(Ivanhoe Newswire) CLEVELAND, Ohio -- In 1990 the Human Genome Project started. It was a massive scientific undertaking that aimed to identify and map out the body's complete set of DNA. This research has paved the way for new genetic discoveries; one of those has allowed scientists to study how to fix bad chromosomes.

Our bodies contain 23 pairs of them, 46 total. But if chromosomes are damaged, they can cause birth defects, disabilities, growth problems, even death.

Case Western scientist Anthony Wynshaw-Boris is studying how to repair damaged chromosomes with the help of a recent discovery. He's taking skin cells and reprogramming them to work like embryonic stem cells, which can grow into different cell types.

You're taking adult or a child's skin cells. You're not causing any loss of an embryo, and you're taking those skin cells to make a stem cell. Anthony Wynshaw-Boris, M.D., PhD, of Case Western Reserve University, School of Medicine told Ivanhoe.

Scientists studied patients with a specific defective chromosome that was shaped like a ring. They took the patients' skin cells and reprogrammed them into embryonic-like cells in the lab. They found this process caused the damaged ring chromosomes to be replaced by normal chromosomes.

It at least raises the possibility that ring chromosomes will be lost in stem cells, said Dr. Wynshaw-Boris.

While this research was only conducted in lab cultures on the rare ring-shaped chromosomes, scientists hope it will work in patients with common abnormalities like Down syndrome.

What we're hoping happens is we might be able to use, modify, what we did, to rescue cell lines from any patient that has any severe chromosome defect, Dr. Wynshaw-Boris explained.

It's research that could one day repair faulty chromosomes and stop genetic diseases in their tracks.

The reprogramming technique that transforms skin cells to stem cells was so ground-breaking that a Japanese physician won the Nobel Prize in medicine in 2012 for developing it.

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Published November 07, 2014

A team of stem cell scientists has identified the biological mechanisms of Parkinsons disease and recreated a model of the disease in a dish.

Researchers at The New York Stem Cell Foundation (NYSCF) Research Institute studied a pair of identical twins one with Parkinsons and one without as well as another unrelated Parkinsons patient and four healthy control subjects to observe key characteristics of the disease. After comparing the individuals biological factors, they noticed differences in the patients neurons ability to produce dopamine. Dopamine production is deficient in Parkinsons disease.

"The unique scenario of identical twins, one with this disease and one without, allowed our scientists an unprecedented look into the mechanisms of Parkinson's disease," Susan L. Solomon, NYSCF chief executive officer, said in a news release. "Advanced stem cell research techniques allow us to push the boundaries of science and see what actually goes wrong at the cellular level, step by step during the disease process."

Parkinsons disease affects an estimated 500,000 people in the United States, according to the National Institutes of Health (NIH). The average age of onset is 60, and the risk of developing it increases with age. Symptoms of Parkinsons include tremor, shaking in the hands, arms, legs, jaw or head; impaired balance or postural instability; slowness of movement; and stiffness of the limbs and trunk.

There is currently no cure for Parkinsons.

While the disease is moderately hereditary, scientists have yet to fully understand the mechanisms of inheritance. The researchers note the DNA mutations that produce the enzyme glucocerebrosidase (GBA) have been linked to a five-fold increased risk of developing Parkinsons, but only 30 percent of people with this mutation have been shown to get the disease by age 80. This suggests that genetic and non-genetic factors cause Parkinsons. In studying the identical twins, scientists were able to analyze these mechanisms.

The scientists made induced pluripotent stem (iPS) cells from skin samples from both twins to generate a cellular model of Parkinsons in a dish, recreating the outstanding features of the disease specifically the dopamine and a-synuclein deficiency.

Scientists saw that the neurons from the twin affected by Parkinsons produced less dopamine and had higher levels of an enzyme called monomine oxidase B (MAO-B), as well as a poorer ability to connect with each other, compared to the twin that did not have the disease.

The findings suggest a possible therapy for Parkinsons: treating neurons with molecules that reduce the activity of MAO-B and GBA, while normalizing -synuclein and dopamine levels.

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Stem Cell Therapy for Pets in Central Florida
http://www.NewmanVets.com Call your local Newman Veterinary Center for more information about stem cell therapy for pets. https://www.youtube.com/watch?v=7X23bmsy0Q8 feature=youtu.be.

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Stem Cell Therapy for Multiple Sclerosis: Ron McGill
Ron McGill suffers from relapsing-remitting multiple sclerosis. He was started experiencing symptoms in 2009 but was not diagnosed with MS until January of 2013. He received several infusion...

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For more than 30 years, stem cells have been the great hope of medical science. Given their remarkable ability to turn into any type of cell in the body, researchers have theorized that they could be used to treat and perhaps even cure all sorts of diseases and conditions from spinal cord injury to baldness.

Progress has been painfully slow for most areas of research but this week researchers in Sweden are reporting a major advancein a possible stem cell treatment for Parkinson's. While the treatment has only been tried in rats, the scientists -- led byMalin Parmar, an associate professor of regenerative neurobiology at the Lund University -- said they believe the results are promising enoughto move to clinical trials in humans within a few years.

A degenerative condition of the central nervous system, Parkinson's affects an estimated 7 to 10 million people worldwide -- including actor Michael J. Fox and Google co-founder Sergey Brin, both of whom have not only raised awareness of the disease through their celebrity but have contributed millions of dollars to advance research.

Parkinson's is caused by the loss of dopamine-producing cells in the brain that help regulate things like movement and emotions. The scientistsat the Lund University found that when they turned human embryonic stem cells into neurons that produce dopamine and injected them into the brains of rats, something remarkable happened. The damage from the disease seemed to reverse.

The scientists wrote that while they believe their research was "rigorous," they pointed out that "a number of crucial issues" still need to be addressed before the treatment can be tested in humans. For instance, they need to make sure the cells continue to work the way are supposed to over longer time periods.

Ariana Eunjung Cha is a national reporter for the Post. She has previously served as the newspapers bureau chief in Beijing, Shanghai and San Francisco, a correspondent in Baghdad and as a tech reporter based in Washington.

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New hope for Parkinsons patients in stem cell treatment

Enliven: Journal of Anesthesiology and Critical Care Medicine ISSN : 2374 - 4448 I e001
Left Ventricular Assist Device and Resident Cardiac Stem Cells in Heart Failure: Human Heart #39;s Potential Matter.

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An eight-year-old girl whose hunt for a bone marrow donor inspired hundreds to take selfies with their pants on their heads has died.

Hollie Clarks family broke the news on the Facebook page that they used to raise awareness of her battle with the bone marrow disorder MDS, Wales Online reports.

Her dad Stephen of Penylan, Cardiff, wrote: Sad news today Im afraid.

"After a seven month battle with MDS Hollie past away peacefully in her parents arms. It is utterly heartbreaking and makes no sense.

We have a million memories and take huge comfort in the number of Anthony Nolan registrations that the campaign made.

We are sure that someday soon one of you will be asked to donate stem cells and give someone like Hollie a chance.

Thank you all for your support. We would appreciate some time and space to try and pick ourselves up. Love from Hollies Dad. The proudest Dad in the world.

The family also used the @HelpHollie Twitter account to tell all those who had supported their campaign the news.

They wrote: Very sadly today our brave little angel passed away. Hollie was the happiest child ever and we are blessed that she was part of our family.

RIP Hollie. We tried our best. You were the best daughter any mum and dad could wish for. Thank you.

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Inspirational girl loses fight with rare condition - after encouraging hundreds to wear pants on head

A team led by New York Stem Cell Foundation (NYSCF) Research Institute scientists conducted a study comparing induced pluripotent stem (iPS) cells and embryonic stem cells created using somatic cell nuclear transfer (SCNT). The scientists found that the cells derived from these two methods resulted in cells with highly similar gene expression and DNA methylation patterns. Both methods also resulted in stem cells with similar amounts of DNA mutations, showing that the process of turning an adult cell into a stem cell introduces mutations independent of the specific method used. This suggests that both methods of producing stem cells need to be further investigated before determining their suitability for the development of new therapies for chronic diseases.

The NYSCF Research Institute is one of the only laboratories in the world that currently pursues all forms of stem cell research including SCNT and iPS cell techniques for creating stem cells. The lack of laboratories attempting SCNT research was one of the reasons that the NYSCF Research Institute was established in 2006.

"We do not yet know which technique will allow scientists to create the best cells for new cellular therapies," said Susan L. Solomon, NYSCF CEO and co-founder. "It is critical to pursue both SCNT and iPS cell techniques in order to accelerate research and bring new treatments to patients."

While both techniques result in pluripotent stem cells, or cells that can become any type of cell in the body, the two processes are different. SCNT consists of replacing the nucleus of a human egg cell or oocyte with the nucleus of an adult cell, resulting in human embryonic stem cells with the genetic material of the adult cell. In contrast, scientists create iPS cells by expressing a few key genes in adult cells, like a skin or blood cell, causing the cells to revert to an embryonic-like state. These differences in methods could, in principle, result in cells with different properties. Advances made earlier this year by NYSCF Research Institute scientists that showed that human embryonic stem cells could be derived using SCNT revived that debate.

"Our work shows that we now have two methods for the generation of a patient's personal stem cells, both with great potential for the development of treatments of chronic diseases. Our work will also be welcome news for the many scientists performing basic research on iPS cells. It shows that they are likely working with cells that are very similar to human embryonic stem cells, at least with regard to gene expression and DNA methylation. How the finding of mutations might affect clinical use of stem cells generated from adult cells is the subject of an ongoing debate," said Dr. Dieter Egli, NYSCF Senior Research Fellow, NYSCF -- Robertson Investigator, Assistant Professor in Pediatrics & Molecular Genetics at Columbia University, and senior author on the paper.

The study, published today in Cell Stem Cell, compared cell lines derived from the same sources using the two differing techniques, specifically contrasting the frequency of genetic coding mutations seen and measuring how closely the stem cells matched the embryonic state through the analysis of DNA methylation and of gene expression patterns. The scientists showed that both methods resulted in cell types that were similar with regard to gene expression and DNA methylation patterns. This suggested that both methods were effective in turning a differentiated cell into a stem cell.

The scientists also showed that cells derived using both SCNT and iPS techniques showed similar numbers of genetic coding mutations, implying that neither technique is superior in that regard. A similar number of changes in DNA methylation at imprinted genes (genes that are methylated differentially at the maternal versus the paternal allele) were also found. It is important to note that both types of techniques led to cells that had more of these aberrations than embryonic stem cells derived from an unfertilized human oocyte, or than embryonic stem cells derived from leftover IVF embryos. These findings suggest that a small number of defects are inherent to the generation of stem cells from adult differentiated cells and occur regardless of the method used.

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The above story is based on materials provided by New York Stem Cell Foundation. Note: Materials may be edited for content and length.

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SCNT derived cells, IPS cells are similar, study finds

David C P. Chen, MD., MHP - Stem Cell Therapy Q A1
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Newswise Hematopoietic stem cells (HSCs) give rise to all blood and immune cells throughout the life of vertebrate organisms, from zebrafish to humans. But details of their genesis remain elusive, hindering efforts to develop induced pluripotent stem cell (iPSC) replacements that might address a host of blood disorders.

In a paper published Nov. 20 in the journal Cell, researchers at the University of California, San Diego School of Medicine describe the surprising and crucial involvement of a pro-inflammatory signaling protein in the creation of HSCs during embryonic development, a finding that could help scientists to finally reproduce HSCs for therapeutic use.

The recent breakthrough of induced pluripotency has made the concept of patient-specific regenerative medicine a reality, said principal investigator David Traver, PhD, professor in the Department of Cellular and Molecular Medicine. The development of some mature cell lineages from iPSCs, such as cardiac and neural, has been reasonably straightforward, but not with HSCs. This is likely due, at least in part, to not fully understanding all of the factors used by the embryo to generate HSCs. We believe the discovery that pro-inflammatory cues are important in vivo will help us recapitulate instruction of HSC fate in vitro from iPSCs.

Traver and colleagues specifically looked at the role of a cytokine (a type of cell signaling protein) called tumor necrosis factor alpha or TNFa, which plays a pivotal role in regulating systemic inflammation and immunity. The work extended previous research by Spanish biologist Victoriano Mulero, who had reported that TNFa was important in the function of the embryonic vascular system and that in animal models where TNF function was absent, blood defects resulted.

The Cell papers first author Raquel Espin-Palazon, a postdoctoral researcher in Travers lab and a former colleague of Muleros, determined that TNFa was required for the emergence of hematopoietic stem cells during embryogenesis in zebrafish a common animal model.

Traver said the finding was completely unexpected because HSCs emerge relatively early in embryonic formation when the developing organism is considered to be largely sterile and devoid of infection.

Thus, there was no expectation that pro-inflammatory signaling would be active at this time or in the blood-forming regions, Traver said. Equally surprising, we found that a population of embryonic myeloid cells, which are transient cells produced before HSCs arise, are the producers of the TNFa needed to establish HSC fate. So it turns out that a small subset of myeloid cells that persist for only a few days in development are necessary to help generate the lineal precursors of the entire adult blood-forming system.

The newly discovered role of TNFa in HSC development mirrors a parallel discovery regarding interferon gamma (INFg), another cytokine and major mediator of pro-inflammatory signaling, highlighting multiple inputs for inflammatory signaling in HSC emergence. Traver said the crucial roles of TNFa and INFg in HSC emergence are likely similar in humans because of the highly conserved nature of HSC development across vertebrate evolution.

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Before There Will Be Blood

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Newswise LA JOLLAResearchers at the Salk Institute have healed injured hearts of living mice by reactivating long dormant molecular machinery found in the animals cells, a finding that could help pave the way to new therapies for heart disorders in humans.

The new results, published November 6 in the journal Cell Stem Cell, suggest that although adult mammals dont normally regenerate damaged tissue, they may retain a latent ability as a holdover from development like their distant ancestors on the evolutionary tree. When the Salk researchers blocked four molecules thought to suppress these programs for regenerating organs, they saw a drastic improvement in heart regeneration and healing in the mice.

The findings provide proof-of-concept for a new type of clinical treatment in the fight against heart disease, which kills about 600,000 people each year in the United Statesmore than AIDS and all cancer types combined, according to the U.S. Centers for Disease Control and Prevention.

Organ regeneration is a fascinating phenomenon that seemingly recapitulates the processes observed during development. However, despite our current understanding of how embryogenesis and development proceeds, the mechanisms preventing regeneration in adult mammals have remained elusive, says the studys senior author Juan Carlos Izpisua Belmonte, a professor in the Gene Expression Laboratory at Salk.

Within the genomes of every cell in our bodies, we have what information we need to generate an organ. Izpisua Belmontes group has for many years focused on elucidating the key molecules involved in embryonic development as well as those potentially underlying healing responses in regenerative organisms such as the zebrafish.

Indeed, back in 2003, Izpisua Belmontes laboratory first identified the signals preceding zebrafish heart regeneration. And in a 2010 Nature paper, the researchers described how regeneration occurred in the zebrafish. Rather than stem cells invading injured heart tissue, the cardiac cells themselves were reverting to a precursor-like state (a process called dedifferentiation), which, in turn, allowed them to proliferate in tissue.

Although in theory it might have seemed like the next logical step to ask whether mammals had evolutionarily conserved any of the right molecular players for this kind of regenerative reprogramming, in practice it was a scientific risk, recalls Ignacio Sancho-Martinez, a postdoctoral researcher in Izpisua Belmontes lab.

When you speak about these things, the first thing that comes to peoples minds is that youre crazy, he says. Its a strange sounding idea, since we associate regeneration with salamanders and fish, but not mammals.

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Salk Scientists Discover a Key to Mending Broken Hearts

TIME Health HIV/AIDS It May Be Possible To Prevent HIV Even Without a Vaccine "We're removing the doorway that HIV uses to get into cells"

Natural immunity is the most reliable way to protect yourself from viruses, bacteria and parasites. And the best way to acquire such immunity, in most cases, is to expose your immune system to the bug in questioneither by getting infected or getting immunized.

Until now, such protection was only possible with diseases like chicken pox or polio. But now, scientists at Harvard University say that people might soon arm themselves against HIV in a similar way, but through a different method.

Chad Cowan and Derrick Rossi, both in the department of stem cell and regenerative biology at Harvard University, and their colleagues report in the journal Cell Stem Cell that they have successfully edited the genomes of blood cells to make them impervious to HIV. In order survive, HIV needs to insert its genome into that of a healthy cell, and to infect these cells, HIV latches onto a protein on their surface called CCR5. If CCR5 is mutated, however, its as if the locks have been changed and HIV no longer has the right key; it cant attach itself and the cells are protected from infection. So the scientists tried a new gene editing technique called CRISPR that allows them to precisely snip out parts of a cells genome, and they spliced out the CCR5 gene. To their surprise, the technique was relatively efficient, transforming about half of the cells they treated with CRISPR into CCR5-free, or HIV-resistant, cells.

It was stunning to us how efficient CRISPR was in doing the genome editing, says Cowan.

Scientists have previously used CRISPR to make another change in how HIV infects cells; they snipped out the HIV genes that the virus inserted into healthy cells. That process essentially returned HIV infected cells back to healthy ones.

The latest results, however, suggest that the technique may be useful even before HIV gets inside cells. CRISPR could be useful in treating HIV patients if it can replace patients own immune cells with the blockaded versions. The cells Cowan and Rossi used were blood stem cells, which give rise to the bodys entire blood and immune system. In order to work as a potential treatment for HIV, patients would provide a sample of blood stem cells from their bone marrow, which would be treated with CRISPR to remove the CCR5 gene, and these cells would be transplanted back to the patient. Since the bone marrow stem cells populate the entire blood and immune system, the patient would eventually have blood cells that were protected, or immunized, against HIV. Were removing the doorway that HIV uses to get into cells, says Cowan.

To test this idea, they are already working with another research group to see if the HIV-impervious cells can treat mice infected with HIV.

Because healthy cells would be barricaded from HIV, the process might also lead to a cure for the disease. While the results are currently being tested to treat animals already infected with HIV, it may also be possible to one day transform a persons immune cell genomes to be protected against the virus. Some people are already fortunate enough to be protected this waya small percentage of people of European ancestry have natural immunity against HIV because they have two copies of mutated CCR5. They have been well studied and so far, their CCR5 aberrations dont seem to be linked to any known health issues. They are totally normal except for the fact that they are resistant to HIV, says Cowan. Thats a heartening thing: to have a group of people who are alive today who have been studied and looked at and seem totally fine.

Thats why clinicians who research the virus and treat HIV patients are excited by the possibilities of CRISPR-aided strategies. If its possible to close the door on HIV, then it may be realistic to start thinking about closing the door on the AIDS epidemic in the near future.

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6-Nov-2014

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation @nyscf

New York, NY (November 6, 2014) - A team led by New York Stem Cell Foundation (NYSCF) Research Institute scientists conducted a study comparing induced pluripotent stem (iPS) cells and embryonic stem cells created using somatic cell nuclear transfer (SCNT). The scientists found that the cells derived from these two methods resulted in cells with highly similar gene expression and DNA methylation patterns. Both methods also resulted in stem cells with similar amounts of DNA mutations, showing that the process of turning an adult cell into a stem cell introduces mutations independent of the specific method used. This suggests that both methods of producing stem cells need to be further investigated before determining their suitability for the development of new therapies for chronic diseases.

The NYSCF Research Institute is one of the only laboratories in the world that currently pursues all forms of stem cell research including SCNT and iPS cell techniques for creating stem cells. The lack of laboratories attempting SCNT research was one of the reasons that the NYSCF Research Institute was established in 2006.

"We do not yet know which technique will allow scientists to create the best cells for new cellular therapies," said Susan L. Solomon, NYSCF CEO and co-founder. "It is critical to pursue both SCNT and iPS cell techniques in order to accelerate research and bring new treatments to patients."

While both techniques result in pluripotent stem cells, or cells that can become any type of cell in the body, the two processes are different. SCNT consists of replacing the nucleus of a human egg cell or oocyte with the nucleus of an adult cell, resulting in human embryonic stem cells with the genetic material of the adult cell. In contrast, scientists create iPS cells by expressing a few key genes in adult cells, like a skin or blood cell, causing the cells to revert to an embryonic-like state. These differences in methods could, in principle, result in cells with different properties. Advances made earlier this year by NYSCF Research Institute scientists that showed that human embryonic stem cells could be derived using SCNT revived that debate.

"Our work shows that we now have two methods for the generation of a patient's personal stem cells, both with great potential for the development of treatments of chronic diseases. Our work will also be welcome news for the many scientists performing basic research on iPS cells. It shows that they are likely working with cells that are very similar to human embryonic stem cells, at least with regard to gene expression and DNA methylation. How the finding of mutations might affect clinical use of stem cells generated from adult cells is the subject of an ongoing debate," said Dr. Dieter Egli, NYSCF Senior Research Fellow, NYSCF - Robertson Investigator, Assistant Professor in Pediatrics & Molecular Genetics at Columbia University, and senior author on the paper.

The study, published today in Cell Stem Cell, compared cell lines derived from the same sources using the two differing techniques, specifically contrasting the frequency of genetic coding mutations seen and measuring how closely the stem cells matched the embryonic state through the analysis of DNA methylation and of gene expression patterns. The scientists showed that both methods resulted in cell types that were similar with regard to gene expression and DNA methylation patterns. This suggested that both methods were effective in turning a differentiated cell into a stem cell.

The scientists also showed that cells derived using both SCNT and iPS techniques showed similar numbers of genetic coding mutations, implying that neither technique is superior in that regard. A similar number of changes in DNA methylation at imprinted genes (genes that are methylated differentially at the maternal versus the paternal allele) were also found. It is important to note that both types of techniques led to cells that had more of these aberrations than embryonic stem cells derived from an unfertilized human oocyte, or than embryonic stem cells derived from leftover IVF embryos. These findings suggest that a small number of defects are inherent to the generation of stem cells from adult differentiated cells and occur regardless of the method used.

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Scientists find that SCNT derived cells and IPS cells are similar

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6-Nov-2014

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation @nyscf

New York, NY (November 6, 2014) - A team of scientists led by The New York Stem Cell Foundation (NYSCF) Research Institute successfully created a human stem cell disease model of Parkinson's disease in a dish. Studying a pair of identical (monozygotic) twins, one affected and one unaffected with Parkinson's disease, another unrelated Parkinson's patient, and four healthy control subjects, the scientists were able to observe key features of the disease in the laboratory, specifically differences in the patients' neurons' ability to produce dopamine, the molecule that is deficient in Parkinson's disease. In addition, the scientists also identified a potential strategy for developing novel therapies for Parkinson's disease.

Attributed to a combination of genetic and nongenetic factors, Parkinson's disease has no completely effective therapy or cure. Parkinson's disease is moderately heritable, but the mechanisms of this inheritance are not well understood. While genetic forms of the disease exist, sporadic forms are far more common.

"The unique scenario of identical twins, one with this disease and one without, allowed our scientists an unprecedented look into the mechanisms of Parkinson's disease," said Susan L. Solomon, NYSCF Chief Executive Officer. "Advanced stem cell research techniques allow us to push the boundaries of science and see what actually goes wrong at the cellular level, step by step during the disease process."

DNA mutations resulting in the production of a specific enzyme called glucocerebrosidase (GBA) have been linked to a five-fold greater risk of developing Parkinson's disease; however, only 30% of individuals with this mutation have been shown to develop Parkinson's disease by the age of 80. This discordance suggests that multiple factors contribute to the development of Parkinson's disease, including both genetic and non-genetic factors. To date, there has been no appropriate model to identify and test multiple triggers leading to the onset of the disease.

In this study, published today in Cell Reports, a set of identical twins, both with a GBA mutation, provided a unique opportunity to evaluate and dissect the genetic and non-genetic contributions to the development of Parkinson's disease in one twin, and the lack of disease in the other. The scientists made induced pluripotent stem (iPS) cells from skin samples from both twins to generate a cellular model of Parkinson's in a dish, recapitulating key features of the disease, specifically the accumulation of -synuclein and dopamine deficiency.

Upon analyzing the cell models, the scientists found that the dopamine-producing neurons from both twins had reduced GBA enzymatic activity, elevated -synuclein protein levels, and a reduced capacity to synthesize and release dopamine. In comparison to his unaffected brother, the neurons generated from the affected twin produced less dopamine, had higher levels of an enzyme called monoamine oxidase B (MAO-B), and poor ability to connect with each other. Treating the neurons with molecules that lowered the activity of MAO-B together with overexpressed GBA normalized -synuclein and dopamine levels in the cell models. This suggests that a combination therapy for the affected twin may be possible by simultaneously targeting these two enzymes.

"The subject of Parkinson's disease discordant twins gave us an incredible opportunity to utilize stem cell models of disease in a dish to unlock some of the biological mechanisms of disease," said Dr. Scott Noggle, NYSCF Vice President, Stem Cell Research and The NYSCF - Charles Evans Senior Research Fellow for Alzheimer's Disease. "Working with these various different groups and scientists added to the depth and value of the research and we hope our findings will be applicable to other Parkinson's disease patients and other neurodegenerative disorders."

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Scientists create Parkinson's disease in a dish

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Induced neural stem cells (iNSCs) created from adult cells hold promise for therapeutic transplantation, but their potential in this capacity has been limited by failed efforts to maintain such cells in the desirable multi-potent NSC state without continuous expression of the transcription factors used initially to reprogram them.

Now, Whitehead Institute scientists have created iNSCs that remain in the multi-potent state without ongoing expression of reprogramming factors. This allows the iNSCs to divide repeatedly to generate cells in quantities sufficient for therapy.

"Therapeutically, it's important to make neural stem cells because they can self-renew and make lots of cells," says Whitehead Institute Founding Member Rudolf Jaenisch, who is also a professor of biology at MIT. "If you just make mature neurons, which has been done by others, you never get enough cells."

To make iNSCs via direct lineage conversion researchers use viruses to insert a cocktail of transcription factors into the genome of mouse adult skin cells. A drug triggers these transcription factors to turn on genes active in neural stem cells. This direct conversion, known as transdifferentiation, bypasses the step of pushing the cells first through an embryonic stem-cell-like state.

In previous research, iNSCs remained addicted to the drug and reprogramming transcription factors; if either the drug or the factors was removed, the cells revert to skin cells.

"If the reprogramming factors are still active, it's horrible for the cells," says John Cassady, a scientist in Jaenisch's lab. "The cells would be unable to differentiate and the resulting cells would not be therapeutically useful."

In a paper published online this week in the current issue of the journal Stem Cell Reports, Cassady and other Whitehead scientists describe how they prevented the cells' relapse without keeping the reprogramming factors active. First, the cells were grown in a special medium that selects for neural stem cells. Then, the drug is removed. Instead of reverting into skin cells, the iNSCs remain in a multi-potent state that can differentiate into neurons and glia cells. Cassady also refined the reprogramming cocktail to contain eight transcription factors, which produces iNSCs that are transcriptionally and epigenetically similar to mouse neural stem cells.

Cassady notes that a random sample of skin cells can contain neural precursor cells, which can more easily make the transition to iNSCs. To eliminate the possibility that his method might actually rely on cells having this sort of "head start", Cassady converted fully mature immune system cells called B-lymphocytes, which have a very specific genetic marker, to iNSCs. The resulting cells had the profile of their new identity as iNSCs, yet retained their B-lymphocyte genetic marker, showing that Cassady's method could indeed convert non-neural cells to iNSCs.

Although promising, all of the work to date has been conducted in mouse cells. According to Cassady, researchers should next test this protocol in human cells to see if it can successfully produce the cell populations necessary for therapeutic use.

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Direct generation of neural stem cells could enable transplantation therapy

New research from McLean Hospital and the Harvard Stem Cell Institute has shown that stem cell therapy reduces seizures in mice.

Researchers used an animal model to transplant seizure-inhibiting, human embryonic stem cell-derived neurons into the brains of mice that had a common form of epilepsy. Half of the mice that received the transplanted neurons no longer had seizures, while the other half experienced a significant drop in seizure frequency.

The transplanted neurons integrated into the mouse brains and began to receive neuronal activity. The neurons then released GABA, an inhibitory response that reversed the electrical hyperactivity that causes seizure.

Previous studies showed increasing inhibition in the epileptic brain can help control the seizure and also a lot of anti-epilepsy drugs are mimicking this GABA, so many of them worked by binding to the GABA receptor inhibitor neuron, researcher Sangmi Chung, associate professor of psychiatry at Harvard, told FoxNews.com.

Researchers initially set out to test the functionality of human neurons, but later decided to test their effect on epilepsy because it is such a devastating disease. About 30 percent of people do not respond to seizure drugs and one out of 26 people will be affected by seizures in their lifetime, Chung said.

Over 65 million people worldwide are affected by epileptic seizures, which can cause convulsions, loss of consciousness and other neurological symptoms. Patients are treated with anti-seizure drugs, and may choose to have a portion of their brain removed.

Because mouse cells mature more quickly than human cells within weeks instead of years its unclear how long a stem cell transplant in a human would take before becoming effective, Chung noted.

If we compare it with the mouse [model], we believe it will be years, not weeks, she said.

However, the study found that, even without full maturation, the cells integrated into the epileptic mouse brains, receive signals and release GABA, therefore preventing seizures.

I think its really good news in terms of transplantation even maturing, not fully mature [cells] still work, Chung said.

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Stem cell transplants may help reduce seizures, study says

Korean stem cells regenerate coach Hiddinks worn-out knee cartilage NOVEMBER 06, 2014 03:02 Korean stem cells regenerate coach Hiddinks worn-out knee cartilage . NOVEMBER 06, 2014 03:02. . Guus Hiddink, the legendary soccer coach who led the Korean national team to the semifinals at the 2002 World Cup Korea-Japan, has been reborn as a figure symbolizing excellence of Korean stem cell treatment. Hiddink, who was suffering from severe arthritis, had his knee cartilage almost completely worn out. Rejecting recommendations to take artificial joint surgery by hospitals in the U.S. and Germany, Hiddink chose to take stem cell treatment in Korea. He started treatment in January this year, and was declared as having fully recovered from the illness 10 months later.

The treatment that gave coach Hiddink a second life is Cartistem, which is made from cord blood stem cells. Cartistem, which was developed by Medipost, a bio venture firm, received product licensure as treatment for knee cartilage that has been damaged due to degenerative conditions and repeated injuries from the Korea Food and Drug Ministry in January 2012. It was the first to receive licensure among stem cell treatments in the world. The treatment is undergoing clinical trials in the U.S. to acquire licensure from the Food and Drug Administration. Despite a highly costly price that is not covered by the national health insurance system, the treatment has been used in more than 1,600 patients thus far.

Stem cell treatments developed in Korea include Hearticellgram, a treatment for cardiac infarction, and Cupistem, a treatment for fistulous opening (a disease that causes holes in tissue between rectums and anus), as well as Cartistem. Hearticellgram and Cupistem use stem cells from tissues of the patients own body. Umbilical cord stem cells and autologous stem cells do not derive from human eggs and hence are free from controversy of bioethics. They are results of steadfast research and investment in stem cells by Korean biotech firms.

In tune with the aging society and a growing number of people with chronic diseases, the bio industry is considered a cash cow industry of the future. Notably, the stem cell sector that treats abnormal bodily organs is the most promising field. Not only bio powerhouses such as the U.S., Japan and the European Union but also China have jumped into the industry. As research on induced pluripotent stem cells (iPS) won the Nobel Prize in physiology and medicine recently, countries worldwide are having mounting interest in the field. The Korean government should create an environment to enable Korean biotech firms to take a leap forward in the global market, by providing generous support for investment and putting in place prompt and predictable licensure and approval process.

The treatment that gave coach Hiddink a second life is Cartistem, which is made from cord blood stem cells. Cartistem, which was developed by Medipost, a bio venture firm, received product licensure as treatment for knee cartilage that has been damaged due to degenerative conditions and repeated injuries from the Korea Food and Drug Ministry in January 2012. It was the first to receive licensure among stem cell treatments in the world. The treatment is undergoing clinical trials in the U.S. to acquire licensure from the Food and Drug Administration. Despite a highly costly price that is not covered by the national health insurance system, the treatment has been used in more than 1,600 patients thus far.

Stem cell treatments developed in Korea include Hearticellgram, a treatment for cardiac infarction, and Cupistem, a treatment for fistulous opening (a disease that causes holes in tissue between rectums and anus), as well as Cartistem. Hearticellgram and Cupistem use stem cells from tissues of the patients own body. Umbilical cord stem cells and autologous stem cells do not derive from human eggs and hence are free from controversy of bioethics. They are results of steadfast research and investment in stem cells by Korean biotech firms.

In tune with the aging society and a growing number of people with chronic diseases, the bio industry is considered a cash cow industry of the future. Notably, the stem cell sector that treats abnormal bodily organs is the most promising field. Not only bio powerhouses such as the U.S., Japan and the European Union but also China have jumped into the industry. As research on induced pluripotent stem cells (iPS) won the Nobel Prize in physiology and medicine recently, countries worldwide are having mounting interest in the field. The Korean government should create an environment to enable Korean biotech firms to take a leap forward in the global market, by providing generous support for investment and putting in place prompt and predictable licensure and approval process.

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donga.com[English donga]

Durham, NC (PRWEB) November 05, 2014

Half of all traumatic injuries to the face result in a loss of teeth and the surrounding tissue and bone that once supported them, which in turn makes these types of injuries very debilitating and difficult to treat. But in a new study published in the latest issue of STEM CELLS Translational Medicine, doctors at the University of Michigan School of Dentistry (UMSoD), Ann Arbor, have found a new way to regenerate a patients jawbone through the use of stem cells.

The procedure, done under local anesthesia, significantly speeds up the healing time relative to that of traditional bone grafting while allowing a patient to experience only a minimal amount of pain.

Part of a larger clinical trial, the findings highlighted in this issue focus on a 45-year-old woman missing seven front teeth plus 75 percent of the bone that once supported them, the result of a blow to her face five years earlier. She was left with severe functional and cosmetic deficiencies, since the missing bone made it impossible for her to have dental implant-based teeth replacements.

Darnell Kaigler, DDS, MS, PhD, an assistant professor of dentistry in the Department of Periodontics and Oral Medicine, was a lead member of the study team. "In small jawbone defects of the mouth created after teeth were extracted, we have placed gelatin sponges populated with stem cells into these areas to successfully grow bone."

Since the sponge material is soft, it does not work in larger areas. Thus, he and his team of researchers decided to try b-tricalcium phosphate (b-TCP) as a scaffold upon which to place the cells instead. "For treating larger jawbone defects, it is important to have a scaffold material that is rigid and more stable to support bone growth," he explained.

They then placed the b-TCP scaffold, which had been seeded with a mixed population of bone marrow-derived autologous stem and progenitor cells 30 minutes prior to treatment at room temperature, into the defective area of the patients mouth during a procedure that requires only local anesthesia. Four months later, 80 percent of her missing jawbone had been regenerated, allowing them to proceed with placing oral implants that supported a dental prosthesis to once again give her a complete set of teeth.

Study team member Sharon Aronovich, DMD, FRCD(C), a clinical assistant professor of dentistry in the Department of Oral and Maxillofacial Surgery at the UMSoD, said, I am very grateful to all the patients and researchers that participated in this study. Thanks to everyone's efforts, we are one step closer to providing patients with a minimally invasive option for implant-based tooth replacement.

As the first report to describe a cell therapy for craniofacial trauma reconstruction, this research serves as the foundation for expanded studies using this approach, said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

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Stem cells help doctors restore womans smile, regenerating bone to hold dental implants