Other common name(s): cellular therapy, fresh cell therapy, live cell therapy, glandular therapy, xenotransplant therapy

Scientific/medical name(s): none

In cell therapy, processed tissue from the organs, embryos, or fetuses of animals such as sheep or cows is injected into patients. Cell therapy is promoted as an alternative form of cancer treatment.

Available scientific evidence does not support claims that cell therapy is effective in treating cancer or any other disease. Serious side effects can result from cell therapy. It may in fact be lethalseveral deaths have been reported. It is important to distinguish between this alternative method involving animal cells and mainstream cancer treatments that use human cells, such as bone marrow transplantation.

In cell therapy, live or freeze-dried cells or pieces of cells from the healthy organs, fetuses, or embryos of animals such as sheep or cows are injected into patients. This is supposed to repair cellular damage and heal sick or failing organs. Cell therapy is promoted as an alternative therapy for cancer, arthritis, heart disease, Down syndrome, and Parkinson disease.

Cell therapy is also marketed to counter the effects of aging, reverse degenerative diseases, improve general health, increase vitality and stamina, and enhance sexual function. Some practitioners have proposed using cell therapy to treat AIDS patients.

The theory behind cell therapy is that the healthy animal cells injected into the body can find their way to weak or damaged organs of the same type and stimulate the body's own healing process. The choice of the type of cells to use depends on which organ is having the problem. For instance, a patient with a diseased liver may receive injections of animal liver cells. Most cell therapists today use cells taken from taken from the tissue of animal embryos.

Supporters assert that after the cells are injected into the body, they are transported directly to where they are most needed. They claim that embryonic and fetal animal tissue contains therapeutic agents that can repair damage and stimulate the immune system, thereby helping cells in the body heal.

The alternative treatment cell therapy is very different from some forms of proven therapy that use live human cells. Bone marrow transplants infuse blood stem cellsfrom the patient or a carefully matched donorafter the patients own bone marrow cells have been destroyed. Studies have shown that bone marrow transplants are effective in helping to treat several types of cancer. In another accepted procedure, damaged knee cartilage can be repaired by taking cartilage cells from the patient's knee, carefully growing them in the laboratory, and then injecting them back into the joint. Approaches involving transplants of other types of human stem cells are being studied as a possible way to replace damaged nerve or heart muscle cells, but these approaches are still experimental.

First, healthy live cells are harvested from the organs of juvenile or adult live animals, animal embryos, or animal fetuses. These cells may be taken from the brain, pituitary gland, thyroid gland, thymus gland, liver, kidney, pancreas, spleen, heart, ovaries, testicles, or even from whole embryos. Patients might receive one or several types of animal cells. Some cell therapists inject fresh cells into their patients. Others freeze them first, which kills the cells, and they may filter out some of the cell components. Frozen cell extracts have a longer "shelf life" and can be screened for disease. Fresh cells cannot be screened. A course of cell therapy to address a specific disease might require several injections over a short period of time, whereas cell therapy designed to treat the effects of aging and "increase vitality" may involve injections received over many months.

More here:
Cell Therapy - American Cancer Society

Autism treatment with stem cells
Stem cell therapy for autism treatment in UCTC. Professor Smikodub introduced the method of fetal stem cell therapy on the basis of which autism treatment wa...

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Autism treatment with stem cells - Video

http://www.CLINICell.com "ACL TEAR alternative with PRP and Stem Cell Therapy"

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http://www.CLINICell.com "ACL TEAR alternative with PRP and Stem Cell Therapy" - Video

For immediate release: September 10, 2012

Baltimore, MD --Researchers at the University of Maryland School of Medicine, who are exploring novel ways to treat serious heart problems in children, have conducted the first direct comparison of the regenerative abilities of neonatal and adult-derived human cardiac stem cells. Among their findings: cardiac stem cells (CSCs) from newborns have a three-fold ability to restore heart function to nearly normal levels compared with adult CSCs. Further, in animal models of heart attack, hearts treated with neonatal stem cells pumped stronger than those given adult cells. The study is published in the September 11, 2012, issue of Circulation.

The surprising finding is that the cells from neonates are extremely regenerative and perform better than adult stem cells, says the study's senor author, Sunjay Kaushal, M.D., Ph.D., associate professor of surgery at the University of Maryland School of Medicine and director, pediatric cardiac surgery at the University of Maryland Medical Center. We are extremely excited and hopeful that this new cell-based therapy can play an important role in the treatment of children with congenital heart disease, many of whom don't have other options.

Dr. Kaushal envisions cellular therapy as either a stand-alone therapy for children with heart failure or an adjunct to medical and surgical treatments. While surgery can provide structural relief for some patients with congenital heart disease and medicine can boost heart function up to two percent, he says cellular therapy may improve heart function even more dramatically. We're looking at this type of therapy to improve heart function in children by 10, 12, or 15 percent. This will be a quantum leap in heart function improvement.

Heart failure in children, as in adults, has been on the rise in the past decade and the prognosis for patients hospitalized with heart failure remains poor. In contrast to adults, Dr. Kaushal says heart failure in children is typically the result of a constellation of problems: reduced cardiac blood flow; weakening and enlargement of the heart; and various congenital malformations. Recent research has shown that several types of cardiac stem cells can help the heart repair itself, essentially reversing the theory that a broken heart cannot be mended.

Stem cells are unspecialized cells that can become tissue- or organ-specific cells with a particular function. In a process called differentiation, cardiac stem cells may develop into rhythmically contracting muscle cells, smooth muscle cells or endothelial cells. Stem cells in the heart may also secrete growth factors conducive to forming heart muscle and keeping the muscle from dying.

To conduct the study, researchers obtained a small amount of heart tissue during normal cardiac surgery from 43 neonates and 13 adults. The cells were expanded in a growth medium yielding millions of cells. The researchers developed a consistent way to isolate and grow neonatal stem cells from as little as 20 milligrams of heart tissue. Adult and neonate stem cell activity was observed both in the laboratory and in animal models. In addition, the animal models were compared to controls that were not given the stem cells.

Dr. Kaushal says it is not clear why the neonatal stem cells performed so well. One explanation hinges on sheer numbers: there are many more stem cells in a baby's heart than in the adult heart. Another explanation: neonate-derived cells release more growth factors that trigger blood vessel development and/or preservation than adult cells.

This research provides an important link in our quest to understand how stem cells function and how they can best be applied to cure disease and correct medical deficiencies, says E. Albert Reece, M.D., Ph.D., M.B.A., vice president for medical affairs, University of Maryland; the John Z. and Akiko K. Bowers Distinguished Professor; and dean, University of Maryland School of Medicine. Sometimes simple science is the best science. In this case, a basic, comparative study has revealed in stark terms the powerful regenerative qualities of neonatal cardiac stem cells, heretofore unknown.

Insights gained through this research may provide new treatment options for a life-threatening congenital heart syndrome called hypoplastic left heart syndrome (HLHS). Dr. Kaushal and his team will soon begin the first clinical trial in the United States to determine whether the damage to hearts of babies with HLHS can be reversed with stem cell therapy. HLHS limits the heart's ability to pump blood from the left side of the heart to the body. Current treatment options include either a heart transplant or a series of reconstructive surgical procedures. Nevertheless, only 50-60 percent of children who have had those procedures survive to age five.

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Cardiac Stem Cells (CSCs) | University of Maryland Medical Center

Surgeon performs bone marrow harvest

The terms "Hodgkin's Disease," "Hodgkin's Lymphoma," and "Hodgkin Lymphoma" are used interchangeably throughout this site.

Bone Marrow Transplants (BMT) and Peripheral Blood Stem Cell Transplants (PBSCT) are emerging as mainstream treatment for many cancers, including Hodgkin's Disease and Medium/High grade aggressive)Non-Hodgkin's lymphoma.

BMTs have been used to treat lymphoma for more than 10 years, but until recently they were used mostly within clinical trials. Now BMTs are being used in conjunction with high doses of chemotherapy as a mainstream treatment.

When high doses of chemotherapy are planned, which can destroy the patients bone marrow, physicians will typically remove marrow from the patients bone before treatment and freeze it. After chemotherapy, the marrow is thawed and injected into a vein to replace destroyed marrow. This type of transplant is called an autologous transplant. If the transplanted marrow is from another person, it is called an allogeneic transplant.

In PBSCTs, another type of autologous transplant, the patient's blood is passed through a machine that removes the stem cells the immature cells from which all blood cells develop. This procedure is called apheresis and usually takes three or four hours over one or more days. After treatment to kill any cancer cells, the stem cells are frozen until they are transplanted back to the patient. Studies have shown that PBSCTs result in shorter hospital stays and are safer and more cost effective than BMTs.

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Stem Cell Transplants and Bone Marrow Transplant to Treat Lymphoma

There are 3 possible sources of stem cells to use for transplants: bone marrow, the bloodstream (peripheral blood), and umbilical cord blood from newborns. Although bone marrow was the first source used in stem cell transplant, peripheral blood is used most often today.

Bone marrow is the spongy tissue in the center of bones. Its main job is to make blood cells that circulate in your body and immune cells that fight infection.

Bone marrow was the first source used for stem cell transplants because it has a rich supply of stem cells. The bones of the pelvis (hip) contain the most marrow and have large numbers of stem cells in them. For this reason, cells from the pelvic bone are used most often for a bone marrow transplant. Enough marrow must be removed to collect a large number of healthy stem cells.

For a bone marrow transplant, the donor gets general anesthesia (drugs are used to put the patient into a deep sleep so they dont feel pain). A large needle is put through the skin and into the back of the hip bone. The thick, liquid marrow is pulled out through the needle. This is repeated several times until enough marrow has been taken out (harvested). (For more on this, see the section called Whats it like to donate stem cells?)

The harvested marrow is filtered, stored in a special solution in bags, and then frozen. When the marrow is to be used, its thawed and then given just like a blood transfusion. The stem cells travel to the recipients bone marrow. There over time, they engraft or take and begin to make blood cells. Signs of the new blood cells usually can be measured in the patients blood tests in about 2 to 4 weeks.

Normally, few stem cells are found in the blood. But giving hormone-like substances called growth factors to stem cell donors a few days before the harvest causes their stem cells to grow faster and move from the bone marrow into the blood.

For a peripheral blood stem cell transplant, the stem cells are taken from blood. A very thin flexible tube (called a catheter) is put into one of the donors veins and attached to tubing that carries the blood to a special machine. The machine separates the blood, and keeps only the stem cells. The rest of the blood goes back to the donor. This takes several hours, and may need to be repeated for a few days to get enough stem cells. The stem cells are filtered, stored in bags, and frozen until the patient is ready for them. (For more on this, see the section called Whats it like to donate stem cells?)

After the patient is treated with chemo and/or radiation, the stem cells are given in an infusion much like a blood transfusion. The stem cells travel to the bone marrow, engraft, and then grow and make new, normal blood cells. The new cells are usually found in the patients blood a few days sooner than when bone marrow stem cells are used, usually in about 10 to 20 days.

Not everyone who needs an allogeneic stem cell transplant can find a well-matched donor among family members or among the people who have signed up to donate. For these patients, umbilical cord blood may be a source of stem cells. Around 30% of unrelated hematopoietic stem cell transplants are done with cord blood.

A large number of stem cells are normally found in the blood of newborn babies. After birth, the blood that is left behind in the placenta and umbilical cord (known as cord blood) can be taken and stored for later use in a stem cell transplant. The cord blood is frozen until needed.

More:
Sources of stem cells for transplant - American Cancer Society

Leukemia is a cancer of white blood cells, or leukocytes. Like other blood cells, leukocytes develop from somatic stem cells. Mature leukocytes are released into the bloodstream, where they work to fight off infections in our bodies.

Leukemia results when leukocytes begin to grow and function abnormally, becoming cancerous. These abnormal cells cannot fight off infection, and they interfere with the functions of other organs.

Successful treatment for leukemia depends on getting rid of all the abnormal leukocytes in the patient, allowing healthy ones to grow in their place. One way to do this is through chemotherapy, which uses potent drugs to target and kill the abnormal cells. When chemotherapy alone can't eliminate them all, physicians sometimes turn to bone marrow transplants.

In a bone marrow transplant, the patient's bone marrow stem cells are replaced with those from a healthy, matching donor. To do this, all of the patient's existing bone marrow and abnormal leukocytes are first killed using a combination of chemotherapy and radiation. Next, a sample of donor bone marrow containing healthy stem cells is introduced into the patient's bloodstream.

If the transplant is successful, the stem cells will migrate into the patient's bone marrow and begin producing new, healthy leukocytes to replace the abnormal cells.

New evidence suggests that bone marrow stem cells may be able to differentiate into cell types that make up tissues outside of the blood, such as liver and muscle. Scientists are exploring new uses for these stem cells that go beyond diseases of the blood.

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Stem Cells In Use - Learn Genetics

A bone marrow transplant is when special cells (called stem cells) that are normally found in the bone marrow are taken out, filtered, and given back either to the same person or to another person.

In diseases such as leukemia and aplastic anemia, the bone marrow is unhealthy. The purpose of a bone marrow transplant is to replace unhealthy stem cells withhealthy ones. This can treat or even cure the disease.

If a family member does not match the recipient, the National Marrow Donor Program Registry database can be searched for an unrelated individual whose tissue type is a close match. It is more likely that a donor who comes from the same racial or ethnic group as the recipient will have the same tissue traits. The chances of a minority person in the United States finding a registry match are lower than that of a white person (see article, Marrow Matches For Minorities Are Harder to Find).

If stem cells are collected by bone marrow harvest (much less likely), the donor will go to the operating room and while asleep under anesthesia, a needle will be inserted into either the hip or the breastbone to take out some bone marrow. After awakening, he/she may feel some pain where the needle was inserted.

Serious problems can occur during the time that the bone marrow is gone or very low. Infections are common, as is anemia, and low platelets in the blood can cause dangerous bleeding internally. Recipients often receive blood transfusions to treat these problems while they are waiting for the new stem cells to start growing.

When a person volunteers to be a donor, his/her particular blood tissue traits, as determined by a special blood test (histocompatibility antigen test), are recorded in the Registry. This "tissue typing" is different than a person's A, B, or O blood type. The Registry record also contains contact information for the donor, should a tissue type match be made.

Note: The author has been a registered donor since 1993.

Source:

"The Donation Procedure." Donor Information. Oct 2005. National Marrow Donor Program. 25 Jul 2007.

More here:
Bone Marrow Transplants - How They Work - About.com Rare Diseases

If youve been following skin care innovations for the last year or so, chances are youve heard about stem cells or have seen the ingredient pop up in various skin creams and serums. Stem cells are said to be able to make skin look refreshed and young, but many of us still have questions. What are they, exactly? Where do they come from? How do they work? Why should we try them? We took a closer look at the products and ingredients behind them to give you the scoop.

Stem cells, which occur in living organisms (including the human body), are different from other cells for two reasons. One, they are capable of renewing themselves, and two, under certain conditions, they can be induced to become cells that serve specific functions for the organism. Theyre important because of their regenerative propertiesstem cells offer a new way to treat certain diseases, and are often used in labs for screening new drugs and other biological research.

The idea behind stem cells in skin care is that by applying them topically, we might stimulate the growth of more stem cells. And because they can regenerate, theyll keep our skin looking youthful and healthy. Most stem cells used in beauty products are derived from plants. And while embryonic stem cells, taken from human embryos, are illegal, one brand we tried actually uses non-embryonic human cells that were extracted from consenting egg donors (yes, really. Read more below; for more general info on stem cells, read this guide from theNational Institutes of Health).

Short answer: we dont entirely know yet. Some research suggests that skin products containing stem cells can stimulate cell turnover and boost collagen, but there isnt a lot of conclusive evidence on the subject. Of course, that doesnt stop skin care companies from capitalizing on the buzzword. And we gotta say, the stem cell treatments we have tried certainly seem to be more effective and fast-acting than your average anti-agers.

Plant-derived stem cells typically are obtained from plants and fruits that can stay fresh for a long time or regenerate on their own, like Swiss apples, gotu kola, and grapes. Extracts of these stem cells are added to products to help neutralize free radicals and fight signs of aging and sun.

Apple: Indie Lee Swiss Apple Facial Serum

After scraping away bark from a particular tree species in Switzerland, scientists found that the tree was capable of regenerating itself. So to continue their research, they isolated the stem cells and tried them as a preservative on top of a tray full of apples and bananas. The team discovered that the stem cells actually prolonged the life of the fruits. Indie Lees Swiss Apple Facial Serum was created around the resilient power of these natural botanical-based stem cells. In addition to the extract from the rare Swiss apple stem cell, the serum contains hyaluronic acid and is highly concentratedyou need one drop for your entire face! Antioxidants and cell production-boosting benefits make this the perfect anti-aging product to add to your regimen.

Faspberry: Erno Laszlo Phormula 3-9 Repair Cream

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What Can Stem Cells Really Do For Your Skin? | Beautylish

What are stem cells?

Stem cells are cells that have the potential to develop into many different or specialized cell types. Stem cells can be thought of as primitive, "unspecialized" cells that are able to divide and become specialized cells of the body such as liver cells, muscle cells, blood cells, and other cells with specific functions. Stem cells are referred to as "undifferentiated" cells because they have not yet committed to a developmental path that will form a specific tissue or organ. The process of changing into a specific cell type is known as differentiation. In some areas of the body, stem cells divide regularly to renew and repair the existing tissue. The bone marrow and gastrointestinal tract are examples areas in which stem cells function to renew and repair tissue.

The best and most readily understood example of a stem cell in humans is that of the fertilized egg, or zygote. A zygote is a single cell that is formed by the union of a sperm and ovum. The sperm and the ovum each carry half of the genetic material required to form a new individual. Once that single cell or zygote starts dividing, it is known as an embryo. One cell becomes two, two become four, four become eight, eight to sixteen, and so on; doubling rapidly until it ultimately creates the entire sophisticated organism. That organism, a person, is an immensely complicated structure consisting of many, many, billions of cells with functions as diverse as those of your eyes, your heart, your immune system, the color of your skin, your brain, etc. All of the specialized cells that make up these body systems are descendants of the original zygote, a stem cell with the potential to ultimately develop into all kinds of body cells. The cells of a zygote are totipotent, meaning that they have the capacity to develop into any type of cell in the body.

The process by which stem cells commit to become differentiated, or specialized, cells is complex and involves the regulation of gene expression. Research is ongoing to further understand the molecular events and controls necessary for stem cells to become specialized cell types.

Stem Cells - Experience Question: Please describe your experience with stem cells.

Stem Cells - Umbilical Cord Question: Have you had your child's umbilical cord blood banked? Please share your experience.

Stem Cells - Available Therapies Question: Did you or someone you know have stem cell therapy? Please discuss your experience.

Medical Author:

Melissa Conrad Stppler, MD, is a U.S. board-certified Anatomic Pathologist with subspecialty training in the fields of Experimental and Molecular Pathology. Dr. Stppler's educational background includes a BA with Highest Distinction from the University of Virginia and an MD from the University of North Carolina. She completed residency training in Anatomic Pathology at Georgetown University followed by subspecialty fellowship training in molecular diagnostics and experimental pathology.

Medical Editor:

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Stem Cells - Types, Uses, and Therapies - MedicineNet

When pluripotent stem cells are made from a patients own cells, it may be also be possible to replace the faulty gene that caused their disease with a normal, healthy copy. The repaired stem cells could then be directed to form the tissue type needed, introduced into the body, allowed to divide, and used to reconstitute the diseased tissue. It's a treatment that should last a lifetime.

Boston Childrens Hospital researcher George Q. Daley, MD, PhD, then at the Whitehead Institute, was the first to demonstrate, in 2002, that pluripotent stem cells could successfully treat a disease. Working with mice that possess a genetic defect caused by an immune deficiency, the research team created genetically-matched embryonic stem cells through nuclear transfer, introduced corrective genes, then derived healthy blood stem cells and infused them into the mice, partially restoring their immune function. Daley, Director of Stem Cell Transplantation at Childrens, would like to do the same for his patients with blood diseases.

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Combining Stem Cell Therapy with Gene Therapy | Boston ...

Cell therapy (or Cellular therapy) is therapy in which cellular material is injected into a patient.[1]

Cell therapy originated in the nineteenth century when scientists experimented by injecting animal material in an attempt to prevent and treat illness.[2] Although such attempts produced no positive benefit, further research found in the mid twentieth century that human cells could be used to help prevent the human body rejecting transplanted organs, leading in time to successful bone marrow transplantation.[3]

Today two distinct categories of cell therapy are recognized.[1]

The first category is cell therapy in mainstream medicine. This is the subject of intense research and the basis of potential therapeutic benefit.[4] Such research, especially when it involves human embryonic material, is controversial.

The second category is in alternative medicine, and perpetuates the practice of injecting animal materials in an attempt to cure disease. This practice, according to the American Cancer Society, is not backed by any medical evidence of effectiveness, and can have deadly consequences.[1]

Cell therapy can be defined as therapy in which cellular material is injected into a patient.[1]

There are two branches of cell therapy: one is legitimate and established, whereby human cells are transplanted from a donor to a patient; the other is dangerous alternative medicine, whereby injected animal cells are used to attempt to treat illness.[1]

The origins of cell therapy can perhaps be traced to the nineteenth century, when Charles-douard Brown-Squard (18171894) injected animal testicle extracts in an attempt to stop the effects of aging.[2] In 1931 Paul Niehans (18821971) who has been called the inventor of cell therapy attempted to cure a patient by injecting material from calf embryos.[1] Niehans claimed to have treated many people for cancer using this technique, though his claims have never been validated by research.[1]

In 1953 researchers found that laboratory animals could be helped not to reject organ transplants by pre-innoculating them with cells from donor animals; in 1968, in Minnesota, the first successful successful human bone marrow took place.[3]

Bone marrow transplants have been found to be effective, along with some other kinds of human cell therapy for example in treating damaged knee cartilage.[1] In recent times, cell therapy using human material has been recognized as an important field in the treatment of human disease.[4] The experimental field of Stem cell therapy has shown promise for new types of treatment.[1]

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Cell therapy - Wikipedia, the free encyclopedia

Stem cells: When will they heal the heart?

Its been 15 years since a University of Wisconsin-Madison researcher isolated embryonic stem cells the do-anything cells that appear in early development. Its been six years since adult human cells were transformed into the related induced pluripotent stem cells.

ENLARGE

Some day, stem cell therapy could restore cells, save hearts, and avoid the need for some heart transplants, such as this one. This heart is ready for its new home.

And yet the early hope to grow spare parts turning stem cells into specialized cells for repairing a failing brain, pancreas or heart, remains mostly promise rather than reality.

Researchers have since found how to transform stem cells into a wide variety of body cells, including heart muscle cells, or cardiomyocytes. But the holy Grail tissue supplementation or replacement remains tantalizingly out of reach.

Last week, Why Files attended a symposium on treating cardiovascular disease with stem cells, at the BioPharmaceutical Technology Center Institute near Madison, Wis. We found the picture unexpectedly complicated: as multiple kinds of stem cells are grown and delivered in a bewildering variety of ways to treat a catalog of conditions.

So far, stem cells have not been approved to treat any heart disease in the United States.

Still, the need remains clear. Disorders of the heart and blood vessels, which deliver oxygen and nutrients to the body, continue to kill. Today, one of every 2.6 Americans will die of some cause related to their heart, writes Columbia University Medical Center.

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Stem cell therapy: When will it help the heart? | The Why Files

1. Introduction

Neuromuscular disease is a very broad term that encompasses many diseases and aliments that either directly, via intrinsic muscle pathology, or indirectly, via nerve pathology, impair the functioning of the muscles. Neuromuscular diseases affect the muscles and/or their nervous control and lead to problems with movement. Many are genetic; sometimes, an immune system disorder can cause them. As they have no cure, the aim of clinical treatment is to improve symptoms, increase mobility and lengthen life. Some of them affect the anterior horn cell, and are classified as acquired (e.g. poliomyelitis) and hereditary (e.g. spinal muscular atrophy) diseases. SMA is a genetic disease that attacks nerve cells, called motor neurons, in the spinal cord. As a consequence of the lost of the neurons, muscles weakness becomes to be evident, affecting walking, crawling, breathing, swallowing and head and neck control. Neuropathies affect the peripheral nerve and are divided into demyelinating (e.g. leucodystrophies) and axonal (e.g. porphyria) diseases. Charcot-Marie-Tooth (CMT) is the most frequent hereditary form among the neuropathies and its characterized by a wide range of symptoms so that CMT-1a is classified as demyelinating and CMT-2 as axonal (Marchesi & Pareyson, 2010). Defects in neuromuscular junctions cause infantile and non-infantile Botulism and Myasthenia Gravis (MG). MG is a antibody-mediated autoimmune disorder of the neuromuscular junction (NMJ) (Drachman, 1994; Meriggioli & Sanders, 2009). In most cases, it is caused by pathogenic autoantibodies directed towards the skeletal muscle acetylcholine receptor (AChR) (Patrick & Lindstrom, 1973) while in others, non-AChR components of the postsynaptic muscle endplate, such as the muscle-specific receptor tyrosine kinase (MUSK), might serve as targets for the autoimmune attack (Hoch et al., 2001). Although the precise origin of the autoimmune response in MG is not known, genetic predisposition and abnormalities of the thymus gland such as hyperplasia and neoplasia could have an important role in the onset of the disease (Berrih et al., 1984; Roxanis et al., 2001).

Several diseases affect muscles: they are classified as acquired (e.g. dermatomyositis and polymyositis) and hereditary (e.g. myotonic disorders and myopaties) forms. Among the myopaties, muscular dystrophies are characterized by the primary wasting of skeletal muscle, caused by mutations in the proteins that form the link between the cytoskeleton and the basal lamina (Cossu & Sampaolesi, 2007). Mutations in the dystrophin gene cause severe form of hereditary muscular diseases; the most common are Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD). DMD patients suffer for complete lack of dystrophin that causes progressive degeneration, muscle wasting and death into the second/third decade of life. Beside, BMD patients show a very mild phenotype, often asymptomatic primarily due to the expression of shorter dystrophin mRNA transcripts that maintain the coding reading frame. DMD patients muscles show absence of dystrophin and presence of endomysial fibrosis, small fibers rounded and muscle fiber degeneration/regeneration. Untreated, boys with DMD become progressively weak during their childhood and stop ambulation at a mean age of 9 years, later with corticosteroid treatment (12/13 yrs). Proximal weakness affects symmetrically the lower (such as quadriceps and gluteus) before the upper extremities, with progression to the point of wheelchair dependence. Eventually distal lower and then upper limb weakness occurs. Weakness of neck flexors is often present at the beginning, and most patients with DMD have never been able to jump. Wrist and hand muscles are involved later, allowing the patients to keep their autonomy in transfers using a joystick to guide their wheelchair. Musculoskeletal contractures (ankle, knees and hips) and learning difficulties can complicate the clinical expression of the disease. Besides this weakness distribution in the same patient, a deep variability among patients does exist. They could express a mild phenotype, between Becker and Duchenne dystrophy, or a really severe form, with the loss of deambulation at 7-8 years. Confinement to a wheelchair is followed by the development of scoliosis, respiratory failure and cardiomyopathy. In 90% of people death is directly related to chronic respiratory insufficiency (Rideau et al., 1983). The identification and characterization of dystrophin gene led to the development of potential treatments for this disorder (Bertoni, 2008). Even if only corticosteroids were proven to be effective on DMD patient (Hyser and Mendell, 1988), different therapeutic approaches were attempted, as described in detail below (see section 7).

The identification and characterization of the genes whose mutations caused the most common neuromuscular diseases led to the development of potential treatments for those disorders. Gene therapy for neuromuscular disorders embraced several concepts, including replacing and repairing a defective gene or modifying or enhancing cellular performance, using gene that is not directly related to the underlying defect (Shavlakadze et al., 2004). As an example, the finding that DMD pathology was caused by mutations in the dystrophin gene allowed the rising of different therapeutic approaches including growth-modulating agents that increase muscle regeneration and delay muscle fibrosis (Tinsley et al., 1998), powerful antisense oligonucleotides with exon-skipping capacity (Mc Clorey et al., 2006), anti-inflammatory or second-messenger signal-modulating agents that affect immune responses (Biggar et al., 2006), agents designed to suppress stop codon mutations (Hamed, 2006). Viral and non-viral vectors were used to deliver the full-length - or restricted versions - of the dystrophin gene into stem cells; alternatively, specific antisense oligonucleotides were designed to mask the putative splicing sites of exons in the mutated region of the primary RNA transcript whose removal would re-establish a correct reading frame. In parallel, the biology of stem cells and their role in regeneration were the subject of intensive and extensive research in many laboratories around the world because of the promise of stem cells as therapeutic agents to regenerate tissues damaged by disease or injury (Fuchs and Segre, 2000; Weissman, 2000). This research constituted a significant part of the rapidly developing field of regenerative biology and medicine, and the combination of gene and cell therapy arose as one of the most suitable possibility to treat degenerative disorders. Several works were published in which stem cell were genetically modified by ex vivo introduction of corrective genes and then transplanted in donor dystrophic animal models.

Stem cells received much attention because of their potential use in cell-based therapies for human disease such as leukaemia (Owonikoko et al., 2007), Parkinsons disease (Singh et al., 2007), and neuromuscular disorders (Endo, 2007; Nowak and Davies, 2004). The main advantage of stem cells rather than the other cells of the body is that they can replenish their numbers for long periods through cell division and, they can produce a progeny that can differentiate into multiple cell lineages with specific functions (Bertoni, 2008). The candidate stem cell had to be easy to extract, maintaining the capacity of myogenic conversion when transplanted into the host muscle and also the survival and the subsequent migration from the site of injection to the compromise muscles of the body (Price et al., 2007). With the advent of more sensitive markers, stem cell populations suitable for clinical experiments were found to derive from multiple region of the body at various stage of development. Numerous studies showed that the regenerative capacity of stem cells resided in the environmental microniche and its regulation. This way, it could be important to better elucidate the molecular composition cytokines, growth factors, cell adhesion molecules and extracellular matrix molecules - and interactions of the different microniches that regulate stem cell development (Stocum, 2001).

Several groups published different works concerning adult stem cells such as muscle-derived stem cells (Qu-Petersen et al., 2002), mesoangioblasts (Cossu and Bianco, 2003), blood- (Gavina et al., 2006) and muscle (Benchaouir et al., 2007)-derived CD133+ stem cells. Although some of them are able to migrate through the vasculature (Benchaouir et al., 2007; Galvez et al., 2006; Gavina et al., 2006) and efforts were done to increase their migratory ability (Lafreniere et al., 2006; Torrente et al., 2003a), poor results were obtained.

Embryonic and adult stem cells differ significantly in regard to their differentiation potential and in vitro expansion capability. While adult stem cells constitute a reservoir for tissue regeneration throughout the adult life, they are tissue-specific and possess limited capacity to be expanded ex vivo. Embryonic Stem (ES) cells are derived from the inner cell mass of blastocyst embryos and, by definition, are capable of unlimited in vitro self-renewal and have the ability to differentiate into any cell type of the body (Darabi et al., 2008b). ES cells, together with recently identified iPS cells, are now broadly and extensively studied for their applications in clinical studies.

Embryonic stem cells are pluripotent cells derived from the early embryo that are characterized by the ability to proliferate over prolonged periods of culture remaining undifferentiated and maintaining a stable karyotype (Amit and Itskovitz-Eldor, 2002; Carpenter et al., 2003; Hoffman and Carpenter, 2005). They are capable of differentiating into cells present in all 3 embryonic germ layers, namely ectoderm, mesoderm, and endoderm, and are characterized by self-renewal, immortality, and pluripotency (Strulovici et al., 2007).

hESCs are derived by microsurgical removal of cells from the inner cell mass of a blastocyst stage embryo (Fig. 1). The ES cells can be also obtained from single blastomeres. This technique creates ES cells from a single blastomere directly removed from the embryo bypassing the ethical issue of embryo destruction (Klimanskaya et al., 2006). Although maintaining the viability of the embryo, it has to be determined whether embryonic stem cell lines derived from a single blastomere that does not compromise the embryo can be considered for clinical studies. Cell Nuclear Transfer (SCNT): Nuclear transfer, also referred to as nuclear cloning, denotes the introduction of a nucleus from an adult donor cell into an enucleated oocyte to generate a cloned embryo (Wilmut et al., 2002).

ESCs differentiation. Differentiation potentiality of human embryonic stem cell lines. Human embryonic stem cell pluripotency is evaluated by the ability of the cells to differentiate into different cell types.

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