin.

"We had a lot of problems in first days just keeping him alive," said Dr. Tobias Rothoeft, consultant at the University Children's Hospital at Katholisches Klinikum Bochum.

Doctors tried to promote healing by changing his dressings and treating him with antibiotics, as well as putting him on an aggressive nutrition schedule, but nothing helped. They even tried transplanting skin from Hassan's father.

"By that time, he had lost 60% of his epidermis, the upper skin layer, and had 60% open wounds all over his body," said Dr. Maximilian Kueckelhaus of the Department of Plastic Surgery at Bochum's Burn Center.

Every approach failed, so the doctors prepared Hassan's family for what end-of-life care would entail. But the parents pleaded, asking the doctors to consult studies and research for experimental treatments that might help.

They found Dr. Michele De Luca at the University of Modena's Center for Regenerative Medicine in Italy. His publications described an experimental treatment transplanting genetically modified epidermal stem cells that healed small, non-life-threatening wounds in adults.

The medical team reached out to De Luca, asking whether he could help them replicate the procedure on a larger scale to help Hassan, and he agreed. De Luca told Hassan's parents that he believed there was a 50% chance of the treatment being successful.

They were more than willing to accept the risk, to do anything to help their son have a chance at a normal life.

Hassan "was in severe pain and was asking a lot of questions: 'Why do I suffer from this disease? Why do I have to live this life? All children can run around and play. Why am I not allowed to play soccer?' I couldn't answer these questions," his father said. "It was a tough decision for us, but we wanted to try for Hassan."

To obtain the skin's stem cells, the doctors took a small biopsy -- only accounting for 1 square inches -- from an unaffected part of Hassan's skin. The stem cells were processed by De Luca in Italy. A healthy version of the gene that is normally defective in epidermolysis bullosa patients was added to the cells, along with retroviral vectors: virus particles that assist the gene transfer.

This genetic transfer would essentially "correct" the cells.

The single cells were grown and cultivated on plastic and fibrin substrate, which is used to treat large skin burns, to form a large piece of epidermis. This method enabled the researchers to grow as much skin as they needed. The whole process took three to four weeks, Kueckelhaus said.

Once the sheets were ready, they were transferred from Italy to Germany and transplanted onto the well-cleaned wounds right away during two surgeries. The first procedure in October 2015 applied the sheets to Hassan's arms and legs. The second surgery, in November, grafted the sheets to Hassan's entire back and the other affected areas.

Hassan began to improve immediately. The researchers noticed that the grafts were not rejected; they bound to all of the areas they were transplanted.

"For everyone that was involved, taking off the bandages and seeing for the first time that this is working out, that the transplants are actually attached to the patient and growing skin, that's an incredible moment," Kueckelhaus said.

Hassan was discharged from the hospital in February 2016.

After steady followups over 21 months, the researchers found that Hassan's new skin healed normally, didn't blister anymore, and was resistant to stress. It was even growing hair. Unlike some skin graft patients, he doesn't require any ointment to keep his skin smooth and hydrated. And like any growing kid, he bruises and recovers normally.

They also learned that only a few stem cells contribute to the long-term maintenance of the epidermis, shedding light on cellular hierarchy in this regard.

"The investigators removed of small piece of patient's skin, isolated cells with stem cell potential for growth, introduced a normal copy of the mutated gene to the cells, propagated a large number of these cells in culture and then grafted them back to the skin," Uitto said. "This concept is not new, but what is remarkable here is that they were able to change essentially the entire skin of the patient with normal cells."

Hassan's family is currently living in Germany. Hassan, now 9, is able to go to school and play sports, but he maintains a schedule of frequent monitoring at the hospital to ensure that the initial success of the treatment continues. The area of his skin that was not treated sometimes shows small blisters, and if it worsens, he may receive transplants there as well.

"Seeing him 18 months after the initial surgery with an intact skin is incredible because he has been in the ICU for so long," Kueckelhaus said. "He had bandages all over his body except his hands, feet and face. He was on extremely strong pain medication. So the quality of life was really, really bad for him. Seeing him play soccer, play sports, play with other kids, that is just amazing because that's something he couldn't do before."

"It felt like a dream for us," the boy's father said. "Hassan feels like a normal person now. He plays. He's being active. He loves life."

Everything points to a good long-term outcome for Hassan.

The researchers will continue to monitor him for complications. Sometimes, genetic modifications can cause malignancies in cells.

"That is of course one thing we really have to be aware of," Kueckelhaus said. "However, analyzing the integration profile of that gene into the boy's DNA, which we did, we saw that it's mostly in areas that don't cause too much concern about developing malignancies."

Epidermolysis bullosa patients can be at a very high risk of developing skin cancer simply because of the disease. Because Hassan now has intact skin and intact DNA, this risk might even decrease, but that will have to be proved through follow-up, Kueckelhaus said.

Given that this was one successful outcome for one patient, the experimental treatment can't be applied for other patients just yet. De Luca is conducting clinical trials using the treatment.

"This is one case with a distinct type of EB, and further studies will show whether this approach is applicable to other forms of EB as well," Uitto said. "It should be noted that in some severe forms of EB, the patients also suffer from fragility of the gastrointestinal and vesico-urinary tract, and some forms are associated with the development of muscular dystrophy. Obviously, gene therapy of the skin cannot correct them, and these issues have to be addressed in further studies."

Hassan's treatment also cost hundreds of thousands of dollars. Although the process could be optimized, doctors would still have to individually grow transplants for each patient, which could get very expensive.

But for patients' families, epidermolysis bullosa is already expensive.

"Standard maintenance treatment of patients with EB, including daily bandaging, antibiotics and special moisturizer, as well as frequent hospitalizations, can be extremely costly, and gene correction as described in this paper may well be cost-effective over the lifetime of these patients," Uitto noted.

Brett Kopelan, executive director of the Dystrophic Epidermolysis Bullosa Research Association of America, has a 10-year-old daughter, Rafi, with recessive dystrophic EB. Between January and August, $751,1778 for wound/burn dressings was charged to Kopelan's insurance company, he says. That doesn't account for drugs or hospital visits and surgeries.

Kopelan's nonprofit sends free supplies and bandages to families. The nonprofit can provide its employees with insurance that covers the medical equipment, but that isn't the case for everyone impacted by the condition, he said.

Kopelan is hopeful about the results of the study. The baths and bandage changes that are necessary for epidermolysis bullosa patients to stave off life-threatening infections can last hours and feel torturous.

"Do you remember the last time you got a paper cut and put Purell on it? It burned, right? Now think of 60% of body being an open wound, and opioids don't really work for this kind of pain," Kopelan wrote in an email. "This is what make EB kids and adults the strongest people on Earth."

The study "confirms our hopes that gene therapy is potentially the most efficacious path forward to providing a significant treatment option for those with epidermolysis bullosa," Kopelan said. "While it's important to remember that this is only one patient and more work needs to be done to demonstrate how effective this gene therapy platform may prove to be, I am very enthused."

"I wish that all children with the same disease could be treated in this way," Hassan's father said.

Here is the original post:
Scientists replace skin using genetically modified stem cells

Skin Stem Cells Comments Off on Scientists replace skin using genetically modified stem cells | November 14th, 2017

During the last decade, an increased interest in somatic stem cells has led to a flurry of research on one of the most accessible tissues of the body: skin. Much effort has focused on such topics as understanding the heterogeneity of stem cell pools within the epidermis and dermis, and their comparative utility in regenerative medicine applications. In Skin Stem Cells: Methods and Protocols, expert researchers in the field detail many of the methods which are now commonly used to study skin stem cells. These include methods and techniques for the isolation, maintenance and characterization of stem cell populations from skin. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls.

Authoritative and practical, Skin Stem Cells: Methods and Protocols seeks to aid scientists in the further understanding of these diverse cell types and the translation of their biological potential to the in vivo setting.

Originally posted here:
Skin Stem Cells - Methods and Protocols | Kursad Turksen ...

Skin Stem Cells Comments Off on Skin Stem Cells – Methods and Protocols | Kursad Turksen … | October 15th, 2017

CHOLLEY PhytocellBooster is ideal for smoothening wrinkles and eliminating the signs of aging or fatigue. It is a perfect product in many situations, such as after waking up in the morning, an exhausting day at work, and prior to attending a business meeting or party.

CHOLLEY Phytocell Booster instantly reduces wrinkles and imparts a lifted and younger-looking appearance to the skin. With Swiss guarantee of quality and excellence, the stem cells serum is clinically tested and found to be suitable for all skin type and complexions.

To exploit the power of IC-RAMP technology and Swiss stem cells technology, use CHOLLEY Phytocell Cream in combination with CHOLLEY Phytocell Booster. They provide your skin with full spectrum, day and night abti-aging care.

For best results, in morning and at night, apply CHOLLEY Phytocell Booster on the face, neck and dcollet. When the product is fully absorbed, complete the treatment with the application of CHOLLEY Phytocell Cream. It is the perfect Anti-aging and Antioxidant program.

Read this article:
Buy Serum Stem Cells Skin Care Products | CHOLLEY

Skin Stem Cells Comments Off on Buy Serum Stem Cells Skin Care Products | CHOLLEY | September 24th, 2017

Stem cells have the remarkable potential to develop into many different cell types in the body during early life and growth. In addition, in many tissues they serve as a sort of internal repair system, dividing essentially without limit to replenish other cells as long as the person or animal is still alive. When a stem cell divides, each new cell has the potential either to remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

Stem cells are distinguished from other cell types by two important characteristics. First, they are unspecialized cells capable of renewing themselves through cell division, sometimes after long periods of inactivity. Second, under certain physiologic or experimental conditions, they can be induced to become tissue- or organ-specific cells with special functions. In some organs, such as the gut and bone marrow, stem cells regularly divide to repair and replace worn out or damaged tissues. In other organs, however, such as the pancreas and the heart, stem cells only divide under special conditions.

Until recently, scientists primarily worked with two kinds of stem cells from animals and humans: embryonic stem cells and non-embryonic "somatic" or "adult" stem cells. The functions and characteristics of these cells will be explained in this document. Scientists discovered ways to derive embryonic stem cells from early mouse embryos more than 30 years ago, in 1981. The detailed study of the biology of mouse stem cells led to the discovery, in 1998, of a method to derive stem cells from human embryos and grow the cells in the laboratory. These cells are called human embryonic stem cells. The embryos used in these studies were created for reproductive purposes through in vitro fertilization procedures. When they were no longer needed for that purpose, they were donated for research with the informed consent of the donor. In 2006, researchers made another breakthrough by identifying conditions that would allow some specialized adult cells to be "reprogrammed" genetically to assume a stem cell-like state. This new type of stem cell, called induced pluripotent stem cells (iPSCs), will be discussed in a later section of this document.

Stem cells are important for living organisms for many reasons. In the 3- to 5-day-old embryo, called a blastocyst, the inner cells give rise to the entire body of the organism, including all of the many specialized cell types and organs such as the heart, lungs, skin, sperm, eggs and other tissues. In some adult tissues, such as bone marrow, muscle, and brain, discrete populations of adult stem cells generate replacements for cells that are lost through normal wear and tear, injury, or disease.

Given their unique regenerative abilities, stem cells offer new potentials for treating diseases such as diabetes, and heart disease. However, much work remains to be done in the laboratory and the clinic to understand how to use these cells for cell-based therapies to treat disease, which is also referred to as regenerative or reparative medicine.

Laboratory studies of stem cells enable scientists to learn about the cells essential properties and what makes them different from specialized cell types. Scientists are already using stem cells in the laboratory to screen new drugs and to develop model systems to study normal growth and identify the causes of birth defects.

Research on stem cells continues to advance knowledge about how an organism develops from a single cell and how healthy cells replace damaged cells in adult organisms. Stem cell research is one of the most fascinating areas of contemporary biology, but, as with many expanding fields of scientific inquiry, research on stem cells raises scientific questions as rapidly as it generates new discoveries.

I.Introduction|Next

Follow this link:
Stem Cell Basics I. | stemcells.nih.gov

Skin Stem Cells Comments Off on Stem Cell Basics I. | stemcells.nih.gov | September 20th, 2017

In simpler times, skincare choices were slotted by agetwenties, thirties, fortiesor skin type, that is, oily, dry or combination. Night creams signalled a milestone, an anti-ageing ritual you transited to once you turned forty. Not anymore. A new generation of night creams and hybrids in a jar are challenging the norm. And millennials, more concerned than ever before about the state of their skin, are discovering a range of choices on offer. Retinols, ferulic acid, hyaluronic acid, ceramides, phospholipids, stem cells, biomimetic peptides, arbutin are just some of the ingredients you find in night creams, and you have to choose keeping a combination of factors in mind, says Dr Malavika Kohli, a Mumbai-based celebrity dermatologist.

Your skin could be older than your age, at any ageAs experts repeatedly tell us, there are two kinds of ageingintrinsic and extrinsic. The first is caused by the ticking of the clock and cant be reversed, though perhaps, can be slowed down. The second is caused by factors we all face dailyharsh sun, pollutants and air conditioning, which can cause skin to age faster on the surface. We have lifestyle habits we find hard to kick like smoking and drinking. I wont even begin to talk of 4pm sugar cravings. Over-exercising and yo-yo dieting are an easy way to get sagging skinyou dont have to wait till you reach forty.

The rhythm of the nightThe day is for protection and coverage, and sunscreens, BB creams, CC creams work hard to battle environmental factors. But the night is for more intensive repair, undisturbed. The night signals a time of rest and restoration as your cells tend to be more relaxed and receptive. If you have problem skin, the night is the time to let a good anti-blemish cream go to work. If your skin is dehydrated, then a moisturising cream will deliver the benefits best at night. Many star ingredients like retinol and vitamin C, in potent form, work best out of the sun.

Cream versus serum versus lotionDo creams score over lotions? New generation night creams are often oil-free, light and easily absorbedall the things you looked for in a lotion. Thanks to their creamy nature, they texturise skin better leaving it silky and soft. Mousse, cream-gels and moisture-whipped creams are blurring the definition of traditional creams and lotions. Serums are specifically targeted to work at the cellular level and dont deliver overall surface moisture, so use a combination of that and a cream as you get older.

Understand your skinKnowing your skin type is important, but so is understanding your skin condition. Dry skin for example, says Dr Kohli, lacks sebum or oil, while dehydrated skin lacks water, but both indicate poor barrier function.

If your skin is very dehydrated on the surface, Este Edit by Este Lauders Pink Peony Overnight Water Pack targets all skin types. It gives skin an antioxidant boost with extracts of goji berry, blueberry and cranberry in a water-based gel. Garnier SkinActive Moisture Bomb, with amla and plant serums, is targeted at dry and sensitive skin. For more intense deep moisturisation try, a cream with hyaluronic acid like Revitalift Laser X3 Night Cream Mask from LOral Paris.

For skin that is irritated and sun-damaged, a cocktail of antioxidants, peptides and vitamins will help. Olay Regenerist Advanced Anti-Ageing Revitalizing Night Skin Cream has an amino-peptide complex that gets the skin into healing mode while you snooze.

Just Herbs Blemfree Anti Blemish Night Cream is SLS, petrochemical and paraben-free and will not irritate damaged skin further. It targets sun spots, uneven and patchy skin with organic sunflower oil.

Dull skin with pigmentation indicates pore-clogging debris accumulation and slow cell turnover. A cream with salicylic acid will provide much-needed but gentle exfoliation at night. Clinique Turnaround Overnight Revitalising Moisturizer is non-acnegenic and has both salicylic acid and beta hydroxy acid to speed up exfoliation.

Kama Ayurvedas Rejuvenating And Brightening Ayurvedic Night Cream has saffron, aloe vera, liquorice and manjistha (a blood purifier), which work to soothe skin and improve cell turnover.

Early crows feet and fine lines will benefit from retinoid-based creams for long-lasting results. Neutrogena Rapid Wrinkle Repair Night Moisturizer with retinol is non-greasy yet promises deep action. For a targeted solution, Yves Rocher Serum Vegetal Wrinkles & Firmness Targeted Filler Eyes And Lips works specifically on crows feet and fine lines around the eye. It can also be used under make-up in the day but use it at night for best results.

The Body Shops Pomegranate Firming Night Cream has organic oil of pomegranate and pomegranate peel, which aresupposed to deliver retinol-like resultspomegranates are a rich source of antioxidants as well.

Natural, organic, ayurvedic or chemicalfor all night creams to deliver results, wait four to six weeks. But most importantly choosing right, investing in the right skin care routine and maintaining it will be the best thing you can do, says Dr Kohli. Make the commitment. You skin will thank you for this.

Take your pick from our edit below:

Este Edit by Este Lauders Pink Peony Overnight Water Pack, Rs 3,202

Garnier SkinActive Moisture Bomb, Rs 1,088

LOral Paris Revitalift Volume Filler Night Cream, Rs 1,450

Olay Regenerist Advanced Anti-Ageing Revitalizing Night Skin Cream, Rs 1,399

Just Herbs Blemfree Anti Blemish Night Cream, Rs 895

Clinique Turnaround Overnight Revitalising Moisturizer, Rs 2,626

Kama Ayurveda Rejuvenating And Brightening Ayurvedic Night Cream, Rs 1,950

Neutrogena Rapid Wrinkle Repair Night Moisturizer, Rs 1,199

Yves Rocher Serum Vegetal Wrinkles & Firmness Targeted Filler Eyes And Lips, Rs 1,400

The Body Shops Pomegranate Firming Night Cream, Rs 1,696

Read more:
How to choose the right night cream for your skin - VOGUE India

Skin Stem Cells Comments Off on How to choose the right night cream for your skin – VOGUE India | September 8th, 2017

Cellular Renovation

Why build something from the ground up when one can just renovate an already existing structure? Essentially, thats what researchers from the University of Washington School of Medicine in St. Louis had in mind when they developed a method for transforming adult human skin cells into motor neurons in a lab. They published their work in the journal Cell Stem Cell.

In this study, we only used skin cells from healthy adults ranging in age from early 20s to late 60s, senior author Andrew S. Yoo said in a press release. Our research revealed how small RNA molecules can work with other cell signals called transcription factors to generate specific types of neurons, in this case motor neurons. In the future, we would like to study skin cells from patients with disorders of motor neurons. Our conversion process should model late-onset aspects of the disease using neurons derived from patients with the condition.

They did this by exposing skin cells in a lab to certain molecular signals usually found at high levels in the human brain. They focused on two short snippets of RNA: microRNAs (mRNAs) called miR-9 and miR-124, which are involved in repurposing the genetic instructions of the cell. These mRNAs, combined with certain transcription factors, successfully turned skin cells into spinal cord motor neurons within just 30 days. These new cells closely resembled normal mouse motor neurons in terms of which genes were turned on and off, and how they functioned.

Usually, when researchers find ways to replace damaged cells or organs, they resort to using stem cells. In particular, they use embryonic stem cells (a type of pluripotent stem cells) to grow the cells or organs needed.

While this type of stem cell has the potential to grow into whatever adult cell type is needed, the procedure carries some ethical concerns. In bypassing a stem cell phase, the new cell transformation technique doesnt have any of these ethical issues.

Keeping the original age of the converted cells can be crucial for studying neurodegenerative diseases that lead to paralysis, such as amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy, the condition the new research focused on. In particular, researchers hope that it could enhance the understanding of these diseases in order to improve regenerative medicine.

Going back through a pluripotent stem cell phase is a bit like demolishing a house and building a new one from the ground up, Yoo explained. What were doing is more like renovation. We change the interior but leave the original structure, which retains the characteristics of the aging adult neurons that we want to study.

Like embryonic stem cells, the technique can also allow for converting human skin cells into other cell types by using different transcription factors. Before this technique can be applied to actual humans with neurodegenerative diseases, the researchers still need to find out how much the cells made in their lab match native human motor neurons. Still, its a promising start.

The rest is here:
Researchers Turn Skin Cells into Motor Neurons Without Using ... - Futurism

Skin Stem Cells Comments Off on Researchers Turn Skin Cells into Motor Neurons Without Using … – Futurism | September 8th, 2017

Nancy Crotti

Researchers have developed tissue nanotransfection, a process for regrowing tissue inside the human body.

Researchers at Ohio State University have developed breakthrough stem cell technology that can regrow tissue inside the human body, rather than in a laboratory.

Their work has implications for critical limb ischemia, brain disorders, and possibly even organ engineering and bone regrowth, according to Chandan Sen, PhD, director of the Center for Regenerative Medicine and Cell-Based Therapies at Ohio State's Wexner Medical Center in Columbus. Sen led the team that developed the technology.

Here's how the process, known as nanotransfection, works: The scientists make synthetic RNA and DNA to match that of the patient. They load it into nanochannels inside tiny needles embedded in a chip and apply the chip to the skin. The needles electrocute about 2% of the cell surface with the patient's nucleic acid. The procedure takes 1/10th of a second, and has been shown to work with up to 98% efficiency.

In experiments on mice, the technology restored blood flow to injured legs by reprogramming the animals' skin cells to become vascular cells. With no other form of treatment, active blood vessels had formed within two weeks, and by the third week, blood flow returned and the legs of the mice were saved.

The researchers also induced strokes in mice and used the chips to grow new brain tissue from the animals' skin and transplant it to their brains. Bodily function damaged by the strokes was restored. The study of the technique, which worked with up to 98% efficiency, was reported in the journal Nature Nanotechnology.

The technology marks an advance over cell regeneration conducted in a laboratory, because those cells mostly underperform or die once transplanted into the body, according to Sen. The researchers use skin cells in their work because, as Sen explained, everybody has some to spare.

"We grow it in you and we move it over to the organ so you have your own cells populating your organ," he said. "It's all coming from you."

The synthetic RNA and DNA reprogram cells in the same way that fetal cells develop different functions to become different body parts, Sen added. The researchers worked on the technology for more than four years, also conducting successful blood flow restoration experiments on pigs. When they begin human trials, their first patients will likely be those whose critical limb ischemic has reached the stage where amputation is the only option.

The scientists' work has generated interest in Europe, Asia, the Middle East, and in the United States. Ohio State will decide where to pursue human trials first, and is searching for industry partners.

"The cost is extremely low and complexity-wise it is extremely low. I see very little barrier to take it to humans," Sen said.

The researchers' work marks another interface between silicon chips and biology. Other applications picked up by manufacturers include DNA sequencing machines, miniaturized diagnostic tests using disposable photonic chips, accurate body monitoring sensors, and brain stimulation probes.

Sen and his team acknowledge that their work will be met with skepticism.

"Whenever you do something that is sort of transformative, you will expect that," Sen said. "Therefore, we actually published this in the most rigorous journal possible. We went through 16 months of criticism and response, after which this was published."

Nancy Crotti is a freelance contributor to MD+DI.

[Image courtesy of THE OHIO STATE UNIVERSITY WEXNER MEDICAL CENTER]

See the article here:
'Nanotransfection' Turns Animal Skin into Blood Vessels and Brain Cells - Medical Device and Diagnostics Industry

Skin Stem Cells Comments Off on ‘Nanotransfection’ Turns Animal Skin into Blood Vessels and Brain Cells – Medical Device and Diagnostics Industry | September 8th, 2017

A Decatur elementary student and her mother are recovering following a stem cell transplant in a Cincinnati-area hospital.

It will be six to eight weeks, however, before doctors know whether the membrane Nicole Richey gave her daughter Phoenix is working.

What were praying for is that Phoenix will start producing her own stem cells, Joey Richey said by telephone Thursday.

In January, Phoenix, a fourth-grade student at Chestnut Grove Elementary, was diagnosed with Stevens-Johnsons syndrome, a rare and serious disorder of the skins mucous membranes.

According to the Mayor Clinic, the syndrome is caused by a reaction to medication or infection and begins with flu-like symptoms, which Phoenix had. The disease is followed by painful rashes and blisters that ultimately cause the top layer of skin to die.

The disease affected between 60 and 65 percent of Phoenixs body and damaged her eyes.

During the outpatient procedure, which is called a limbal stem cell transplant, said Joey Richey, surgeons took about 40 percent of the cornea from his wifes left eye and placed it in Phoenixs right eye.

Nicole Richey said she was close to a perfect match, but doctors have put her daughter on immunosuppression therapy to lower the possibility of Phoenix rejecting the stem cells.

I am still having a hard time after my surgery, but were OK, Nicole Richey said.

The procedure took place at St. Elizabeth North Kentucky Surgical Center, and Phoenix will miss about six weeks of school.

Chestnut Grove Principal Luke Bergeson said Phoenix will continue to receive homebound instructional services until she is ready to return to school.

Phoenixs body is still not able to grow its own skin, so she has been fitted with a prokera ring, which is a therapeutic device to protect her eyes. Her left eye is temporarily sewn shut and will be until doctors see how the right eye reacts to the transplant, Joey Richey said.

They are working on one eye at a time, he said.

Phoenix was diagnosed with Stevens-Johnsons syndrome while she was out of school on Christmas break last year. The outer layer of skin started to die and was peeling two days before she was admitted to Huntsville Hospital. On Jan. 12, doctors transferred Phoenix to the burn unit at Childrens Hospital of Birmingham for treatment.

She stayed there a week before being moved to Shriners Hospital for Children in Cincinnati, where she stayed until Feb. 8.

Phoenix missed the remainder of the school term, but she came back to Chestnut Grove when classes started in August.

Read more here:
Decatur elementary student gets stem cells from mother - The Decatur Daily

Skin Stem Cells Comments Off on Decatur elementary student gets stem cells from mother – The Decatur Daily | September 8th, 2017

docent/ShutterstockWhether or not theres a scientific benefit to being baldwell let the follically challenged among us be the judge of thatscientists continue to search for a balding cure. According to UCLA researchers, that isnt completely out of the question. A team, led by Heather Christofk, PhD, and William Lowry, PhD, found a new way to activate the stem cells in the hair follicle to make hair grow. Their findings, published in the journal Nature Cell Biology, may lead to new drugs to promote hair growth or work as a cure for baldness or alopecia (hair loss linked to factors like hormonal imbalance, stress, aging or chemotherapy).

Working at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, the researchers discovered that the metabolism of the stem cells embedded in hair follicles is different from the metabolism of other cells of the skin. When they altered that metabolic pathway in mice, they discovered they could either stop hair growth, or make hair grow rapidly. They did this by first blocking, then increasing, the production of a metabolitelactategenetically.

Before this, no one knew that increasing or decreasing the lactate would have an effect on hair follicle stem cells, says Dr. Lowry, a professor of molecular, cell and developmental biology, as reported on ScienceDaily. Once we saw how altering lactate production in the mice influenced hair growth, it led us to look for potential drugs that could be applied to the skin and have the same effect.

Two drugs in particularknown by the generic designations of RCGD423 and UK5099influenced hair follicle stem cells in distinct ways to promote lactate production. The use of both drugs to promote hair growth are covered by provisional patent applications. However, they are experimental drugs and have been used in preclinical tests only. They wont be ready for prime time until theyve been tested in humans and approved by the Food and Drug Administration as safe and effective. (While youre waiting for a male pattern baldness cure, check out these natural remedies for hair loss.)

So while it may be some time before these drugs are availableif everto treat baldless or alopecia, researchers are optimistic about the future. Through this study, we gained a lot of interesting insight into new ways to activate stem cells, says Aimee Flores, a predoctoral trainee in Lowrys lab and first author of the study. The idea of using drugs to stimulate hair growth through hair follicle stem cells is very promising given how many millions of people, both men and women, deal with hair loss. I think weve only just begun to understand the critical role metabolism plays in hair growth and stem cells in general; Im looking forward to the potential application of these new findings for hair loss and beyond.

This 7-year-old girl living with alopecia will inspire you.

See more here:
This New, Cutting-Edge Treatment Could Be the End of Baldness - Reader's Digest

Skin Stem Cells Comments Off on This New, Cutting-Edge Treatment Could Be the End of Baldness – Reader’s Digest | September 7th, 2017

Press Release

Researchers at Ohio State University Wexner Medical Center uncovered a novel pathway for hair follicular regeneration. Palm tocotrienol complex (EVNol SupraBio) is shown to induce hair follicle growth via protein expression of epidermal E-cadherin dependent beta-catenin - the key signaling molecule for inducing pluripotent stem cells in the adult skin.

In this study (1), male mice with mutated leptin receptor were applied with either 5mg/cm2 palm tocotrienol rich fraction (TRF) (ie. EVNol SupraBio - bioenhanced palm tocotrienol complex, supplied by ExcelVite) or placebo on shaved dorsal skin thrice per week for 21 days and the evaluation of hair growth was recorded by the color of dorsal skin. The mechanism of palm TRF-induced hair growth, the dependency on the loss of E-cadherin and the activation of beta-catenin for hair follicle formation were examined by quantification of gene expressions, immunoprecipitation and immunoblots.

When compared to placebo, palm TRF treated group showed significantly increased number of anagen (ie. cycle of growth) hair follicles, increased fetal characteristics of hair follicular development in the adult skin, increased epidermal keratinocyte proliferation, significant decreased E-cadherin expression that was associated with high translocation of beta-catenin-Tf3, leading to upregulation of gene expressions of Oct4, Sox9, Klf4, c-Myc and Nanog skin-specific pluripotent factors that support hair follicular regeneration. These factors are also known as the Yamanaka Transcription Factors discovered by Dr. Shinya Yamanaka, joint-recipient of the 2012 Nobel Prize in Physiology or Medicine. Prof. Yamanaka discovered that mature cells can be reprogrammed to become pluripotent.

The researchers concluded that palm TRF suppression of epidermal E-cadherin induced beta-catenin and nuclear translocation is the novel pathway that leads to expressions of pluripotent factors and subsequently promotes anagen hair cycling in adult skin.

What we have shown is that Palm TRF can induce hair folliculogenesis, which means that it can enrich the skin stem cell reserves. This novel epidermal pathway of hair follicular regeneration can have widespread impact on skin function including skin aging and repair, says Prof. Chandan Sen, the lead researcher at Ohio State University Wexner Medical Center.

Prior to the above discovery, researchers from University Science Malaysia had reported and patented the unique benefits of tocotrienols (EVNol SupraBio) in supporting hair growth in subjects with on-going hair loss (2).

We are thrilled with this new discovery, especially this novel pathway that affirmed our previous clinical findings for EVNol SupraBio in hair growth, (US Patent No: 7,211,274; Trop. Life Sci. Res. 2010). Taken together this latest study and previous published papers explain the mechanism as to how EVNol SupraBio may help in promoting hair growth in subjects experiencing hair loss, says Bryan See, Business Development Manager, ExcelVite.

Source:

About ExcelVite

ExcelVite Sdn. Bhd., incorporated in Malaysia in 2013, is the leading and largest producer of natural full spectrum tocotrienol / tocopherol complex (EVNol, and EVNol SupraBio), natural mixed-carotene complex (EVTene), phytosterol complex (EVRol), and red palm oil concentrate (EVSpectra) in the world via a patented technology.

ExcelVite is the only tocotrienol producer that operates in accordance to GMP (PIC/S) Guide to Good Manufacturing Practice for Medicinal Products. Its laboratory is accredited with ISO/IEC 17025 accreditation.

EVNol SupraBio is a patented (US Patent No. 6,596,306) self-emulsifying palm tocotrienol complex that ensures optimal tocotrienols oral absorption.

ExcelVite manufactures and markets its products under the tradenames: EVNol, EVNol SupraBio, EVTene, EVRol, and EVSpectra. These branded ingredients are Non-GMO, Kosher and Halal certified. ExcelVite supports the production of certified sustainable palm oil (CSPO) through RSPO Credits.

Websites:www.excelvite.com andwww.tocotrienol.org

The rest is here:
Latest Research Unveiled Novel Pathway For T3 In Hair Follicle Regeneration - Natural Products INSIDER

Skin Stem Cells Comments Off on Latest Research Unveiled Novel Pathway For T3 In Hair Follicle Regeneration – Natural Products INSIDER | September 7th, 2017

GUANGZHOU, China, Sept. 5, 2017 /PRNewswire/ -- Chinese Grand Fan Group formally signed the agreement to acquire the French CICABEL brand on September 4th. Grand Fan Group is openly optimistic about CICABEL's technology and development prospects, while the investment into the French brand represents the first step in the execution of the strategy behind the group's entry into the skin care market. The signing ceremony took place in France.

Santinov is a 130-year-old French traditional pharmaceutical manufacturer founded in 1887. Santinov created and launched the CICABEL Mask, a three-step revitalizing and hydration face mask set using stem cells as the principal component, following years of research and development on the back of strong technological competence. At variance with traditional skin care products, the set is expected to become a disruptor and transform the public's expectations from the beauty industry.

A Grand Fan Group executive said "By adopting the management and operations model commonly deployed by international brands, we put in place partnerships with several leading international beauty and health brands based on our own brand, achieving a diversified brand scenario as well as access to advanced technology R&D. These moves will serve to offer more and better choices to consumers."

With the enhancement of the general public's awareness of skin care, traditional skin care products no longer meet the basic expectations and needs of consumers. Brands with an ill-defined image or a hodge-podge of seemingly unrelated products, uneven quality, inadequate supervision and other issues have led the industry to be subject to a high level of criticism. To add insult to injury, most traditional skin care products actually do little for the skin. In line with accepted biotechnology and medical standards, the CICABEL Mask is expected to reverse the perception.

Through the activation of skin stem cells, the mask provides nutrition that penetrates deep into the dermis and promotes the regeneration of new cells, delivering an in-depthreplenishment effect. Put in another way, CICABEL uses the body's own multifunctional cells to achieve a new level of skin beauty. The CICABEL Mask from France is expectedto become the "Terminator" of traditional masks available in the market.

CICABEL will formally go on sale in China soon, with plans for roll outs in several global markets shortly thereafter.

Contact: +86-400-639-1958, rel="nofollow">hantao@1958difo.com

View original content with multimedia:http://www.prnewswire.com/news-releases/china-based-grand-fan-group-acquires-leading-french-skincare-brand-300513684.html

SOURCE CICABEL

View post:
China-based Grand Fan Group acquires leading French skincare brand - Markets Insider

Skin Stem Cells Comments Off on China-based Grand Fan Group acquires leading French skincare brand – Markets Insider | September 7th, 2017

Microneedling has quickly become one of the most popular skin rejuvenation treatments. If youre considering trying it, here is what you need to know.

Microneedling, also called collagen-induction therapy, uses small needles that pierce the outermost layer of skin to create tiny microchannels. These microchannels help stimulate the production of collagen and elastin within the skin. They also promote new capillaries.

This can lead to an improved skin texture, reduction of acne or other scarring and help with discoloration, such as brown spots caused by sun damage. Microneedling may be combined with platelet-rich plasma, stem cells, or pure hyaluronic acid to enhance results further.

Microneedling can also be used on the scalp to help stimulate hair rejuvenation.

Prior to your first microneedling session, you will be asked to avoid sun exposure for at least 24 hours. Some doctors will tell you to avoid blood-thinning medications and herbal supplements like aspirin, ibuprofen and St. Johns wort to reduce bruising.

Each microneedling session takes about 20 to 30 minutes. First, your face will be cleansed and a numbing cream will be applied. Multiple treatment sessions, spaced a few weeks apart, are recommended. Most doctors recommend three to six treatments but many will notice an improvement in the tone and texture of their skin after just one treatment.

Immediately after your microneedling session, you will likely notice some redness that can last for several days. In my practice, we recommend that patients do not touch their face for at least four hours after treatment and do not apply anything to the face for 24 hours. It is crucial to avoid sun exposure for three days after the procedure.

You should avoid strenuous activity and exercise for the first 12 hours after treatment to prevent redness and bruising. For the first three days after treatment, you should use a gentle non-foaming cleanser, a barrier repair moisturizer, and a physical SPF. If swelling or bruising are a concern, you can take arnica supplements both before and after treatment to help minimize these side effects.

Once any redness or swelling diminishes, you should notice an immediate improvement in the way your skin looks and feels. Over the next several weeks, your skins appearance should continue to improve.

Read the rest here:
What is microneedling and why is the skin treatment so popular? - Miami Herald

Skin Stem Cells Comments Off on What is microneedling and why is the skin treatment so popular? – Miami Herald | September 5th, 2017

Chemical changes in the eye that can lead to blindness have been identified by scientists, a conference will hear tomorrow (Tuesday 5 September).

Their findings aid understanding of a genetic condition that causes sight loss for one in 3,000 people in the UK.

They will be presented at the Eye Development and Degeneration 2017 conference in Edinburgh.

Eye condition

Scientists examined how changes in a gene known as RPGR damage eye cells to cause a disorder known as X-linked retinitis pigmentosa.

The condition is incurable and affects night and peripheral vision before gradually causing blindness in middle age.

Skin samples

A team led by Edinburgh researchers took skin samples from two patients and transformed stem cells which can change into any cell type into light-sensing eye cells known as photoreceptors.

They compared these with cells from healthy relatives of the patients.

Photoreceptors which decay in retinitis pigmentosa patients differed in their fundamental structure when compared with those from family members.

Key molecules

Follow-up studies in mice identified key molecules that interact with RPGR to maintain the structure of photoreceptors.

When RPGR is flawed, the structure is compromised and photoreceptors cannot function correctly, leading to sight loss.

The study is published in the journal Nature Communications and was carried out at the University of Edinburgh's Medical Research Council Centre for Regenerative Medicine.

It was funded by the Wellcome Trust and the charity Retinitis Pigmentosa Fighting Blindness.

"By furthering our understanding of the RPGR gene and its effects on photoreceptor cells, we hope our findings bring us closer to developing a possible treatment for this devastating disease," says Dr Roly Megaw of the Medical Research Council Institute for Genetics and Molecular Medicine.

Explore further: New trial for blindness rewrites the genetic code

See the article here:
Gene study sheds light on middle-age sight loss - Medical Xpress

Skin Stem Cells Comments Off on Gene study sheds light on middle-age sight loss – Medical Xpress | September 4th, 2017

Monkeys with Parkinsons disease symptoms show significant improvement over two years after being transplanted neurons prepared from human induced pluropontent stem cells, scientists at the Center for iPS Cell Research and Application (CiRA), Kyoto University, report. One of the last steps before treating patients with an experimental cell therapy for the brain is confirmation that the therapy works in monkeys.

Parkinsons disease degenerates a specific type of cells in the brain known as dopaminergic (DA) neurons. It has been reported that when symptoms are first detected, a patient will have already lost more than half of his or her DA neurons.

Several studies have shown the transplantation of DA neurons made from fetal cells can mitigate the disease.

The use of fetal tissues is controversial, however. On the other hand, iPS cells can be made from blood or skin.

Our research has shown that DA neurons made from iPS cells are just as good as DA neurons made from fetal midbrain. Because iPS cells are easy to obtain, we can standardize them to only use the best iPS cells for therapy,

said Professor Jun Takahashi, a neurosurgeon specializing in Parkinsons disease, who plans to use DA neurons made from iPS cells to treat patients.

To test the safety and effectiveness of DA neurons made from human iPS cells, Tetsuhiro Kikuchi, a neurosurgeon working in the Takahashi lab, transplanted the cells into the brains of monkeys.

We made DA neurons from different iPS cells lines. Some were made with iPS cells from healthy donors. Others were made from Parkinsons disease patients,

said Kikuchi, who added that the differentiation method used to convert iPS cells into neurons is suitable for clinical trials.

It is generally assumed that the outcome of a cell therapy will depend on the number of transplanted cells that survive, but Kikuchi found this was not the case. More important than the number of cells was the quality of the cells.

Each animal received cells prepared from a different iPS cell donor. We found the quality of donor cells had a large effect on the DA neuron survival, Kikuchi said.

To understand why, he looked for genes that showed different expression levels, finding 11 genes that could mark the quality of the progenitors. One of those genes was Dlk1.

Dlk1 is one of the predictive markers of cell quality for DA neurons made from embryonic stem cells and transplanted into rat. We found Dlk1 in DA neurons transplanted into monkey. We are investigating Dlk1 to evaluate the quality of the cells for clinical applications.

Another feature of the study that is expected to extend to clinical study is the method used to evaluate cell survival in the host brains. The study demonstrated that magnetic resonance imaging (MRI) and position electron tomography (PET) are options for evaluating the patient post surgery.

MRI and PET are non-invasive imaging modalities. Following cell transplantation, we must regularly observe the patient. A non-invasive method is preferred,

said Takahashi.

The group is hopeful that it can begin recruiting patients for this iPS cell-based therapy before the end of next year. The study is the teams answer to bring iPS cells to clinical settings, said Takahashi.

Tetsuhiro Kikuchi, Asuka Morizane, Daisuke Doi, Hiroaki Magotani, Hirotaka Onoe, Takuya Hayashi, Hiroshi Mizuma, Sayuki Takara, Ryosuke Takahashi, Haruhisa Inoue, Satoshi Morita, Michio Yamamoto, Keisuke Okita, Masato Nakagawa, Malin Parmar, Jun TakahashiHuman iPS cell-derived dopaminergic neurons function in a primate Parkinsons disease modelNature, 2017; 548 (7669): 592 DOI: 10.1038/nature23664

Image: Annie Cavanagh / Wellcome Images

Continue reading here:
iPS Cell-based Neuron Therapy Benefits Monkeys With Parkinson's - ReliaWire

Skin Stem Cells Comments Off on iPS Cell-based Neuron Therapy Benefits Monkeys With Parkinson’s – ReliaWire | September 3rd, 2017

| Marlowe Hood |

PARIS (AFP) Lab monkeys with Parkinsons symptoms regained significant mobility after neurons made from human stem cells were inserted into their brains, researchers reported Wednesday in a study hailed as groundbreaking.

The promising results were presented as the last step before human clinical trials, perhaps as early as next year, the studys senior author, Jun Takahashi, a professor at Kyoto University, told AFP.

Parkinsons is a degenerative disease that erodes motor functions. Typical symptoms include shaking, rigidity and difficulty walking. In advanced stages, depression, anxiety and dementia are also common.

Worldwide, about 10 million people are afflicted with the disease, according to the Parkinsons Disease Foundation.

Earlier experiments had shown improvements in patients treated with stem cells taken from human foetal tissue and likewise coaxed into the dopamine-producing brain cells that are attacked by Parkinsons.

Dopamine is a naturally occurring chemical that plays several key roles in the brain and body.

But the use of foetal tissue is fraught with practical and ethical problems.

So Takahashi and his colleagues, in a medical first, substituted so-called induced pluripotent stem cells (iPSCs), which can be easily made from human skin or blood. Within a year, some monkeys who had could barely stand up gradually recovered mobility.

They became more active, moving more rapidly and more smoothly, Takahashi said by email. Animals that had taken to just sitting start walking around in the cage.

These findings are strong evidence that human iPSC-derived dopaminergic neurons can be clinically applicable to treat Parkinsons patients, he said.

Experts not involved in the research described the results as encouraging.

The treatment, if proven viable, has the potential to reverse Parkinsons by replacing the dopamine cells that have been lost a groundbreaking feat, said David Dexter, deputy research director at Parkinsons UK.

Not only did the new cells survive but they also integrated with the existing neuronal network, he said.

Neurons made from foetal tissue grafted into brains have been known to survive for more than a decade, and the researchers said they expected those derived from iPSCs to last just as long.

Tilo Kunath, Parkinsons Senior Research Fellow at the University of Edinburgh, said the outcome was extremely promising, and highlighted the advantage of avoiding stem cells extracted from human foetal tissue.

It means that this therapy can be used in any country worldwide, including Ireland and most of South America, where medical use of human embryonic stem cells is banned.

The results, reported in the journal Nature, were not the same for the dozen monkeys in the experiment, each of which received donor neurons from a different person.

Some were made with cells from healthy donors, while others were made from Parkinsons disease patients, said lead author Tetsuhiro Kikuchi, also from Kyoto University.

See original here:
Study shows human stem cells restore mobility in Parkinson's monkeys - Borneo Bulletin Online

Skin Stem Cells Comments Off on Study shows human stem cells restore mobility in Parkinson’s monkeys – Borneo Bulletin Online | September 1st, 2017