Cardiac stem cells derived from young hearts helped reverse the signs of aging when directly injected into the old hearts of elderly rats, astudypublished Monday in the European Heart Journal demonstrated.

The old rats appeared newly invigorated after receiving their injections. As hoped, the cardiac stem cells improved heart function yet also provided additional benefits. The rats fur, shaved for surgery, grew back more quickly than expected, and their chromosomal telomeres, which commonly shrink with age, lengthened.

Its extremely exciting, said Dr. Eduardo Marbn, primary investigator on the research and director of the Cedars-Sinai Heart Institute. Witnessing the systemic rejuvenating effects, he said, its kind of like an unexpected fountain of youth.

The working hypothesis is that the cells secrete exosomes, tiny vesicles that contain a lot of nucleic acids, things like RNA, that can change patterns of the way the tissue responds to injury and the way genes are expressed in the tissue, Marbn said.

It is the exosomes that act on the heart and make it better as well as mediating long-distance effects on exercise capacity and hair regrowth, he explained.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Unexpected fountain of youth found in cardiac stem cells, says researcher

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On Monday, a group of scientists at Cedars-Sinai Heart Institute in Los Angeles, CA, discovered througha world-first experimenta form to rejuvenate elder rats old hearts by injecting cardiac stem cells from much younger rats with healthier hearts. They hope this process might eventually become useful to humans.

The first time an experiment like this was carried out was in 2009 by the same Los Angeles-based team. Now, they also proved the possibility of reversing aging in old hearts.

Heart failure is a typical cause of death in humans. Around 48 percent of women and 46 percent of men die a year from heart attacks and other heart-related diseases. They are the first reason of death worldwide, and a leading cause of death in the United States, killing over 375,000 Americans a year. Nearly half of all African-American population suffers from heart diseases.

Researchers took stem cells from the hearts of 4-month-old rats, shaped them into cardiosphere-derived cells and injected them into the hearts of other rats of 22 monthsold, an age that makes them be considered as old. They carried out a similar process to another group of rats but injected saline instead. Scientists later compared both groups.

After receiving the stem cells injection, researchers noted a significant change in the way old rats continued to live. They turned much more active and improved their functionalities. Not just their heart rates got better and faster, but also the way they ran and breathed. Their hair started to grow faster, their chromosomal telomeres which commonly shrink with age lengthened, plus other benefits. The rodents began to progressively improve their capacity of exercise along with their stamina overall.

The animals could exercise further than they could before by about 20%, and one of the most striking things, especially for me (because Im kind of losing my hair) the animals regrew their fur a lot better after theyd gotten cells compared with the placebo rats, said Dr Eduardo Marbn, director of the Cedars-Sinai Heart Institute and lead author, who is also extremely excited for having witnessed the unexpected fountain of youth.

In 2009, his team successfully repaired the damaged heart of a man who had suffered a heart attack, using his own heart tissue.

Stem cells are a really basic type of cells that can be molded and converted into other much-specialized cells through a process called differentiation, which is basicallyshaping them into any kind of body cell.They form in embryos like embryonic stem cells -, which help in the growth process of babies, along with the millions of other different cell types they need before their birth.

One of many cells scientists generated from stem cells is called progenitor cell, which shares some of the same properties. But unlike the original cells, progenitor cells are not able to divide and reproduce indefinitely. Dr. Marbn also said they discovered cardiosphere-derived cells, which tend to promote the healing of a condition that affects more than 50 percent of patients suffering from heart failure.

Our previous lab studies and human clinical trials have shown promise in treating heart failure using cardiac stem cell infusions, said Dr Marbn. Now we find that these specialized stem cells could turn out to reverse problems associated with aging of the heart.

According to Dr. Marbn, stem cells secrete exosomes, tiny vesicles which contain a lot of nucleic acids, things like RNA, that can change patterns of the way the tissue responds to injuries, and the way genes are expressed in the tissue. They are placed into the heart, and act to transform it into a better organ, helping it at the same time to improve exercise capacity and hair regrowth, he explained.

Now, Dr. Marbn is exploring a much easier way to deliver the stem cells intravenously, instead of injecting them directly into the heart. Thus avoiding surgeries, which tend to be more complicated and expensive for the patient.

Striking benefits are demonstrated not only from a cardiac perspective but across multiple organ systems, said Dr. Gary Gerstenblith, a professor of medicine in the cardiology division of Johns Hopkins Medicine, who did not contribute to the new research. The results suggest that stem cell therapies should be studied as an additional therapeutic option in the treatment of cardiac and other diseases common in the elderly.

Now, scientistsneed to make more extensive studies before using the technique in humans.

Source: CNN

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Scientists discovered how to rejuvenate rats by injecting stem cells ... - Pulse Headlines

18 Aug 2017

Astrocytes are more than bystanders in neurotransmissionthey take an active role in synaptic activity. However, their functions are hard to study because the cells are difficult to grow in vitro and its hard to coax them to mature from progenitors. Now, researchers from the labs of Sergiu Paca and Ben Barres, both at Stanford University School of Medicine, California, report that astrocytes come of age in spherical balls of human brain cells cultured in a dish for almost two years. As reported in the August 16 Neuron, these astrocytes develop much like those from real brains, undergoing similar transcriptomic, morphologic, and functional changes. Studying the processes involved in this astrocyte maturation will help researchers understand neurodevelopmental disorders such as autism and schizophrenia, researchers say, and might even shed light on problems in adultbrains.

That these 3D cultures can be maintained for such a long time allows us to capture an interesting transition in astrocytes, said Paca. We are starting to appreciate aspects of human brain development to which we would not otherwise haveaccess.

The breakthrough is that they can develop human astrocytes very close to maturity in their 3D culture models, said Doo Yeon Kim, Massachusetts General Hospital, Charlestown, who uses 3D culture models to study pathological process that occur in Alzheimers disease. Some researchers are using 3D cultures to model other neurodegenerative disorders, such as ALS, and still others are planning to use cultured astrocytes for cell therapy. If astrocytes are not mature enough in culture, patterns [we see] may not be the same as in the diseased brain, saidKim.

This developing human astrocyte (red), which comes from a 350-day-old cortical spheroid, is taking shape as a mature cell. [Image courtesy of Sloan et al.Neuron]

A few years back, Pacas group developed a method for differentiating human induced pluripotent stem cells (hiPSCs) into a 3D culture of brain cells. They used special dishes that the cells could not easily attach to, coaxing them to stick to each other instead. Under these conditions the iPSCs balled up into neural spheroids that grew to about 4 mm in diameter. A cocktail of growth factors early on encouraged them to form excitatory pyramidal cells like those in the cortex, and the cells spontaneously organized into layers. These cortical spheroids survived a year or more and spontaneously grew astrocytes in addition to neurons (Paca et al., 2015). Not long after, the Barres lab reported that astrocytes in the adult human brain look different from those isolated from fetuses. They called the latter astrocyte progenitor cells (APCs). Each had their own transcriptional patterns and functions (Jan 2016 news). Together, Barres and Paca wondered if it was possible to see the APCs morph into mature astrocytes in these long-lived corticalspheroids.

To find out, first author Steven Sloan and colleagues examined spheroids generated from iPSCs derived from healthy human fibroblasts. Sloan grew the spheroids for about 20 months. Along the way, he took samples, isolated the astrocytes, and compared them to those isolated from fetal and postnatal humanbrain.

At about 100 days in culture, astrocytes began to sprout spontaneously from within the mostly neuronal milieu of the cortical spheroids. At first, these cells were simple, adorned by few branches and expressing genes akin to those active in APCs. But as the spheroids reached about 250 days, the astrocytes therein looked more mature, having numerous processes. After this point, APC gene expression tapered off and the astrocytes started producing proteins typical of matureastrocytes.

Astrocytes also underwent functional changes as they matured. Early versions divided in fast and furious fashion, much like their counterparts from the fetal tissue. That division slowed as the spheroids aged. Dividing APCs dropped from 35 percent of all astrocytes at day 167 to 3 percent at day 590. Taken from the spheroids at day 150 and cultured in a 2D layer, immature astrocytes also harbored a voracious appetite for added synaptosomes, much like immature astrocytes recently characterized in mice (see image below; Dec 2013 conference news on Chung et al., 2013). However, that hunger waned as astrocytes approached the 590-daymark.

At the older end of the spectrum, mature astrocytes seemed to take on a supportive role, strengthening calcium signaling in nearbyneurons.

Studying the neurons and astrocytes in these cortical spheroids could be useful for addressing certain unanswered questions about human biology, said other researchers. This could be a very strong opportunity to understand what goes wrong in human genetic disorders that affect astrocyte function, said M. Kerry OBanion, University of Rochester Medical Center, New York. Its also possible that such cultures could reveal as yet unknown facets of familial mutations that cause Alzheimers disease, he suggested. However, given that these cultures take a long time to grow and develop, they are unlikely to completely supplant other types of cultures or faster-maturing animal models, hesaid.

Kim agreed, saying, The results are very exciting, but not practical yet for disease modeling." However, Kim hopes that researchers will make progress on accelerating the maturationprocess.

The Barres and Paca labs are trying just that with the spheroid. They will also analyze what they secrete to support neuronal signaling. In addition, they are exploring how to make the astrocytes reactive, as they often are in neurodegenerative diseases, such as Alzheimers. Doing so might reveal how such astrocytes interact withneurons.

An immature astrocyte taken from a 150-day-old spheroid gobbles up added synaptosomes (red). [Neuron, Sloan et al.2017]

To Pacas knowledge, these cortical spheroids are some of the longest human cell cultures ever reported. His group has continued to cultivate these clumps, with the oldest still going strong at day 850. Granted, these systems are missing many cell types: endothelial cells, oligodendrocytes, and microglia to name a few, he said. However, his lab has introduced new ways to add in other cells. Earlier this year, he reported 3D cultures of cortical glutamatergic neurons and GABAergic interneurons that fused together when they were placed side-by-side (Birey et al., 2017).

Clive Svendsen, Cedars-Sinai Medical Center in Los Angeles, California, saw clinical implications for this paper. It shows iPSC derived astrocytes can mature to an adult phenotype, he said. This further supports their use in clinical transplantation, as we are planning to do. His group has begun a Phase 1 clinical trial that implants human fetal astrocytes into the spinal cords of ALS patients.Gwyneth DickeyZakaib

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Brain Spheroids Hatch Mature Astrocytes - Alzforum

A pregnant British mom hopes she and her unborn baby will be the answer to help prolong her ailing brothers life.

Georgina Russell, of Preston, England, said she was desperate to help her brother, Ashley, when doctors diagnosed him with a slow-growing but deadly brain tumor earlier this year, according to the Daily Mail.

Georgina said she began researching his condition, glioblastoma, online and looking for answers that could save his life. She found one: her pregnancy.

Stem cells produced in the umbilical cord between her and her unborn baby potentially could be used in a treatment to shrink Ashleys tumor, according to the report. Once Georgina gives birth, she said doctors will be able to harvest and store the stem cells until Ashley needs them.

There is no harm to the baby or the mother when doctors harvest stem cells from the umbilical cord unlike embryonic stem cells, which only can be taken by killing a human life in the embryonic stage.

Georgina told the Mail: The blood from the cord is being used in trials across the world. It can do amazing things to help the body repair itself. If we store the stem cells, they can be kept to be used throughout Ashleys treatment when he needs them.

They might be able to inject them into the spinal fluid, to shrink the tumour on the brain, or they may be able to use the tissue grown from them to repair any damage to other parts of his body, if he has to have chemotherapy or radiotherapy.

Ashley Russell, a British military veteran, husband and father, said doctors found the tumor after he began suffering from headaches, dizzy spells and mini-seizures about six months ago. Later, he said he also began having blurred vision. Doctors ran a series of tests before discovering the tumor on his brain.

He said doctors suggested surgery, but the procedure has high risks. They gave him about five years to live, according to the report.

Georgina said she was devastated for her brother and his family, and she began researching ways to help him. In her research online, she said she discovered how stem cells collected from the umbilical cord are helping to treat people with tumors and other diseases.

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Her brother said the idea seemed odd at first, but he is willing to try anything.

I am quite a positive person so although the diagnosis was difficult, I am determined to do whatever I can to keep going, Ashley said. I did think about not being around to see my little girl get married and knew that if there was anything that might help, I would give it a go.

Georgina currently is 33 weeks pregnant with her unborn child, the report states.

Stem cells are so powerful and his new niece or nephew could save his life, she said.

The family set up a JustGiving page to help pay for the storage of the stem cells and Ashleys treatment.

Adult stem cells and those from umbilical cords are proving to be live-saving, while life-destroying embryonic stem cells have not been effective.

David Prentice, vice president and research director for the Charlotte Lozier Institute, explained more about the effectiveness of these life-saving stem cells in 2014:

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Umbilical cord blood stem cells have become an extremely valuable alternative to bone marrow adult stem cell transplants, ever since cord blood stem cells were first used for patients over 25 years ago. The first umbilical cord blood stem cell transplant was performed in October 1988, for a 5-year-old child with Fanconi anemia, a serious condition where the bone marrow fails to make blood cells. That patient is currently alive and healthy, 25 years after the cord blood stem cell transplant.

Prentice said more than 30,000 cord blood stem cell transplants have been done across the world. These stem cells have helped treat people with blood and bone marrow diseases, leukemia and genetic enzyme diseases, he said.

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Woman Will Use Stem Cells From Her Baby's Umbilical Cord To Save Her Brother, Who Has a Brain Tumor - LifeNews.com

Twelve years ago, Robb Martin was an active police officer with Prescott Police Department when a recreational accident left him paralyzed from the chest down.

I was on a four-wheeler in the sand dunes, Martin, 42, said. I was on my way back to camp just putting along when I hit a bump. It threw me off the front, my helmet got stuck in the sand, my legs just kept going and I broke my back right at the chest level.

After getting out of the hospital and going through some rehabilitation to get his arms, shoulders and neck moving normally, he continued to work for the police department in the dispatch center and has been there ever since.

Despite his condition, Martin has remained incredibly active.

The guy is always busy, said Tom Newell, a longtime friend of Martins.

With some help from his friends, he managed to build a workshop on his property and is consistently in there modifying objects to fit his needs or assisting friends and family with various projects.

If hes not helping his wife with her business, hes in his shop welding something, making something or building something to help somebody else out, Newell said.

Since the accident, Martin has looked for ways to improve his mobility. Physical therapy has been helping, allowing him to regain back and stomach muscles in recent years.

I can do pushups and actually support my waist, which is amazing, he said.

His goal, however, is to once again be on his feet.

Just to even stand up and grab something out of a cabinet would be phenomenal, Martin said.

That dream might come true if he can raise the funds to have a recently developed procedure done in Thailand by a company called Unique Access.

The procedure, referred to as epidural stimulation, involves surgically implanting a device along a damaged portion of the nervous system, according to the companys website. The device then applies a continuous electrical current.

It acts kind of like a jumper cable, for lack of a better term, Martin said. It just connects above the affected area and allows the brain to reconnect with the spinal cord under the affected area.

In combination with the implant, several million stem cells are injected into the area to help the regenerative process. These, as well as

an assisted rehabilitation process, take about 40 days to complete.

The procedure has yet to be seriously implemented in the U.S., Martin said, because of how new it is to the medical industry. So far, however, he hasnt heard of any unusual risks associated with the procedure and has spoken with two individuals who successfully went through it.

One guy is walking up to 30 meters unassisted, Martin said. Another guy, the day after surgery, he was standing up by himself in a pool.

Altogether, Martin said its going to cost him $100,000 out of pocket.

Not able to afford that between him and his wife, hes turned to the community for help. Friends and family have already been busy contributing and organizing events.

Just last Saturday, Aug. 12, about $5,000 was raised on his behalf from two fundraising events hosted by his friends Tony and Liko Harwood.

Tony wanted to be involved and couldnt just sit still and not make any money for Rob so here we are, Liko said Saturday during one of the events.

Another $2,000 was raised from a donation bucket placed inside Scouts Gourmet Grub in Prescott.

Quite a bit more was also raised by fundraisers hosted by the Northern Arizona Regional Training Academy (NARTA), the local police academy.

Sitting at about $15,000, Martin is hoping to continue raising money in whatever way he can to reach the full $100,000.

My surgery is approved, theyre just waiting for me to set up a date, Martin said. The funding is really all Im waiting on.

For more information about Martins story and to donate, go to RobbMartin.com.

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Disabled former police officer raising money for operation in Thailand - The Daily Courier

In a new studyin Nature Communications, Stephanopoulos and his colleague Ronit Freeman successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways. Credit: Northwestern University

What if one day, we could teach our bodies to self-heal like a lizard's tail, and make severe injury or disease no more threatening than a paper cut?

Or heal tissues by coaxing cells to multiply, repair or replace damaged regions in loved ones whose lives have been ravaged by stroke, Alzheimer's or Parkinson's disease?

Such is the vision, promise and excitement in the burgeoning field of regenerative medicine, now a major ASU initiative to boost 21st-century medical research discoveries.

ASU Biodesign Institute researcher Nick Stephanopoulos is one of several rising stars in regenerative medicine. In 2015, Stephanopoulos, along with Alex Green and Jeremy Mills, were recruited to the Biodesign Institute's Center for Molecular Design and Biomimetics (CMDB), directed by Hao Yan, a world-recognized leader in nanotechnology.

"One of the things that that attracted me most to the ASU and the Biodesign CMDB was Hao's vision to build a group of researchers that use biological molecules and design principles to make new materials that can mimic, and one day surpass, the most complex functions of biology," Stephanopoulos said.

"I have always been fascinated by using biological building blocks like proteins, peptides and DNA to construct self-assembled structures, devices and materials, and the interdisciplinary and highly collaborative team in the CMDB is the ideal place to put this vision into practice."

Yan's research center uses DNA and other basic building blocks to build their nanotechnology structuresonly at a scale 1,000 times smaller than the width of a human hair.

They've already used nanotechnology to build containers to specially deliver drugs to tissues, build robots to navigate a maze or nanowires for electronics.

To build a manufacturing industry at that tiny scale, their bricks and mortar use a colorful assortment of molecular Legos. Just combine the ingredients, and these building blocks can self-assemble in a seemingly infinite number of ways only limited by the laws of chemistry and physicsand the creative imaginations of these budding nano-architects.

Learning from nature

"The goal of the Center for Molecular Design and Biomimetics is to use nature's design rules as an inspiration in advancing biomedical, energy and electronics innovation through self-assembling molecules to create intelligent materials for better component control and for synthesis into higher-order systems," said Yan, who also holds the Milton Glick Chair in Chemistry and Biochemistry.

Prior to joining ASU, Stephanopoulos trained with experts in biological nanomaterials, obtaining his doctorate with the University of California Berkeley's Matthew Francis, and completed postdoctoral studies with Samuel Stupp at Northwestern University. At Northwestern, he was part of a team that developed a new category of quilt-like, self-assembling peptide and peptide-DNA biomaterials for regenerative medicine, with an emphasis in neural tissue engineering.

"We've learned from nature many of the rules behind materials that can self-assemble. Some of the most elegant complex and adaptable examples of self-assembly are found in biological systems," Stephanopoulos said.

Because they are built from the ground-up using molecules found in nature, these materials are also biocompatible and biodegradable, opening up brand-new vistas for regenerative medicine.

Stephanopoulos' tool kit includes using proteins, peptides, lipids and nucleic acids like DNA that have a rich biological lexicon of self-assembly.

"DNA possesses great potential for the construction of self-assembled biomaterials due to its highly programmable nature; any two strands of DNA can be coaxed to assemble to make nanoscale constructs and devices with exquisite precision and complexity," Stephanopoulos said.

Proof all in the design

During his time at Northwestern, Stephanopoulos worked on a number of projects and developed proof-of-concept technologies for spinal cord injury, bone regeneration and nanomaterials to guide stem cell differentiation.

Now, more recently, in a new study in Nature Communications, Stephanopoulos and his colleague Ronit Freeman in the Stupp laboratory successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways.

In the new technology, materials are first chemically decorated with different strands of DNA, each with a unique code for a different signal to cells.

To activate signals within the cells, soluble molecules containing complementary DNA strands are coupled to short protein fragments, called peptides, and added to the material to create DNA double helices displaying the signal.

By adding a few drops of the DNA-peptide mixture, the material effectively gives a green light to stem cells to reproduce and generate more cells. In order to dynamically tune the signal presentation, the surface is exposed to a soluble single-stranded DNA molecule designed to "grab" the signal-containing strand of the duplex and form a new DNA double helix, displacing the old signal from the surface.

This new duplex can then be washed away, turning the signal "off." To turn the signal back on, all that is needed is to now introduce a new copy of single-stranded DNA bearing a signal that will reattach to the material's surface.

One of the findings of this work is the possibility of using the synthetic material to signal neural stem cells to proliferate, then at a specific time selected by the scientist, trigger their differentiation into neurons for a while, before returning the stem cells to a proliferative state on demand.

One potential use of the new technology to manipulate cells could help cure a patient with neurodegenerative conditions like Parkinson's disease.

The patient's own skin cells could be converted to stem cells using existing techniques. The new technology could help expand the newly converted stem cells back in the laband then direct their growth into specific dopamine-producing neurons before transplantation back to the patient.

"People would love to have cell therapies that utilize stem cells derived from their own bodies to regenerate tissue," Stupp said. "In principle, this will eventually be possible, but one needs procedures that are effective at expanding and differentiating cells in order to do so. Our technology does that."

In the future, it might be possible to perform this process entirely within the body. The stem cells would be implanted in the clinic, encapsulated in the type of material described in the new work, and injected into a particular spot. Then the soluble peptide-DNA molecules would be given to the patient to bind to the material and manipulate the proliferation and differentiation of transplanted cells.

Scaling the barriers

One of the future challenges in this area will be to develop materials that can respond better to external stimuli and reconfigure their physical or chemical properties accordingly.

"Biological systems are complex, and treating injury or disease will in many cases necessitate a material that can mimic the complex spatiotemporal dynamics of the tissues they are used to treat," Stephanopoulos said.

It is likely that hybrid systems that combine multiple chemical elements will be necessary; some components may provide structure, others biological signaling and yet others a switchable element to imbue dynamic ability to the material.

A second challenge, and opportunity, for regenerative medicine lies in creating nanostructures that can organize material across multiple length scales. Biological systems themselves are hierarchically organized: from molecules to cells to tissues, and up to entire organisms.

Consider that for all of us, life starts simple, with just a single cell. By the time we reach adulthood, every adult human body is its own universe of cells, with recent estimates of 37 trillion or so. The human brain alone has 100 billion cells or about the same number of cells as stars in the Milky Way galaxy.

But over the course of a life, or by disease, whole constellations of cells are lost due to the ravages of time or the genetic blueprints going awry.

Collaborative DNA

To overcome these obstacles, much more research funding and recruitment of additional talent to ASU will be needed to build the necessary regenerative medicine workforce.

Last year, Stephanopoulos' research received a boost with funding from the U.S. Air Force's Young Investigator Research Program (YIP).

"The Air Force Office of Scientific Research YIP award will facilitate Nick's research agenda in this direction, and is a significant recognition of his creativity and track record at the early stage of his careers," Yan said.

They'll need this and more to meet the ultimate challenge in the development of self-assembled biomaterials and translation to clinical applications.

Buoyed by the funding, during the next research steps, Stephanopoulos wants to further expand horizons with collaborations from other ASU colleagues to take his research team's efforts one step closer to the clinic.

"ASU and the Biodesign Institute also offer world-class researchers in engineering, physics and biology for collaborations, not to mention close ties with the Mayo Clinic or a number of Phoenix-area institutes so we can translate our materials to medically relevant applications," Stephanopoulos said.

There is growing recognition that regenerative medicine in the Valley could be a win-win for the area, in delivering new cures to patients and building, person by person, a brand-new medicinal manufacturing industry.

Explore further: New technology to manipulate cells could help treat Parkinson's, arthritis, other diseases

More information: Ronit Freeman et al. Instructing cells with programmable peptide DNA hybrids, Nature Communications (2017). DOI: 10.1038/ncomms15982

More:
Bio-inspired materials give boost to regenerative medicine - Medical Xpress

Stem cell science involves many technical terms. This glossary covers many of the common terms you will encounter in reading about stem cells.

Adult stem cellsA commonly used term for tissue-specific stem cells, cells that can give rise to the specialized cells in specific tissues. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent stem cells.

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AutologousCells or tissues from the same individual; an autologous bone marrow transplant involves one individual as both donor and recipient.

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Basic researchResearch designed to increase knowledge and understanding (as opposed to research designed with the primary goal to solve a problem).

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BlastocystA transient, hollow ball of 150 to 200 cells formed in early embryonic development that contains the inner cell mass, from which the embryo develops, and an outer layer of cell called the trophoblast, which forms the placenta.

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Bone marrow stromal cellsA general term for non-blood cells in the bone marrow, such as fibroblasts, adipocytes (fat cells) and bone- and cartilage-forming cells that provide support for blood cells. Contained within this population of cells are multipotent bone marrow stromal stem cells that can self-renew and give rise to bone, cartilage, adipocytes and fibroblasts.

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CardiomyocytesThe functional muscle cells of the heart that allow it to beat continuously and rhythmically.

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Clinical translationThe process of using scientific knowledge to design, develop and apply new ways to diagnose, stop or fix what goes wrong in a particular disease or injury; the process by which basic scientific research becomes medicine.

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Clinical trialTests on human subjects designed to evaluate the safety and/or effectiveness of new medical treatments.

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Cord bloodThe blood in the umbilical cord and placenta after child birth. Cord blood contains hematopoietic stem cells, also known as cord blood stem cells, which can regenerate the blood and immune system and can be used to treat some blood disorders such as leukemia or anemia. Cord blood can be stored long-term in blood banks for either public or private use. Also called umbilical cord blood.

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CytoplasmFluid inside a cell, but outside the nucleus.

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DifferentiationThe process by which cells become increasingly specialized to carry out specific functions in tissues and organs.

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Drug discoveryThe systematic process of discovering new drugs.

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Drug screeningThe process of testing large numbers of potential drug candidates for activity, function and/or toxicity in defined assays.

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EmbryoGenerally used to describe the stage of development between fertilization and the fetal stage; the embryonic stage ends 7-8 weeks after fertilization in humans.

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Embryonic stem cells (ESCs)Undifferentiated cells derived from the inner cell mass of the blastocyst; these cells have the potential to give rise to all cell types in the fully formed organism and undergo self-renewal.

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FibroblastA common connective or support cell found within most tissues of the body.

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GlucoseA simple sugar that cells use for energy.

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HematopoieticBlood-forming; hematopoietic stem cells give rise to all the cell types in the blood.

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ImmunomodulatoryThe ability to modify the immune system or an immune response.

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Induced pluripotent stem cells (iPSCs)Embryonic-like stem cells that are derived from reprogrammed, adult cells, such as skin cells. Like ESCs, iPS cells are pluripotent and can self-renew.

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In vitroLatin for in glass. In biomedical research this refers to experiments that are done outside the body in an artificial environment, such as the study of isolated cells in controlled laboratory conditions (also known as cell culture).

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In vivoLatin for within the living. In biomedical research this refers to experiments that are done in a living organism. Experiments in model systems such as mice or fruit flies are an example of in vivo research.

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Islets of LangerhansClusters in the pancreas where insulin-producing beta cells live.

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MaculaA small spot at the back of the retina, densely packed with the rods and cones that receive light, which is responsible for high-resolution central vision.

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Mesenchymal stem cells (MSCs)A term used to describe cells isolated from the connective tissue that surrounds other tissues and organs. MSCs were first isolated from the bone marrow and shown to be capable of making bone, cartilage and fat cells. MSCs are now grown from other tissues, such as fat and cord blood. Not all MSCs are the same and their characteristics depend on where in the body they come from and how they are isolated and grown. May also be called mesenchymal stromal cells.

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Multipotent stem cellsStem cells that can give rise to several different types of specialized cells in specific tissues; for example, blood stem cells can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain.

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NeuronAn electrically excitable cell that processes and transmits information through electrical and chemical signals in the central and peripheral nervous systems.

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Pancreatic beta cellsCells responsible for making and releasing insulin, the hormone responsible for regulating blood sugar levels. Type I diabetes occurs when these cells are attacked and destroyed by the body's immune system.

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PhotoreceptorsRod or cone cells in the retina that receive light and send signals to the optic nerve, which passes along these signals to the brain.

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PlaceboA pill, injection or other treatment that has no therapeutic benefit; often used as a control in clinical trials to see whether new treatments work better than no treatment.

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Placebo effectPerceived or actual improvement in symptoms that cannot be attributed to the placebo itself and therefore must be the result of the patient's (or other interested person's) belief in the treatment's effectiveness.

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Pluripotent stem cellsStem cells that can become all the cell types that are found in an embryo, fetus or adult, such as embryonic stem cells or induced pluripotent (iPS) cells.

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Preclinical researchLaboratory research on cells, tissues and/or animals for the purpose of discovering new drugs or therapies.

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Precursor cellsAn intermediate cell type between stem cells and differentiated cells. Precursor cells have the potential to give rise to a limited number or type of specialized cells. Also called progenitor cells.

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Progenitor cellsAn intermediate cell type between stem cells and differentiated cells. Progenitor cells have the potential to give rise to a limited number or type of specialized cells and have a reduced capacity for self-renewal. Also called precursor cells.

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Regenerative MedicineAn interdisciplinary branch of medicine with the goal of replacing, regenerating or repairing damaged tissue to restore normal function. Regenerative treatments can include cellular therapy, gene therapy and tissue engineering approaches.

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ReprogrammingIn the context of stem cell biology, this refers to the conversion of differentiated cells, such as fibroblasts, into embryonic-like iPS cells by artificially altering the expression of key genes.

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Retinal pigment epitheliumA single-cell layer behind the rods and cones in the retina that provide support functions for the rods and cones.

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RNARibonucleic acid; it "reads" DNA and acts as a messenger for carrying out genetic instructions.

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Scientific methodA systematic process designed to understand a specific observation through the collection of measurable, empirical evidence; emphasis on measurable and repeatable experiments and results that test a specific hypothesis.

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Self-renewalA special type of cell division in stem cells by which they make copies of themselves.

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Somatic stem cellsScientific term for tissue-specific or adult stem cells.

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Stem cellsCells that have both the capacity to self-renew (make more stem cells by cell division) and to differentiate into mature, specialized cells.

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Stem cell tourismThe travel to another state, region or country specifically for the purpose of undergoing a stem cell treatment available at that location. This phrase is also used to refer to the pursuit of untested and unregulated stem cell treatments.

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TeratomaA benign tumor that usually consists of several types of tissue cells that are foreign to the tissue in which the tumor is located.

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TissueA group of cells with a similar function or embryological origin. Tissues organize further to become organs.

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Tissue-specific stem cellsStem cells that can give rise to the specialized cells in specific tissues; blood stem cells, for example, can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent cells. Also called adult or somatic stem cells.

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TotipotentThe ability to give rise to all the cells of the body and cells that arent part of the body but support embryonic development, such as the placenta and umbilical cord.

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Translational researchResearch that focuses on how to use knowledge gleaned from basic research to develop new drugs, treatments or therapies.

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ZygoteThe single cell formed when a sperm cell fuses with an egg cell.

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Stem Cell Glossary

Digital Trends

Want to live longer? Forever Labs wants to help, using your stem cells Digital Trends Using a patented device, Forever Labs collects stem cells from your blood marrow, which the team calls a wellspring for stem cells that replenish your blood, bone, immune system, and other vital tissues. The whole process is said to take around 15 ...

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Want to live longer? Forever Labs wants to help, using your stem cells - Digital Trends

The study suggests it may encourage blood cancer stem cells to die.

Researchers say Vitamin C may "tell" faulty stem cells in the bone marrow to mature and die normally, instead of multiplying to cause blood cancers.

They explained that certain genetic changes are known to reduce the ability of an enzyme called TET2 to encourage stem cells to become mature blood cells, which eventually die, in many patients with certain kinds of leukaemia.

The new study, published online by the journal Cell. found that vitamin C activated TET2 function in mice engineered to be deficient in the enzyme.

Study corresponding author Professor Benjamin Neel, of the Perlmutter Cancer Centre in the United States, said: "We're excited by the prospect that high-dose vitamin C might become a safe treatment for blood diseases caused by TET2-deficient leukemia stem cells, most likely in combination with other targeted therapies."

He said changes in the genetic code that reduce TET2 function are found in 10 per cent of patients with acute myeloid leukaemia (AML), 30 per cent of those with a form of pre-leukaemia called myelodysplastic syndrome, and in nearly 50 per cent of patients with chronic myelomonocytic leukaemia.

Such cancers cause anaemia, infection risk, and bleeding as abnormal stem cells multiply in the bone marrow until they interfere with blood cell production, with the number of cases increasing as the population ages.

Prof Neel said the study results revolve around the relationship between TET2 and cytosine, one of the four nucleic acid "letters" that comprise the DNA code in genes.

To determine the effect of mutations that reduce TET2 function in abnormal stem cells, the researchers genetically engineered mice such that the scientists could switch the TET2 gene on or off.

Similar to the naturally occurring effects of TET2 mutations in mice or humans, using molecular biology techniques to turn off TET2 in mice caused abnormal stem cell behaviour.

Prof Neel said, remarkably, the changes were reversed when TET2 expression was restored by a genetic trick.

Previous work had shown that vitamin C could stimulate the activity of TET2 and its relatives TET1 and TET3.

Because only one of the two copies of the TET2 gene in each stem cell is usually affected in TET2-mutant blood diseases, the researchers hypothesised that high doses of vitamin C, which can only be given intravenously, might reverse the effects of TET2 deficiency by turning up the action of the remaining functional gene.

They found that vitamin C did the same thing as restoring TET2 function genetically.

By promoting DNA demethylation, high-dose vitamin C treatment induced stem cells to mature, and also suppressed the growth of leukaemia cancer stem cells from human patients implanted in mice.

Study first author Doctor Luisa Cimmino, of New York University Langone Health, said: "Interestingly, we also found that vitamin C treatment had an effect on leukaemic stem cells that resembled damage to their DNA.

"For this reason, we decided to combine vitamin C with a PARP inhibitor, a drug type known to cause cancer cell death by blocking the repair of DNA damage, and already approved for treating certain patients with ovarian cancer."

The researchers found that the combination had an enhanced effect on leukaemia stem cells, further shifting them from self-renewal back toward maturity and cell death.

Dr Cimmino said the results also suggest that vitamin C might drive leukaemic stem cells without TET2 mutations toward death, given that it turns up any TET2 activity normally in place.

Corresponding author Professor Iannis Aifantis, also of NYU Langone Health, added: "Our team is working to systematically identify genetic changes that contribute to risk for leukaemia in significant groups of patients.

"This study adds the targeting of abnormal TET2-driven DNA demethylation to our list of potential new treatment approaches."

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Blood cancer: High doses of vitamin C could encourage stem cells ... - Express.co.uk

WASHINGTON D.C: Good news! A study has recently revealed that vitamin C may tell faulty stem cells in the bone marrow to mature and die normally, instead of multiplying to cause blood cancers.

According to researchers, certain genetic changes are known to reduce the ability of an enzyme called TET2 to encourage stem cells to become mature blood cells, which eventually die, in many patients with certain kinds of leukemia.

The new study found that vitamin C activated TET2 function in mice engineered to be deficient in the enzyme.

Corresponding study author Benjamin G. Neel said, "We're excited by the prospect that high-dose vitamin C might become a safe treatment for blood diseases caused by TET2-deficient leukemia stem cells, most likely in combination with other targeted therapies."

The results suggested that changes in the genetic code (mutations) that reduce TET2 function are found in 10 percent of patients with acute myeloid leukemia (AML), 30 percent of those with a form of pre-leukemia called myelodysplastic syndrome, and in nearly 50 percent of patients with chronic myelomonocytic leukemia.

The study results revolve around the relationship between TET2 and cytosine, one of the four nucleic acid "letters" that comprise the DNA code in genes.

To determine the effect of mutations that reduce TET2 function in abnormal stem cells, the team genetically engineered mice such that the scientists could switch the TET2 gene on or off.

The findings indicated that vitamin C did the same thing as restoring TET2 function genetically. By promoting DNA demethylation, high-dose vitamin C treatment induced stem cells to mature, and also suppressed the growth of leukemia cancer stem cells from human patients implanted in mice.

"Interestingly, we also found that vitamin C treatment had an effect on leukemic stem cells that resembled damage to their DNA," said first study author Luisa Cimmino.

"For this reason, we decided to combine vitamin C with a PARP inhibitor, a drug type known to cause cancer cell death by blocking the repair of DNA damage, and already approved for treating certain patients with ovarian cancer," Cimmino added.

The findings appear in journal Cell.

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Vitamin C could help genes kill blood cancer stem cells - Economic Times

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Bone Marrow Transplant gives local cancer patient more time with his family - KTBS

Scientists recently used a gene-editing tool to fix a mutation in a human embryo. Around the world, researchers are chasing cures for other genetic diseases.

Now that the gene-editing genie is out of the bottle, what would you wish for first?

Babies with perfect eyes, over-the-top intelligence, and a touch of movie star charisma?

Or a world free of disease not just for your family, but for every family in the world?

Based on recent events, many scientists are working toward the latter.

Earlier this month, scientists from the Oregon Health & Science University used a gene editing tool to correct a disease-causing mutation in an embryo.

The technique, known as CRISPR-Cas9, fixed the mutation in the embryos nuclear DNA that causes hypertrophic cardiomyopathy, a common heart condition that can lead to heart failure or cardiac death.

This is the first time that this gene-editing tool has been tested on clinical-quality human eggs.

Had one of these embryos been implanted into a womans uterus and allowed to fully develop, the baby would have been free of the disease-causing variation of the gene.

This type of beneficial change would also have been passed down to future generations.

None of the embryos in this study were implanted or allowed to develop. But the success of the experiment offers a glimpse at the potential of CRISPR-Cas9.

Still, will we ever be able to gene-edit our world free of disease?

According to the Genetic Disease Foundation, there are more than 6,000 human genetic disorders.

Scientists could theoretically use CRISPR-Cas9 to correct any of these diseases in an embryo.

To do this, they would need an appropriate piece of RNA to target corresponding stretches of genetic material.

The Cas9 enzyme cuts DNA at that spot, which allows scientists to delete, repair, or replace a specific gene.

Some genetic diseases, though, may be easier to treat with this method than others.

Most people are focusing, at least initially, on diseases where there really is only one gene involved or a limited number of genes and theyre really well understood, Megan Hochstrasser, PhD, science communications manager at the Innovative Genomics Institute in California, told Healthline.

Diseases caused by a mutation in a single gene include sickle cell disease, cystic fibrosis, and Tay-Sachs disease. These affect millions of people worldwide.

These types of diseases, though, are far outnumbered by diseases like cardiovascular disease, diabetes, and cancer, which kill millions of people across the globe each year.

Genetics along with environmental factors also contribute to obesity, mental illness, and Alzheimers disease, although scientists are still working on understanding exactly how.

Right now, most CRISPR-Cas9 research focuses on simpler diseases.

There are a lot of things that have to be worked out with the technology for it to get to the place where we could ever apply it to one of those polygenic diseases, where multiple genes contribute or one gene has multiple effects, said Hochstrasser.

Although designer babies gain a lot of media attention, much CRISPR-Cas9 research is focused elsewhere.

Most people who are working on this are not working in human embryos, said Hochstrasser. Theyre trying to figure out how we can develop treatments for people that already have diseases.

These types of treatments would benefit children and adults who are already living with a genetic disease, as well as people who develop cancer.

This approach may also help the 25 million to 30 million Americans who have one of the more than 6,800 rare diseases.

Gene editing is a really powerful option for people with rare disease, said Hochstrasser. You could theoretically do a phase I clinical trial with all the people in the world that have a certain [rare] condition and cure them all if it worked.

Rare diseases affect fewer than 200,000 people in the United States at any given time, which means there is less incentive for pharmaceutical companies to develop treatments.

These less-common diseases include cystic fibrosis, Huntingtons disease, muscular dystrophies, and certain types of cancer.

Last year researchers at the University of California Berkeley made progress in developing an ex vivo therapy where you take cells out of a person, modify them, and put them back into the body.

This treatment was for sickle cell disease. In this condition, a genetic mutation causes hemoglobin molecules to stick together, which deforms red blood cells. This can lead to blockages in the blood vessels, anemia, pain, and organ failure.

Researchers used CRISPR-Cas9 to genetically engineer stem cells to fix the sickle cell disease mutation. They then injected these cells into mice.

The stem cells migrated to the bone marrow and developed into healthy red blood cells. Four months later, these cells could still be found in the mices blood.

This is not a cure for the disease, because the body would continue to make red blood cells that have the sickle cell disease mutation.

But researchers think that if enough healthy stem cells take root in the bone marrow, it could reduce the severity of disease symptoms.

More work is needed before researchers can test this treatment in people.

A group of Chinese researchers used a similar technique last year to treat people with an aggressive form of lung cancer the first clinical trial of its kind.

In this trial, researchers modified patients immune cells to disable a gene that is involved in stopping the cells immune response.

Researchers hope that, once injected into the body, the genetically edited immune cells will mount a stronger attack against the cancer cells.

These types of therapies might also work for other blood diseases, cancers, or immune problems.

But certain diseases will be more challenging to treat this way.

If you have a disorder of the brain, for example, you cant remove someones brain, do gene editing and then put it back in, said Hochstrasser. So we have to figure out how to get these reagents to the places they need to be in the body.

Not every human disease is caused by mutations in our genome.

Vector-borne diseases like malaria, yellow fever, dengue fever, and sleeping sickness kill more than 1 million people worldwide each year.

Many of these diseases are transmitted by mosquitoes, but also by ticks, flies, fleas, and freshwater snails.

Scientists are working on ways to use gene editing to reduce the toll of these diseases on the health of people around the world.

We could potentially get rid of malaria by engineering mosquitoes that cant transmit the parasite that causes malaria, said Hochstrasser. We could do this using the CRISPR-Cas9 technique to push this trait through the entire mosquito population very quickly.

Researchers are also using CRISPR-Cas9 to create designer foods.

DuPont recently used gene editing to produce a new variety of waxy corn that contains higher amounts of starch, which has uses in food and industry.

Modified crops may also help reduce deaths due to malnutrition, which is responsible for nearly half of all deaths worldwide in children under 5.

Scientists could potentially use CRISPR-Cas9 to create new varieties of food that are pest-resistant, drought-resistant, or contain more micronutrients.

One benefit of CRISPR-Cas9, compared to traditional plant breeding methods, is that it allows scientists to insert a single gene from a related wild plant into a domesticated variety, without other unwanted traits.

Gene editing in agriculture may also move more quickly than research in people because there is no need for years of lab, animal, and human clinical trials.

Even though plants grow pretty slowly, said Hochstrasser, it really is quicker to get [genetically engineered plants] out into the world than doing a clinical trial in people.

Safety and ethical concerns

CRISPR-Cas9 is a powerful tool, but it also raises several concerns.

Theres a lot of discussion right now about how best to detect so-called off-target effects, said Hochstrasser. This is what happens when the [Cas9] protein cuts somewhere similar to where you want it to cut.

Off-target cuts could lead to unexpected genetic problems that cause an embryo to die. An edit in the wrong gene could also create an entirely new genetic disease that would be passed onto future generations.

Even using CRISPR-Cas9 to modify mosquitoes and other insects raises safety concerns like what happens when you make large-scale changes to an ecosystem or a trait in a population that gets out of control.

There are also many ethical issues that come with modifying human embryos.

So will CRISPR-Cas9 help rid the world of disease?

Theres no doubt that it will make a sizeable dent in many diseases, but its unlikely to cure all of them any time soon.

We already have tools for avoiding genetic diseases like early genetic screening of fetuses and embryos but these are not universally used.

We still dont avoid tons of genetic diseases, because a lot of people dont know that they harbor mutations that can be inherited, said Hochstrasser.

Some genetic mutations also happen spontaneously. This is the case with many cancers that result from environmental factors such as UV rays, tobacco smoke, and certain chemicals.

People also make choices that increase their risk of heart disease, stroke, obesity, and diabetes.

So unless scientists can use CRISPR-Cas9 to find treatments for these lifestyle diseases or genetically engineer people to stop smoking and start biking to work these diseases will linger in human society.

Things like that are always going to need to be treated, said Hochstrasser. I dont think its realistic to think we would ever prevent every disease from happening in a human.

Continue reading here:
Will Gene Editing Allow Us to Rid the World of Diseases? - Healthline

Forever Labs, a startup in Y Combinators latest batch, is preserving adult stem cells with the aim to help you live longer and healthier.

Stem cells have the potential to become any type of cell needed in the body. Its very helpful to have younger stem cells from your own body on hand should you ever need some type of medical intervention, like a bone marrow transplant as the risk of rejection is greatly reduced when the cells are yours.

Mark Katakowski spent the last 15 years studying stem cells. What he found is that not only do we have less of them the older we get, but they also lose their function as we age.So, he and his co-founders Edward Cibor and Steve Clausnitzer started looking at how to bank them while they were young.

Clausnitzer banked his cells two years ago at the age of 38. So, while he is biologically now age 40, his cells remain the age in which they were harvested or as he calls it, stem cell time travel.

Steven Clausnitzer with his 38-year-old banked stem cells.

There are places offering stem cell therapy and Botox, he said.

Forever Labs is backed by a team of Ivy League-trained scientists with decades of experience between them. Jason Camm, chief medical officer for Thiel Capital, is also one of the companys medical advisors however, the startup is quick to point out it is not associated with Thiel Capital.

The process involves using a patented device to collect the cells. Forever Labs can then grow and bank your cells for $2,500, plus another $250 for storage per year (or a flat fee of $7,000 for life).

The startup is FDA-approved to bank these cells and is offering the service in seven states. What it does not have FDA approval for is the modification of those cells for rejuvenation therapy.

Katakowski refers to what the company is doing as longevity as a service, with the goal being to eventually take your banked cells and modify them to reverse the biological clock.

But that may take a few years. There are hundreds of clinical trials looking at stem cell uses right now. Forever Labs has also proposed its own clinical trial to take your stem cells and give them to your older cells.

Youll essentially young-blood effect yourself, Katakowski joked of course, in this case, youd be using your own blood made from your own stem cells, not the blood of random teens.

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Forever Labs preserves young stem cells to prevent your older self from aging - TechCrunch

Ball-and-stick model of the L-ascorbic acid (vitamin C) molecule, C6H8O6, as found in the crystal structure. Credit: public domain

Vitamin C may "tell" faulty stem cells in the bone marrow to mature and die normally, instead of multiplying to cause blood cancers. This is the finding of a study led by researchers from Perlmutter Cancer Center at NYU Langone Health, and published online August 17 in the journal Cell.

Certain genetic changes are known to reduce the ability of an enzyme called TET2 to encourage stem cells to become mature blood cells, which eventually die, in many patients with certain kinds of leukemia, say the authors. The new study found that vitamin C activated TET2 function in mice engineered to be deficient in the enzyme.

"We're excited by the prospect that high-dose vitamin C might become a safe treatment for blood diseases caused by TET2-deficient leukemia stem cells, most likely in combination with other targeted therapies," says corresponding study author Benjamin G. Neel, MD, PhD, professor in the Department of Medicine and director of the Perlmutter Cancer Center.

Changes in the genetic code (mutations) that reduce TET2 function are found in 10 percent of patients with acute myeloid leukemia (AML), 30 percent of those with a form of pre-leukemia called myelodysplastic syndrome, and in nearly 50 percent of patients with chronic myelomonocytic leukemia. Such cancers cause anemia, infection risk, and bleeding as abnormal stem cells multiply in the bone marrow until they interfere with blood cell production, with the number of cases increasing as the population ages.

Along with these diseases, new tests suggest that about 2.5 percent of all U.S. cancer patients - or about 42,500 new patients each year - may develop TET2 mutations, including some with lymphomas and solid tumors, say the authors.

Cell Death Switch

The study results revolve around the relationship between TET2 and cytosine, one of the four nucleic acid "letters" that comprise the DNA code in genes. Every cell type has the same genes, but each gets different instructions to turn on only those needed in a given cellular context.

These "epigenetic" regulatory mechanisms include DNA methylation, the attachment of a small molecule termed a methyl group to cytosine bases that shuts down the action of a gene containing them.

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The back- and-forth attachment and removal of methyl groups also fine-tunes gene expression in stem cells, which can mature, specialize and multiply to become muscle, bone, nerve, or other cell types. This happens as the body first forms, but the bone marrow also keeps pools of stem cells on hand into adulthood, ready to become replacement cells as needed. In leukemia, signals that normally tell a blood stem cell to mature malfunction, leaving it to endlessly multiply and "self-renew" instead of producing normal white blood cells needed to fight infection.

The enzyme studied in this report, Tet methylcytosine dioxygenase 2 (TET2), enables a change in the molecular structure (oxidation) of methyl groups that is needed for them to be removed from cytosines. This "demethylation" turns on genes that direct stem cells to mature, and to start a count-down toward self-destruction as part of normal turnover. This serves as an anti-cancer safety mechanism, one that is disrupted in blood cancer patients with TET2 mutations, says Neel.

To determine the effect of mutations that reduce TET2 function in abnormal stem cells, the research team genetically engineered mice such that the scientists could switch the TET2 gene on or off.

Similar to the naturally occurring effects of TET2 mutations in mice or humans, using molecular biology techniques to turn off TET2 in mice caused abnormal stem cell behavior. Remarkably, these changes were reversed when TET2 expression was restored by a genetic trick. Previous work had shown that vitamin C could stimulate the activity of TET2 and its relatives TET1 and TET3. Because only one of the two copies of the TET2 gene in each stem cell is usually affected in TET2-mutant blood diseases, the authors hypothesized that high doses of vitamin C, which can only be given intravenously, might reverse the effects of TET2 deficiency by turning up the action of the remaining functional gene.

Indeed, they found that vitamin C did the same thing as restoring TET2 function genetically. By promoting DNA demethylation, high-dose vitamin C treatment induced stem cells to mature, and also suppressed the growth of leukemia cancer stem cells from human patients implanted in mice.

"Interestingly, we also found that vitamin C treatment had an effect on leukemic stem cells that resembled damage to their DNA," says first study author Luisa Cimmino, PhD, an assistant professor in the Department of Pathology at NYU Langone Health. "For this reason, we decided to combine vitamin C with a PARP inhibitor, a drug type known to cause cancer cell death by blocking the repair of DNA damage, and already approved for treating certain patients with ovarian cancer."

Researchers found that the combination had an enhanced effect on leukemia stem cells, further shifting them from self-renewal back toward maturity and cell death. The results also suggest that vitamin C might drive leukemic stem cells without TET2 mutations toward death, says Cimmino, given that it turns up any TET2 activity normally in place.

"Our team is working to systematically identify genetic changes that contribute to risk for leukemia in significant groups of patients," says corresponding author Iannis Aifantis, PhD, professor and chair of the Department of Pathology at NYU Langone Health. "This study adds the targeting of abnormal TET2-driven DNA demethylation to our list of potential new treatment approaches."

Explore further: A tumor-suppressing gene can be harmful in some cancers

Journal reference: Cell

Provided by: NYU Langone Health / NYU School of Medicine

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Vitamin C may encourage blood cancer stem cells to die - Medical Xpress

HELENA ExplorationWorks is hosting the Be The Match bone marrow donor drive this week at the Great Northern Town Center.

The drive is intended to support those in need of bone marrow or blood stem cell transplants around the world. Its being held in conjunction with ExplorationWorks Kids Kicking Cancer Camp.

The camp is open to children who are directly affected by cancer in their lives. Campers had the opportunity to make a card for Be the Match child who is currently undergoing or awaiting treatment.

Our hope is that the kids attending our camp will be able to connect with the Be The Match kids on a level most other children wouldnt understand. Knowing someone else is fighting the same fight will hopefully be a healing activity for all of the kids involved, said ExplorationWorks Education Director Lauren Rivers.

John Philpott of Be the Match said that sadly, some of the Be The Match kids children are still waiting to be matched with a donor.

There are still thousands of patients every year who have to hear their doctor say theres no match for you, said Phillpott, One Montanan [donation] can mean the difference for one patient.

According to Be the Match, someone is diagnosed with blood cancer every three minutes and every 10 minutes someone dies from not receiving a transplant.

The Marrow Donor Registry Drive will continue at ExplorationWorks from 10 a.m. to 5 p.m. Friday and from 12:30 to 3 p.m. on Saturday.

Registration takes around 10 minutes to complete and only involves some paper work and a few cheek swabs. You must be between the ages of 18 and 44 in order to register.

For more information about bone marrow donation and how to register click here.

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Bone marrow drive held at ExplorationWorks - KTVH

US researchers found high doses of vitamin C found in fruits such as oranges and green leafy vegetables such as kale and broccoli may be a new weapon against the disease.

The study suggests that vitamin C may tell faulty stem cells in our bone marrow to mature and die.

That means the traditional blood cancer danger cells would naturally disappear instead of multiplying to cause leukaemia.

The findings were uncovered by researchers from Perlmutter Cancer Center in New York and published in the cancer journal Cell. Perlmutter director Professor Benjamin G. Neel said: Were excited by the prospect that highdose vitamin C might become a safe treatment for blood diseases caused by leukaemia stem cells, most likely in combination with other targeted therapies.

Vitamin C is an antioxidant and several previous studies had hinted that high levels could affect cancer cells. High vitamin C fruit and vegetables include bell peppers, dark leafy greens, kiwifruit, broccoli, berries, oranges, tomatoes, green peas, and papayas.

The current recommended daily value for vitamin C is 60mg taken from either fruit and vegetables or tablet supplements.

The New York study explored the link between vitamin C and a tumour suppressor protein enzyme in the human body called TET2.

The enzyme helps to guard against blood cancers such as leukaemia and is believed also to protect against heart disease.

But mutations in the gene affect about one per cent of the over-65s, making them extremely susceptible to blood cancer.

Although TET2 loss does not create cancer, it helps to create the conditions for blood cancers to thrive.

Scientists in the New York study found that, in mice engineered to have just small amounts of TET2, high doses of vitamin C given intravenously dramatically activated the enzyme.

The study found changes in the genetic code that reduces TET2 function are found in 10 per cent of patients with acute myeloid leukaemia (AML).

The scientists also claim that, when they implanted leukaemia cancer stem cells from human patients into mice, high doses of vitamin C suppressed the cells growth.

Anna Perman, Cancer Research UKs senior science information manager, said: Some doctors think that antioxidants like vitamin C might interfere with chemotherapy which, we know can be effective treatment.

The important thing for cancer patients to remember is that this study is looking at the action of vitamin C in the laboratory, not the effect of eating foods or supplements that contain vitamin C.

This should not prompt anyone receiving treatment for cancer to change their diet or treatment plan.

The rest is here:
Vitamin C could help to fight cancer, a study claims - Express.co.uk

Fertile sperm are rare in men with an extra sex chromosome

Dennis Kunkle Microscopy/SPL

By Andy Coghlan

Turning skin cells into sperm may one day help some infertile men have babies. Research in mice has found a way to make fertile sperm from animals born with too many sex chromosomes.

Most men have two sex chromosomes one X and one Y but some have three, which makes it difficult to produce fertile sperm. Around 1 in 500 men are born with Klinefelter syndrome, caused by having an extra X chromosome, while roughly 1 in 1000 have Double Y syndrome.

James Turner of the Francis Crick Institute in London and his team have found a way to get around the infertility caused by these extra chromosomes. First, they bred mice that each had an extra X or Y chromosome. They then tried to reprogram skin cells from the animals, turning them into induced pluripotent stem cells (iPS), which are capable of forming other types of cell.

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To their surprise, this was enough to make around a third of the skin cells jettison their extra chromosome. When these cells were then coaxed into forming sperm cells and used to fertilise eggs, 50 to 60 per cent of the resulting pregnancies led to live births.

This suggests that a similar technique might enable men with Klinefelter or Double Y-related infertility to conceive. But there is a significant catch.

We dont yet know how to fully turn stem cells into sperm, so the team got around this by injecting the cells into mouse testes for the last stages of development. While this led to fertile sperm, it also caused tumours to form in between 29 and 50 per cent of mice.

What we really need to make this work is being able to go from iPS cells to sperm in a dish, says Turner.

It has to be done all in vitro, so only normal sperm cells would be used to fertilise eggs, says Zev Rosenwaks of the Weill Cornell Medical College in New York. The danger with all iPS cell technology is cancer.

Journal reference: Science, DOI: 10.1126/science.aam9046

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Stem cell technique could reverse a major type of infertility - New Scientist

Blumen has been diagnosed with Chronic Myeloid Leukemia and is awaiting a donor stem cell that matches his DNA

Running a difficult race isn't something that's new to Chennai-based BlumenRajan Sathya. An exceptionally gifted track athlete, a state record holder, a University gold medalist and a national level silver medalist in the 400m sprint, Blumenhad always been one to push his physical limits.

But this time, he's facing the most difficult track of his life. In December 2014, there was a sudden drop in his blood count. He was soon diagnosed with Chronic Myeloid Leukemia, a type of cancer which starts in certain blood-forming cells of the bone marrow.

CML is a treatable condition, where the first level of treatment is oral chemotherapy, followed by the usual induction chemo. However, the most-effective proven treatment is stem cell transplant, which is basically where you transplant a stem cell from a donor whose DNA matches with you."We've been hunting for donors. The only problem is that the probability of finding a match is one in a lakh. We're looking at international registries as well. I had contacted a registry in Germany while I did my homework online. But, they replied saying that the patient couldn't contact them directly," says the 27-year-old.

A graduate in Social Work from Madras Christian College and currently working with a local church, he adds, "In another three or four days, we will go ahead with the closest match available. We will wait for a hundred per cent match, but we can't wait too long."

When asked what kept him going strong throughout his whole battle, he says it was his faith in God and the support of his local church. Friends, family, colleagues and college mates have spread the word on social media, hoping for a miracle. Blumennow wants to ensure that there is awareness created about stem cell donation. "Most people have no clue about it. Most of us have never registered anywhere. There should be more awareness camps in colleges. If more people register, it would be much easier to find the right match. There won't be any trouble of finding volunteers," he says.

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Chennai sprinter Blumen Rajan is in a race against time to beat cancer. Are you the stem cell donor who can help out ... - EdexLive (press release)...

New research funded by the National Institutes of Health used 3-D collections of brain tissue grown from human cells to study the brains star-shaped astrocytes. (Image credit: Sergiu Pasca, Stanford University)

BETHESDA, Md. (CN) Its been two years since Stanford neurobiologists published a method for converting adult skin cells into induced pluripotent stem cells that could then be grown into 3-D clusters of brain cells.

The National Institutes of Health reported Wednesday that another crop of scientists have been studying the growth of star-shaped brain cells known as human cortical spheroids (hCSs) in these cell clusters.

Their findings, published in Neuron, confirm that the maturation of lab-grown cells largely mimics that of cells taken directly from brain tissue during very early life, a critical time for brain growth.

Because of the critical role this process plays in normal brain development, further study of lab-grown hCSs could uncover the underlying developmental biology at the core of various neurological and mental health disorders, such as schizophrenia and autism.

The hCS system makes it possible to replay astrocyte development from any patient, said Ben Barres, a Stanford professor of neurobiology who co-led the 2015 study, as quoted the NIH in a Wednesday article.

Thats huge, Barres added. Theres no other way one could ever do that without this method.

Steven Sloan, a student in Stanfords MD/Ph.D. program, led the astrocyte-comparison study published in the latest issue of Neuron.

The team grew the hCSs for 20 months, one of the longest-ever studies of lab-grown human brain cells, according to the report by the NIH, which funded the research in part through its National Institute of Neurological Disorders and Stroke.

Jill Morris, who directs the NINDS, said the work by Sloans team addresses a significant gap in human brain research by providing an invaluable technique to investigate the role of astrocytes in both normal development and disease.

David Panchision, program director at the National Institute of Mental Health, which also helped fund the study, also spoke to the studys importance.

Since astrocytes make up a greater proportion of brain cells in humans than in other species, it may reflect a greater need for astrocytes in normal human brain function, with more significant consequences when they dont work correctly, Panchision added.

One point that the researchers emphasized, however, is that hCSs are only a model and lack many features of real brains.

Moreover, certain genes that are active in fully mature astrocytes never switched on in the hCS-grown astrocytes, which they could conceivably do if the cells had more time to develop, the NIH article says. To address this question, the researchers now hope to identify ways to produce mature brain cells more quickly. hCSs could also be used to scrutinize precisely what causes astrocytes to change over time and to screen drugs that might correct any differences that occur in brain disease.

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New Cell Study Pulls Curtain on Schizophrenia, Autism - Courthouse News Service

JENNIE McKEON @JennieMnwfdn

NICEVILLE Jack Massey is ready to go back to school.

Only this time, the University of Florida senior will head back to campus with his mom and a new outlook on life.

Massey suffered a spinal cord injury in a pool accident in March and is paralyzed from the chest down. After months of rehab, he's eager to get back into a familiar routine.

"It's definitely boring," the 21-year-old said at his parents' home in Niceville. "There's not a lot to do. I want to go back to school. I still have my brain. I still have everything I need to be successful."

After the accident March 17, Massey was treated at the University of Florida Shands Hospital and then was transferred to Shepherd Center, a spinal cord and brain injury rehab center in Atlanta. At Shepherd Center he met with a peer mentor, counselors and physical therapists to help him find a new normal.

Jack has remained positive throughout the past six months.

"Jack has been a fighter through all of this," said his mother, Julie. "I think he's done well. I only saw him break down once."

Before the accident, Jack was a well-rounded athlete who playing baseball and basketball and ran. He was a star on the track and field team at Niceville High School, with his 4 X 800 relay winning state his senior year.

He says the biggest challenge now is not being able to do the same things he could before.

"I can't get up and go," he said. "It didn't really start to set in until after I got out of rehab."

Jack has had to find enjoyment in other things, like reading or playing with the dogs. His friends have learned to transfer him from his wheelchair to a car so they can take him to the movies or out to eat. When they recently took a trip to the beach, Julie said five of Jack's friends carried him out to the sand a lesson on how hard it is to navigate the world in a wheelchair.

Jack said he believes technology one day will advance enough that he won't be paralyzed forever. He also volunteered to do stem cell surgery to allow doctors to study the affects of stem cells on his spine for the next 15 years. Instead of wallowing in self pity, he's moving forward. But he'll need help.

"I'm appreciating everything in the now," he said.

Doctors have said Jack has adapted faster than expected, but there are still some everyday essential tasks that are out of his reach. He cannot write or cook. He can shower himself but can't dry himself or transfer himself in and out of his wheelchair. The Massey family hopes to secure a personal care attendant for Jack at school, but until then Julie will be in Gainesville to help him transition. An occupational therapy student from the university will also help Jack on a temporary basis.

Finding proper care for her son has proven to be a learning experience for Julie and her husband, Lance.

"I don't know how people do it," she said. "We have good health care, but then there's hidden costs. There's travel expenses. ... It's kind of humbling. Nobody should have to go to GoFundMe for medical help."

Jack wants to spend his final year as an undergrad as independent as possible. After months of helping him recover, Julie said it will be hard to let her son go. Jack is the oldest of three; his brother Lance is 19 and a student at UF and his sister Alina is 14 and attends Ruckel Middle School.

"It's like letting him go off to kindergarten again," she said.

As for life after college, Jack said he doesn't feel limited in career choices. One of his professors in the geology department encouraged him by saying that there were plenty of opportunities he could pursue in that field. Jack said he may also consider law school. One thing he's learned through this life-altering experience is that there are no limits to what he can achieve.

"I haven't done that much deep thinking. I just go with the flow," he said. "But I learned I have more perseverance. I'm more mentally tough than I thought I was. I'm appreciative for life in general. That's one of the big things."

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'I still have my brain' - The Northwest Florida Daily News