Genetherapy

Personalized Medicine 2 THIRD – Video

February 25th, 2014

Personalized Medicine 2 THIRD

By: Grace Earley

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Personalized Medicine 2 THIRD - Video

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Autonomic Dysreflexia and Spinal Cord Injury – Video

February 25th, 2014

Autonomic Dysreflexia and Spinal Cord Injury
This video explains Autonomic Dysreflexia from the perspective of a person with a C6 C7 complete Spinal Cord Injury. How it can be dangerous if not treated u...

By: Matt Valente

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Autonomic Dysreflexia and Spinal Cord Injury - Video

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16 week old paralyzed Aussie pup gets her second set of Eddie’s Wheels – Video

February 25th, 2014

16 week old paralyzed Aussie pup gets her second set of Eddie #39;s Wheels
Abby, a 16 week old Australian Shepherd puppy, is renting a succession of wheelchairs from Eddie #39;s Wheels. A spinal cord injury one week after her birth has ...

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Scientists transform human skin cells into mature liver cells

February 24th, 2014

In a medical first, scientists at the Gladstone Institutes and the University of California, San Francisco (UCSF) have transformed human skin cells into mature, fully functioning liver cells.

Additionally, these cells can thrive on their own after being transplanted into laboratory animals a positive step for future treatment for liver failure.

So far, scientists have been able turn skin cells into cells closely resembling heart cells and pancreas cells, but there hasnt been a method to generate cells that are fully mature. And previous studies on liver-cell reprogramming had difficulties getting the stem-cell-derived liver cells to survive and flourish once transplanted inside the body.

But in this latest study, published in the journal Nature, researchers figured out a way to overcome these obstacles.

Earlier studies tried to reprogram skin cells back into a pluripotent, stem cell-like state in order to then grow liver cells, senior author Sheng Ding, a professor of pharmaceutical chemistry at UCSF, said in a press release. However, generating these so-called induced pluripotent stem cells, or iPS cells, and then transforming them into liver cells wasnt always resulting in complete transformation. So we thought that, rather than taking these skin cells all the way back to a pluripotent, stem cell-like state, perhaps we could take them to an intermediate phase.

Dings regeneration method involved using a specific cocktail of reprogramming genes and chemical compounds. This mixture helped to transform the skin cells into cells resembling those in the endoderm an embryonic cell layer that eventually forms many of the bodys major organs. According to the researchers, this state allowed the cells to be more easily coaxed into becoming liver cells.

Then, using another set of genes and compounds, Ding and his team transformed the endoderm-like cells into nearly indistinguishable liver cells. To see how well these cells performed on their own, the researchers implanted them into the livers of mice that had been genetically altered to experience liver failure. Nine months post-transplantation, the team saw an overall rise in human liver protein levels an indication that the liver cells were growing and thriving.

This study has major implications for those suffering from liver failure, as a costly liver transplant is often the only form of treatment.

Many questions remain, but the fact that these cells can fully mature and grow for months post-transplantation is extremely promising, said Dr. Holger Willenbring, associate director of the UCSF Liver Center and the papers other senior author. In the future, our technique could serve as an alternative for liver-failure patients who dont require full-organ replacement, or who dont have access to a transplant due to limited donor organ availability.

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Scientists transform human skin cells into mature liver cells

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Skin cells transformed into functioning liver cells in mouse study

February 24th, 2014

The power of regenerative medicine now allows scientists to transform skin cells into cells that closely resemble heart cells, pancreas cells and even neurons. However, a method to generate cells that are fully mature -- a crucial prerequisite for life-saving therapies -- has proven far more difficult. But now, scientists at the Gladstone Institutes and the University of California, San Francisco (UCSF), have made an important breakthrough: they have discovered a way to transform skin cells into mature, fully functioning liver cells that flourish on their own, even after being transplanted into laboratory animals modified to mimic liver failure.

In previous studies on liver-cell reprogramming, scientists had difficulty getting stem cell-derived liver cells to survive once being transplanted into existing liver tissue. But the Gladstone-UCSF team figured out a way to solve this problem. Writing in the latest issue of the journal Nature, researchers in the laboratories of Gladstone Senior Investigator Sheng Ding, PhD, and UCSF Associate Professor Holger Willenbring, MD, PhD, reveal a new cellular reprogramming method that transforms human skin cells into liver cells that are virtually indistinguishable from the cells that make up native liver tissue.

These results offer new hope for the millions of people suffering from, or at risk of developing, liver failure -- an increasingly common condition that results in progressive and irreversible loss of liver function. At present, the only option is a costly liver transplant. So, scientists have long looked to stem cell technology as a potential alternative. But thus far they have come up largely empty-handed.

"Earlier studies tried to reprogram skin cells back into a pluripotent, stem cell-like state in order to then grow liver cells," explained Dr. Ding, one of the paper's senior authors, who is also a professor of pharmaceutical chemistry at UCSF, with which Gladstone is affiliated. "However, generating these so-called induced pluripotent stem cells, or iPS cells, and then transforming them into liver cells wasn't always resulting in complete transformation. So we thought that, rather than taking these skin cells all the way back to a pluripotent, stem cell-like state, perhaps we could take them to an intermediate phase."

This research, which was performed jointly at the Roddenberry Center for Stem Cell Research at Gladstone and the Broad Center of Regeneration Medicine and Stem Cell Research at UCSF, involved using a 'cocktail' of reprogramming genes and chemical compounds to transform human skin cells into cells that resembled the endoderm. Endoderm cells are cells that eventually mature into many of the body's major organs -- including the liver.

"Instead of taking the skin cells back to the beginning, we took them only part way, creating endoderm-like cells," added Gladstone and CIRM Postdoctoral Scholar Saiyong Zhu, PhD, one of the paper's lead authors. "This step allowed us to generate a large reservoir of cells that could more readily be coaxed into becoming liver cells."

Next, the researchers discovered a set of genes and compounds that can transform these cells into functioning liver cells. And after just a few weeks, the team began to notice a transformation.

"The cells began to take on the shape of liver cells, and even started to perform regular liver-cell functions," said UCSF Postdoctoral Scholar Milad Rezvani, MD, the paper's other lead author. "They weren't fully mature cells yet -- but they were on their way."

Now that the team was encouraged by these initial results in a dish, they wanted to see what would happen in an actual liver. So, they transplanted these early-stage liver cells into the livers of mice. Over a period of nine months, the team monitored cell function and growth by measuring levels of liver-specific proteins and genes.

Two months post-transplantation, the team noticed a boost in human liver protein levels in the mice, an indication that the transplanted cells were becoming mature, functional liver cells. Nine months later, cell growth had shown no signs of slowing down. These results indicate that the researchers have found the factors required to successfully regenerate liver tissue.

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Skin cells transformed into functioning liver cells in mouse study

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Mature, functioning liver cells made from skin cells

February 24th, 2014

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There have been several reports in recent years of scientists reprogramming skin cells so they transform into cells that are similar to cells from other organs, such as the heart, the pancreas and even brain cells. However, these have fallen short of producing mature, fully functioning versions of organ cells - essential if they are to be of any use in life-saving regenerative medicine.

Now, a new study reported in Nature shows how it may be possible, with a new method, to transform skin cells into mature, fully functioning liver cells that are practically identical to native cells in liver tissue.

Not only this, but the new cells also flourish on their own, even after being transplanted into the livers of animals with engineered liver failure.

The results raise hopes for millions of people who have or who are at risk of developing liver failure. Currently, their only option is a liver transplant.

The breakthrough is the work of scientists at the Gladstone Institutes and the University of California, San Francisco (UCSF).

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Mature, functioning liver cells made from skin cells

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STAP stem cell doubts keep proliferating

February 24th, 2014

Doubts keep growing about the stunning discovery that super stem cells could be created merely by placing white blood cells from young mice in acid or otherwise stressing them, says Paul Knoepfler, a stem cell researcher at UC Davis.

Among other inconsistencies, Knoepfler referred to several unexplained anomalies in images of these STAP cells in two papers, published by the prestigious journal Nature on Jan. 29. One image appears to suggest signs that virtually all cells treated with an acid bath were being reprogrammed, a result that would be extraordinary. Stem cell reprogramming to date has been inefficient, with a low percentage of treated cells being reprogrammed.

"The more I look at these two STAP papers, the more concerned I get ... The bottom line for me now is that some level a part of me still clings to a tiny and receding hope this has all been overblown due to simple misunderstandings, but that seems increasingly unlikely," Knoepfler wrote Sunday on his blog, IPS Cell.

This undated image made available by the journal Nature shows a mouse embryo formed with specially-treated cells from a newborn mouse that had been transformed into stem cells. Researchers in Boston and Japan say they created stem cells from various tissues of newborn mice. If the same technique works for humans, it may provide a new way to grow tissue for treating illnesses like diabetes and Parkinson's disease. The report was published online on Wednesday, Jan. 29, 2014 in the journal Nature. (AP Photo/RIKEN Center for Developmental Biology, Haruko Obokata)

Nature is conducting its own investigation, Knoepfler noted. But in addition, the journal should release "unmodified, original versions" of the images and data in the papers, Knoepfler wrote.

The images contained "minor errors" that didn't change the basic findings, said Charles Vacanti, a Harvard University professor who is part of the scientific team reporting the discovery, according to a Feb. 22 article in a Japanese newspaper, the Asahi Shimbun.

Controversy is normal for any major scientific advance. Skeptics must be converted, and the only way to do that is to show the data. The 1997 announcement of the first mammalian clone, Dolly the sheep, was greeted with considerable doubt because it was believed that genetic imprinting made such cloning impossible. But others were eventually able to confirm the finding.

In this case, doubters say such an apparently easy method of reprogramming cells would generate pluripotent stem cells far too easily, because stress is common in animals. Such stem cells are known to cause tumors, so evolution should have selected against such a response.

Nature's own role has been criticized. The journal was taken to task for its handling of online journalism Feb. 20 by another stem cell blogger, Alexey Bersenev. He chided Nature for not linking to sources.

"In scientific journalism, every claim must be linked to appropriate original source," Berseney wrote. "Nature consistently refuses to acknowledge bloggers, online discussions and other web resources with valid credible information. This is not acceptable for sci journalism."

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STAP stem cell doubts keep proliferating

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GROW YOUR HAIR LONG! GIVEAWAY – Video

February 24th, 2014

GROW YOUR HAIR LONG! GIVEAWAY
okay so who want long gorgeous healthy hair!!!!???? SAY I, so I am giving away the whole ovation cell therapy line to one of you lovely ladies so you can be ...

By: REAL BEAUTY by Cynthia

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GROW YOUR HAIR LONG! GIVEAWAY - Video

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Genetic engineering genes proteomics gene bioinformatics – Video

February 24th, 2014

Genetic engineering genes proteomics gene bioinformatics

By: Iridium

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Genetic engineering genes proteomics gene bioinformatics - Video

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Exclusive David Gancberg article in Human Gene Therapy

February 24th, 2014

PUBLIC RELEASE DATE:

24-Feb-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, February 24, 2014Over the past three funding stages, the European Commission has invested nearly $475 million in 100 projects in the gene transfer and gene therapy field. David Gancberg, Directorate-General for Research and Innovation, European Commission (Brussels), describes the substantial opportunities for funding to support basic and clinical research in gene and cell therapy to find new treatments for chronic and rare diseases and novel regenerative medicine approaches in a Commentary article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website.

Dr. Gancberg and co-author Ruxandra Draghia-Akli state, "More than ever, the European Union offers to the gene and cell therapy sectors the possibilities of financial support to bright and innovative consortia ready to develop, possibly in collaboration with industry, new therapeutic applications to be tested in clinical trials, or novel products for the market, and build sustainable networks of expertise in the field," in their article "Gene and Cell Therapy Funding Opportunities in Horizon 2020: An Overview 2014-2015."

"Funding for gene therapy provided by the European Commission over the last decade has been critical to the current success we are seeing in the clinics. The commentary provided by doctors Gancberg and Draghia-Akli describe exciting new programs." says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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About the Journal

Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

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Exclusive David Gancberg article in Human Gene Therapy

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Is previous hypoglycemia a risk factor for future hypoglycemic episodes?

February 24th, 2014

PUBLIC RELEASE DATE:

24-Feb-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, February 24, 2014The automatic "threshold suspend" (TS) feature of an insulin pump helps prevent life-threatening hypoglycemic events when the device's sensor detects blood glucose concentrations below the preset threshold. However, in individuals with type 1 diabetes who have had previous episodes of hypoglycemia the TS feature may be less effective at preventing subsequent events, according to important new results from the ASPIRE study published in Diabetes Technology & Therapeutics (DTT), a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the DTT website at http://www.liebertpub.com/DTT.

In the article "Hypoglycemia Begets Hypoglycemia: The Order Effect in the ASPIRE In-Clinic Study," Editor-in-Chief of Diabetes Technology & Therapeutics Satish Garg, MD (Professor of Medicine and Pediatrics at the University of Colorado Denver) and coauthors compared the effects of an automatic insulin pump with TS at a preset sensor threshold to those of continued basal insulin delivery in adults with type 1 diabetes following induced hypoglycemia via overnight fasting and exercise.

The different outcomes seen between the two insulin delivery methods in this crossover study design led the authors to conclude that "By mitigating the duration of hypoglycemic episodes, automatic pump suspension may help to preserve the normal autonomic response to hypoglycemia in patients with type 1 diabetes."

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February 23rd, 2014

Researchers in Bern have discovered that, during a viral infection, immune cells control the blood stem cells in the bone marrow and therefore also the body's own defenses. The findings could allow for new forms of therapy, such as for bone marrow diseases like leukemia.

During a viral infection, the body needs various defense mechanisms -- amongst other things, a large number of white blood cells (leukocytes) must be produced in the bone marrow within a short period of time. In the bone marrow, stem cells are responsible for this task: the blood stem cells. In addition to white blood cells, blood stem cells also produce red blood cells and platelets.

The blood stem cells are located in specialized niches in the bone marrow and are surrounded by specialized niche cells. During an infection, the blood stem cells must complete two tasks: they must first recognise that more blood cells have to be produced and, secondly, they must recognise what kind of.

Now, for the first time, researchers at the Department of Medical Oncology at the University of Bern and Bern University Hospital headed by Prof. Adrian Ochsenbein have investigated how the blood stem cells in the bone marrow are regulated by the immune system's so-called T killer cells during a viral infection. As this regulation mechanism mediated by the immune system also plays an important role in other diseases such as leukemia, these findings could lead to novel therapeutic approaches. The study is being published in the peer-reviewed journal "Cell Stem Cell" today.

T Killer cells trigger defenses

One function of T killer cells is to "patrol" in the blood and remove pathogen-infected cells. However, they also interact with the blood stem cells in the bone marrow. The oncologists in Bern were able to show that messenger substances secreted by the T killer cells modulate the niche cells. In turn, the niche cells control the production and also the differentiation of the blood stem cells.

This mechanism is important in order to fight pathogens such as viruses or bacteria. However, various forms of the bone marrow disease leukemia are caused by a malignant transformation of exactly these blood stem cells. This leads to the formation of so-called leukemia stem cells. In both cases, the mechanisms are similar: the "good" mechanism regulates healthy blood stem cells during an infection, whilst the "bad" one leads to the multiplication of leukemia stem cells. This in turn leads to a progression of the leukemia.

This similarity has already been investigated in a previous project by the same group of researchers. "We hope that this will enable us to better understand and fight infectious diseases as well as bone marrow diseases such as leukemia," says Carsten Riether from the Department of Clinical Research at the University of Bern and the Department of Medical Oncology at Bern University Hospital and the University of Bern.

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The above story is based on materials provided by University of Bern. Note: Materials may be edited for content and length.

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Immune cells regulate blood stem cells, research shows

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McClellan: Bone marrow registry drives often pay it forward

February 23rd, 2014

On a Saturday last September, Be the Match Foundation sponsored a 5-kilometer walk and run in Creve Coeur Park to promote donor awareness. The foundation is an international bone marrow registry, and it coordinates marrow and stem cell transplants that are used to treat blood disorders.

Mark Pearl was at the event. Two of his three kids were born with a rare blood disorder called Fanconi anemia. Alexandra was diagnosed on Christmas Day 2000. She was 5. Her younger brother, Matthew, was diagnosed shortly thereafter. A marrow donor in Sweden was quickly found for Alexandra, but no matches were found for Matthew.

Mark and his wife, Diane, began organizing donor drives. Its easy to register as a donor. A couple of swabs on the inside of a cheek to collect DNA is all that is required. At their first drive in February 2001, they registered more than 4,000 potential donors. No matches. Over the next five and a half years, they organized more than 1,000 drives and registered more than 100,000 potential donors.

A donor was eventually found in North Carolina. As is almost always the case, the donor registered at someone elses drive. Matthew received his transplant in 2006.

He and his sister are fine.

Also at the event in Creve Coeur was Brian Jakubeck. He did not know Mark, but he had registered as a potential donor at one of the drives the Pearls had organized for Matthew. One of the last drives, actually.

How did that happen? Mark has season tickets for the Rams and sits next to Ted Cassimatis, who is a college friend of Brians brother. So as the Pearls reached out well beyond their own circle of friends, Ted sent out a mass email to his friends, and that email reached Brian. He and his wife, Kathy, registered as potential donors at a drive in May 2006.

Sometime later, Brian heard the good news from Ted that a donor had been found for his friends son.

Several years passed. In August 2012, Brian heard from Be the Match. He appeared to be a match. Would he agree to have some blood samples taken to confirm that he was a match? Sure, he said.

The results were positive. He was a match. He had more tests shortly before Christmas, and in January of last year, he went to St. Louis University Hospital and gave his stem cells. This was done in a process called apheresis. It is similar to giving plasma or platelets. The blood goes through an IV, passes through a machine that collects the stem cells, and then is returned through another IV. Its painless, but takes about six hours.

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McClellan: Bone marrow registry drives often pay it forward

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Scientists transform skin cells into functioning liver cells

February 23rd, 2014

PUBLIC RELEASE DATE:

23-Feb-2014

Contact: Anne Holden anne.holden@gladstone.ucsf.edu 415-734-2534

Jeff Norris JNorris@pubaff.ucsf.edu 415-476-8255

Gladstone Institutes

SAN FRANCISCO, CAFebruary 23, 2014The power of regenerative medicine now allows scientists to transform skin cells into cells that closely resemble heart cells, pancreas cells and even neurons. However, a method to generate cells that are fully maturea crucial prerequisite for life-saving therapieshas proven far more difficult. But now, scientists at the Gladstone Institutes and the University of California, San Francisco (UCSF), have made an important breakthrough: they have discovered a way to transform skin cells into mature, fully functioning liver cells that flourish on their own, even after being transplanted into laboratory animals modified to mimic liver failure.

In previous studies on liver-cell reprogramming, scientists had difficulty getting stem cell-derived liver cells to survive once being transplanted into existing liver tissue. But the Gladstone-UCSF team figured out a way to solve this problem. Writing in the latest issue of the journal Nature, researchers in the laboratories of Gladstone Senior Investigator Sheng Ding, PhD, and UCSF Associate Professor Holger Willenbring, MD, PhD, reveal a new cellular reprogramming method that transforms human skin cells into liver cells that are virtually indistinguishable from the cells that make up native liver tissue.

These results offer new hope for the millions of people suffering from, or at risk of developing, liver failurean increasingly common condition that results in progressive and irreversible loss of liver function. At present, the only option is a costly liver transplant. So, scientists have long looked to stem cell technology as a potential alternative. But thus far they have come up largely empty-handed.

"Earlier studies tried to reprogram skin cells back into a pluripotent, stem cell-like state in order to then grow liver cells," explained Dr. Ding, one of the paper's senior authors, who is also a professor of pharmaceutical chemistry at UCSF, with which Gladstone is affiliated. "However, generating these so-called induced pluripotent stem cells, or iPS cells, and then transforming them into liver cells wasn't always resulting in complete transformation. So we thought that, rather than taking these skin cells all the way back to a pluripotent, stem cell-like state, perhaps we could take them to an intermediate phase."

This research, which was performed jointly at the Roddenberry Center for Stem Cell Research at Gladstone and the Broad Center of Regeneration Medicine and Stem Cell Research at UCSF, involved using a 'cocktail' of reprogramming genes and chemical compounds to transform human skin cells into cells that resembled the endoderm. Endoderm cells are cells that eventually mature into many of the body's major organsincluding the liver.

Continue reading here:
Scientists transform skin cells into functioning liver cells

Recommendation and review posted by Bethany Smith