45 minutes ago A new method resulted in a colony of stem cells, glowing green, derived from one adult immune cell. Credit: Laboratory of Matthias Stadtfeld at NYU Langone Medical center

Scientists at NYU Langone Medical Center have found a way to boost dramatically the efficiency of the process for turning adult cells into so-called pluripotent stem cells by combining three well-known compounds, including vitamin C. Using the new technique in mice, the researchers increased the number of stem cells obtained from adult skin cells by more than 20-fold compared with the standard method. They say their technique is efficient and reliable, and thus should generally accelerate research aimed at using stem cells to generate virtually any tissue. Stem cells are immature or uncommitted cells that are theoretically capable of becoming any cell type.

"This big boost in efficiency gives us an opportunity now to study stem cell programming mechanisms at high resolution," says Matthias Stadtfeld, PhD, assistant professor of cell biology and a member of the Skirball Institute of Biomolecular Medicine and the Helen L. and Martin S. Kimmel Center for Stem Cell Biology at NYU Langone Medical Center, who led the research.

"This is a very exciting advance," says Ruth Lehmann, PhD, director of the Kimmel Center for Stem Cell Biology and the Skirball Institute at NYU Langone and chair of the Department of Cell Biology. "The new technology developed by the Stadtfeld lab to reprogram differentiated cells efficiently and effectively brings the prospect of stem cell technology for safe use in regenerative medicine ever so much closer."

The standard method for reprogramming skin, blood, or other tissue-specific cell types into "induced pluripotent stem cells" (iPSCs) was reported in 2006 by the laboratory of Kyoto University's Shinya Yamanaka, who later won a Nobel Prize for the achievement. The method involves the artificial expression of four key genes dubbed OKSM (for Oct4, Klf4, Sox2 and myc) whose collective activity slowly prods cells into an immature state much like that of an early embryonic cell.

In principle, one could take a sample of cells from a person, induce the cells to become iPSCs, then multiply the iPSCs in a lab dish and stimulate them to mature towards desired adult cell types such as blood, brain or heartwhich then could be used to replace injured or diseased tissue in that same individual.

But there are many formidable technical obstacles, among which is the low efficiency of currently used protocols. Converting most cell types into stable iPSCs occurs at rates of 1 percent or less, and the process can take weeks.

Researchers throughout the world have been searching for ways to boost this efficiency, and in some cases have reported significant gains. These procedures, however, often alter vital cellular genes, which may cause problems for potential therapies. For the new study, reported online today in Stem Cell Reports, Dr. Stadtfeld and his laboratory team decided to take a less invasive approach and investigate chemical compounds that transiently modulate enzymes that are present in most cells. "We especially wanted to know if these compounds could be combined to obtain stem cells at high efficiency," Dr. Stadtfeld says.

Two of these compounds influence well known signaling pathways, called Wnt and TGF-, which regulate multiple growth-related processes in cells. The third is vitamin C (also known as ascorbic acid). Best known as a powerful antioxidant, the vitamin was recently discovered to assist in iPSC induction by activating enzymes that remodel chromatinthe spiral scaffold for DNAto regulate gene expression.

Simon Vidal, a graduate student in the Stadtfeld lab, and Bhishma Amlani, a postdoctoral researcher, looked first at mouse skin fibroblasts, the most common cell type used for iPSC research. Adding to fibroblasts engineered to express OKSM either vitamin C, a compound to activate Wnt signaling, or a compound to inhibit TGF- signaling increased iPSC-induction efficiency weakly to about 1% after a week of cell culture. Combining any two worked a bit better. But combining all three brought the efficiency to about 80 percent in the same period of time.

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Released: 17-Sep-2014 10:00 AM EDT Source Newsroom: Alzforum Contact Information

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Newswise Rising to fame from humble beginnings in bacteria, the CRISPR gene editing system has taken the biomedical field by storm. Using an ever-evolving genetic toolbox to change, turn off, turn on, add, or delete genes, scientists can tinker with any region of the genome. The technology may one day allow clinicians to correct mutations in genetic diseases or deliver therapeutic genes to mend damaged cells or kill cancerous ones, scientists predict.

Neuroscientists have adopted CRISPR to neurons, both in a dish and in living animals, and they have set their sights on using the technology to treat neurodegenerative diseases such as ALS, Parkinsons, Huntingtons, and Alzheimers. In a two-part series, Alzforum explains the CRISPR technology and its first neuroscience forays (Part I), and delves into how scientists will engineer transgenic animals and perhaps treat disease through CRISPR gene therapy (Part II).

About Alzforum: Founded in 1996, Alzforum is a dynamic, Web-based scientific community dedicated to understanding Alzheimer's disease and related disorders. Access to the website is free to all. Our editorial priorities are as diverse as the needs of the research community. The website reports on the latest scientific findings from basic research to clinical trials, creates and maintains public databases of essential research data and reagents, and produces discussion forums to promote debate, speed the dissemination of new ideas, and break down barriers across the numerous disciplines that can contribute to the global effort to cure Alzheimer's disease. The Alzforum team of professional science writers and editors, information technology experts, Web developers, and producers all work closely with our distinguished Advisory Board to ensure a high quality of information and services. We very much welcome our readers' participation in all aspects of the website.

Alzforum is developed and operated by the Biomedical Research Forum (BRF) LLC. BRF is a wholly owned subsidiary of FMR LLC. FMR LLC and its affiliates invest broadly in many companies, including life sciences and pharmaceutical companies. The Alzforum website does not endorse any specific product or scientific approach.

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Products containing genetically modified ingredients would have to disclose that information on the back of the package near the nutritional facts under Vermont s bill. (Photo by John Herrick/VTDigger)

BRATTLEBORO -- Contending Vermont's GMO labeling law is meant "to pacify a vocal segment of the population that opposes genetic engineering," the Grocery Manufacturers Association is asking a federal court to prevent the state from implementing Act 120 until its lawsuit against Vermont "has run its course."

Act 120, which establishes labeling requirements for what it calls genetically engineered foods, was passed by the Legislature earlier this year and signed into law in May by Gov. Peter Shumlin.

According to the motion for an injunction, because GE varieties of corn and soybean account for 90 percent of those types of plantings in the United States, Act 120 will affect most of the grocery products sold in Vermont.

"Federal law does not require food labeling to also include plant labeling because there is no rational justification for such a regime," wrote attorneys for the GMA, the Snack Food Association, the International Dairy Association and the National Association of Manufacturers. "Act 120, however, is not concerned with rational justification. It caters to beliefs and biases that a government has no business endorsing."

Not only does Act 120 not serve any legitimate governmental interests, wrote the attorneys, it violates the First Amendment and intrudes upon federal labeling requirements, and is thus preempted by the Supremacy Clause.

In addition, they wrote, the members of the associations bringing suit against the state "will suffer irreparable injury" without an injunction because they have no way "to reliably distinguish ingredients derived from genetically engineered plant varieties from those that are not."

"The changes manufacturers would need to demand from their suppliers and initiate in their own facilities to segregate ingredients require money and time -- much more time than the Act's July 1, 2016, effective date allows," wrote the attorneys.

And then there are the costs related to building out Vermont-specific supply and distribution chains that do not currently exist.

"Plaintiff's members will not be able to recoup the cost of those efforts from the State if they prevail, nor could they easily return their business to the status quo ante."

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BRATTLEBORO -- Contending Vermont's GMO labeling law is meant "to pacify a vocal segment of the population that opposes genetic engineering," the Grocery Manufacturers Association is asking a federal court to prevent the state from implementing Act 120 until its lawsuit against Vermont "has run its course."

Act 120, which establishes labeling requirements for what it calls genetically engineered foods, was passed by the Legislature earlier this year and signed into law in May by Gov. Peter Shumlin.

According to the motion for an injunction, because GE varieties of corn and soybean account for 90 percent of those types of plantings in the United States, Act 120 will affect most of the grocery products sold in Vermont.

"Federal law does not require food labeling to also include plant labeling because there is no rational justification for such a regime," wrote attorneys for the GMA, the Snack Food Association, the International Dairy Association and the National Association of Manufacturers. "Act 120, however, is not concerned with rational justification. It caters to beliefs and biases that a government has no business endorsing."

Not only does Act 120 not serve any legitimate governmental interests, wrote the attorneys, it violates the First Amendment and intrudes upon federal labeling requirements, and is thus preempted by the Supremacy Clause.

In addition, they wrote, the members of the associations bringing suit against the state "will suffer irreparable injury" without an injunction because they have no way "to reliably distinguish ingredients derived from genetically engineered plant varieties from those that are not."

"The changes manufacturers would need to demand from their suppliers and initiate in their own facilities to segregate ingredients require money and time -- much more time than the Act's July 1, 2016, effective date allows," wrote the attorneys.

And then there are the costs related to building out Vermont-specific supply and distribution chains that do not currently exist.

"Plaintiff's members will not be able to recoup the cost of those efforts from the State if they prevail, nor could they easily return their business to the status quo ante."

Even though the plaintiff's plan to present evidence that food produced from genetically engineered crops is safe, "vehement opposition to genetic engineering persists," wrote the attorneys. Much of that opposition is based in philosophical or religiousbeliefs, concerns about large-scale agricultural operations, or biases against certain companies, maintain the attorneys.

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Noir Genetics - Black Ops II Game Clip
Game Clip.

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The Sims 4- Testing genetics by making sisters
Testing the genetics side of the Sims and made sisters. Not bad as I didn #39;t need the parents.

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Week 14-lecture 8-conservation genetics
Biol 252.

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Newswise The Genetics Society of America today announced new appointments to the editorial board of its flagship journal GENETICS. The recent additions complement the renewed focus of the peer-reviewed, peer-edited journal in the areas of genomics, human genetics, and methods, among other fields.

The newest members of the board bring cutting-edge expertise and reflect the changing, interdisciplinary landscape of our field, while exemplifying GENETICS reputation for rigorous scholarship, said Mark Johnston, Editor-in-Chief of GENETICS and Professor and Chair, Department of Biochemistry and Molecular Genetics, University of Colorado School of Medicine. Theyre already adding to the momentum of the journals transformation over recent years.

Two former Associate Editors have also taken on Senior Editor roles to lead the journals expanded coverage of methods and technology development:

New methods and technologies often drive important discoveries in genetics, and the journal welcomes papers that describe these new approaches, said Fields.

The new Statistical Genetics and Genomics section will handle articles describing statistical methods, which were formerly published in the Methods, Technology, and Resources section.

Statistics is an integral part of our field, so its only fitting that it has a dedicated section and its own team of Associate Editors, said Churchill.

The new Senior Editors are joined by several Associate Editor appointments this year:

New Editor Details: Hugo J. Bellen Baylor College of Medicine & Howard Hughes Medical Institute GENETICS Associate Editor, Developmental and Behavioral Genetics http://flypush.imgen.bcm.tmc.edu/lab/index.html

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Newswise The Genetics Society of America today announced new additions to the editorial board of its peer-reviewed, peer-edited journal G3: Genes|Genomes|Genetics. Since the journals launch in June 2011, its editorial board of academic experts has been instrumental in shaping G3 into an important forum for the publication of useful genetics findings and resources.

As the breadth of research published in the journal continues to grow, Editor-in-Chief Brenda Andrews, PhD, has appointed two new Deputy Editors-in-Chief, who will contribute to the oversight of key sections:

Also, Stephen Wright, PhD, University of Toronto, has been appointed as a new Senior Editor for Population and Evolutionary Genetics and Genomics and will spearhead the journals efforts to strengthen coverage of in this area.

The new Deputy Editors-in-Chief and Senior Editor are joined by several new Associate Editor appointments to the editorial board this year:

Genetics is a fast-paced field. The expanded editorial board will help us keep up with the growing volume and diversity of research being submitted to G3, said Brenda Andrews, G3: Genes|Genomes|Genetics Editor-in-Chief and Professor and Chair of the Banting & Best Department of Medical Research, University of Toronto.

G3 was created by the Genetics Society of America to meet the critical and growing need of the genetics community for rapid review and publication. The journal offers an opportunity to publish the puzzling finding, useful dataset, or highly focused research that may not have been submitted for publication due to a lack of perceived impact.

New Editor Details:

Eduard Akhunov Kansas State University G3 Associate Editor

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Newswise ST. LOUIS Patients who years ago breathed a sigh of relief because their genetic tests showed they were not at increased risk of developing cancer are not necessarily home free, said Suzanne Mahon, DNSc, genetic counselor for Saint Louis University Cancer Center.

There is this group of people who think they dont need to worry about getting cancer and believe they dont have a high family risk of getting cancer, but unfortunately do, said Mahon, a professor in internal medicine at Saint Louis University.

Mahon says her requests for genetic testing for breast cancer have more than tripled since 2013, when actress Angelina Jolie announced she had a double mastectomy because she was at genetic risk of developing breast cancer.

Older genetic screenings were for the BRCA 1 and 2 genes, which are linked to the development of breast, ovarian, prostate, melanoma, pancreatic and other cancers.

However a new generation of genetic tests can detect as many as 25 other, less common genes that show a familial predisposition to cancer.

Its important for people to understand what genetic test they were given and how complete they were, Mahon said.

Many people have tested negative for BRCA 1 or 2 and mistakenly think they do not have an increased hereditary risk for developing cancer. It is possible that newer testing, through next generation platforms, could identify a less common susceptibility gene and clarify their risk.

For those who had early, less complete genetic tests, catch up panels are available to screen for other genes that raise their risk of developing cancer. Most of these genes are associated with multiple cancers, Mahon said.

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3 Minute Thesis 2014 Winner - Harshavardini Padmanabhan - Interview
Gene therapy spray: A breath of fresh air Presenter: Harshavardini Padmanabhan Faculty of Health Sciences School of Paediatrics Thesis area: Aerosol Airway G...

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3 Minute Thesis 2014 Winner - Gene therapy spray: A breath of fresh air - Presentation
Presenter: Harshavardini Padmanabhan Faculty of Health Sciences School of Paediatrics Thesis area: Aerosol Airway Gene Transfer Technique for Clinical Use Su...

By: University of Adelaide

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Chatting with Kelly Brush Davisson!
Our first interview as a part of our #AthletesWeLove series! Team AbleThrive sat down with athlete Kelly Brush Davisson to chat with her about her life and athletic endeavors. Tune in to see...

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Regenerative Medicine TBI
Allen Brown, M.D., Associate Professor of PM R at Mayo Clinic discusses regenerative medicine and traumatic brain injury.

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ABC Ralph Bright harvests stem cells using the liposuction.

Serious questions have been raised about a stem cell doctor working in Western Sydney who charges $9,000 per procedure and uses methods that are untested by clinical trials.

An investigation by the ABC's 7.30 program has revealed that Dr Ralph Bright bought his liposuction-based technology from an American company.

The US company is now the subject of a multi-million dollar fraud action, which has revealed the cells being marketed as live were in fact dead.

Dr Bright, of Macquarie Stem Cells, is a former GP and self-taught cosmetic surgeon.

He has been working with stem cells for four years, treating more than 400 patients, including the late model Charlotte Dawson, cricketer Geoff Lawson and Olympic volleyballer Kerri Pottharst.

Dr Bright has licensed his methods to other practitioners around the country and because they use the patients' own cells he is not regulated by the Therapeutic Goods Administration (TGA).

Stem cells are often hailed as a miracle cure, but the nation's top stem cell scientists are warning that buyers should beware of these sorts of procedures, which are yet to be subjected to clinical trials.

Professor of Stem Cell Science at the University of Melbourne, Martin Pera, said almost all stem cell therapy was experimental.

"Actually, this whole science of cell therapy is relatively new and it's very, very important to understand that," he said.

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ABC Ralph Bright harvests stem cells using the liposuction.

Serious questions have been raised about a stem cell doctor working in Western Sydney who charges $9,000 per procedure and uses methods that are untested by clinical trials.

An investigation by the ABC's 7.30 program has revealed that Dr Ralph Bright bought his liposuction-based technology from an American company.

The US company is now the subject of a multi-million dollar fraud action, which has revealed the cells being marketed as live were in fact dead.

Dr Bright, of Macquarie Stem Cells, is a former GP and self-taught cosmetic surgeon.

He has been working with stem cells for four years, treating more than 400 patients, including the late model Charlotte Dawson, cricketer Geoff Lawson and Olympic volleyballer Kerri Pottharst.

Dr Bright has licensed his methods to other practitioners around the country and because they use the patients' own cells he is not regulated by the Therapeutic Goods Administration (TGA).

Stem cells are often hailed as a miracle cure, but the nation's top stem cell scientists are warning that buyers should beware of these sorts of procedures, which are yet to be subjected to clinical trials.

Professor of Stem Cell Science at the University of Melbourne, Martin Pera, said almost all stem cell therapy was experimental.

"Actually, this whole science of cell therapy is relatively new and it's very, very important to understand that," he said.

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UPDATE: After searching for more than two months for a bone marrow donor, Montreal leukemia patient Mai Duong has found a compatible cord blood donor. Its not the perfect solution, but doctors say its the next best thing.

It is not being released where the donor came from.

A woman has given birth to her child and has donated her babys umbilical cord to save another life, said Duong in a statement.

Thank you dear mommy, we cannot fathom the importance of your gesture. I am very moved and I profoundly thank you for what youve done.

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VANCOUVER Mai Duong, 34, only has six weeksleft to get a life-saving stem cell or bone marrow transplant and shes pleading with the Lower Mainlands Asian population tosave her.

The mother of one was born and raised in Montreal. Shes had good health for most of her life, until she was diagnosed with leukemia in January 2013, while pregnant with her second child. Doctors told her she had to terminate the pregnancy she was at 15 weeks and start chemotherapy immediately.

Duongwent into remission, but ten months later the cancer was back. And this time it was more aggressive and chemotherapy wouldnt work, she was told. Instead, she needed stem cells or a bone marrow transplant.

Even though Im on the international registry list for donors, I did not have a match for the bone marrow. I was devastated when they told me that, she toldGlobal News.

It turns out the problem of finding a match, and a perfect one at that, is more common among those of Asian descent. In 2012, 2-year-old Jeremy Kong of San Francisco was diagnosed with leukemia and couldnt find a match until he went public. After doing so, he found a nine out of tenbone marrow donor match and underwent a transplant, but died a year later. Experts say Vietnamese, Chinese, Japanese, Korean, Filipino and other South Asian populations are behind Caucasians when it comes to donating blood and organs.

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MISSISSAUGA Dorothy Vernon-Brown is on the search of her life.

When she was diagnosed with acute myaloid leukemia in August, 2013, the Mississauga mother said it was like a huge kick in the gut.

She underwent chemotherapy shortly after the diagnosis, which helped her into remission, but to survive, she has been told her only chance is a bone marrow transplant.

Bone marrow cells rescue patients from the lethal effect of chemotherapy, says Vernon-Browns oncologist, Dr. Mark Minden.

Since starting a relationship with Canadas stem cell and marrow network, One Match, Vernon-Browns search has come up empty.

Vernon-Brown says she is desperate: Recent tests show lower white blood cell counts in her blood an indication the cancer may be coming back.

But shes also worried about how few donors are from the black community. Vernon-Brown has been told her chance of finding a compatible donor are one in 10,000.

Genetics are key in finding potential stem cell and bone marrow donors says, One Match patient and transplant liaison, MaryLynn Pride said.

There is only a 25 per cent chance that even a sibling will be a match, says Pride. We have patients from all ethnic communities that are currently in need of a stem cell transplant.

Black Canadians makes up only 1 percent of all Canadians registered as potential donors, according to Pride.

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Stem cells broke into the public consciousness in the early 1990s, but progress has been slow. Photo: Bloomberg

Edgar Irastorza was just 31 when his heart stopped beating in October 2008.

A Miami property manager, break-dancer and former high school wrestler, Irastorza had recently gained weight as his wife's third pregnancy progressed. "I kind of got pregnant, too," he said.

During a workout one day, he felt short of breath and insisted that friends rush him to the hospital. Minutes later, his pulse flatlined.

He survived the heart attack, but the scar tissue that resulted cut his heart's pumping ability by a third. He couldn't pick up his children. He couldn't dance. He fell asleep every night wondering if he would wake up in the morning.

Desperation motivated Irastorza to volunteer for a highly unusual medical research trial: getting stem cells injected directly into his heart.

"I just trusted my doctors and the science behind it, and said, 'This is my only chance,'" he said recently.

Over the past five years, by studying stem cells in lab dishes, test animals and intrepid patients like Irastorza, researchers have brought the vague, grandiose promises of stem cell therapies closer to reality.

Stem cells broke into the public consciousness in the early 1990s, alluring for their potential to help the body beat back diseases of degeneration like Alzheimer's, and to grow new parts to treat conditions like spinal cord injuries.

Progress has been slow. The Michael J. Fox Foundation for Parkinson's Research, an early supporter of stem cell research, pulled its financial backing two years ago, saying that it preferred to invest in research that was closer to providing immediate help for Parkinson's disease patients.

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Masayo Takahashi is the first to implant tissue derived from induced pluripotent stem cells into a person.

Its awesome, its amazing, Im thrilled, Ive been waiting for this, says Jeanne Loring, a stem-cell biologist at the Scripps Research Institute in La Jolla, California. She is one of several researchers around the world to welcome the news that a Japanese woman with visual impairment had become the first person to receive a therapy derived from stem cells known as induced pluripotent stem (iPS) cells.

A lot rides on this trial. If the procedure proves safe, it could soften the stance of regulatory bodies in other nations towards human trials of iPS cells, and it could pave the way for treatments for other conditions, such as Parkinsons disease and diabetes. It could also cement Japan, recently plagued by a stem-cell scandal, as a frontrunner in iPS-cell research.

Pioneered in 2006 by Shinya Yamanaka, now director of the Center for iPS Cell Research and Applications at Kyoto University, iPS cells are created by inserting certain genes into the DNA of adult cells to reprogram the cells back to an embryonic-like state. The cells can then be turned into almost any tissue type, much as embryonic stem cells can. But because iPS cells can be derived from a patients own tissue, the hope is that they will dodge some of the controversial aspects and safety concerns of those derived from embryos.

In 2012, Yamanaka received a Nobel prize for his work, and the field has now matured, with teams across the world champing at the bit to test therapies based on iPS cells in people. Loring, for example, uses the cells to create dopamine-producing neurons as a potential therapy for Parkinsons disease, and says that she will start clinical trials as soon as the US Food and Drug Administration (FDA) gives the go-ahead.

Still, tissues made from iPS cells carry their own concerns, and that had stopped any country from approving them for a clinical trial. The bodys immune system could attack them, or they might contain some cells that are still in the pluripotent state and cause cancerous growths although Loring points out that this has not happened with human trials of therapies based on embryonic stem cells, for which the same concerns would apply.

In July 2013, however, Japans regulatory authorities gave the go-ahead for a team led by ophthalmologist Masayo Takahashi at the RIKEN Center for Developmental Biology (CDB) in Kobe to collect cells to be used in a clinical iPS-cell pilot study.

His team took skin cells from the first patient, a woman in her seventies who had retinal damage owing to a condition known as age-related macular degeneration. The researchers then reprogrammed the skin cells into iPS cells and coaxed the unspecialized cells into becoming retinal tissue. On 8September, Takahashi provided evidence that those cells were genetically stable and safe, a prerequisite for them to be transplanted into the eye. The procedure took place four days later, and RIKEN has reported that the patient experienced no serious side effects.

In this instance, the womans vision is unlikely to improve. However, researchers around the world are watching to see whether the cells stop the retina from deteriorating further and whether any side effects develop. Should the woman experience serious consequences, iPS-cell research could be set back years, much as gene therapy was in 1999 when a patient died in a trial that attempted to use a modified gene to correct a type of liver disease. That wakes me up at night, Loring admits.

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18 hours ago by Stuart Mason Dambrot The T4 capsid-derived specific exogenous DNA plus protein packaging and eukaryotic cell delivery scheme. (A) DNA encoding a 10-amino acid N-terminal CTS peptide fused to the phage P1 Cre allows synthesis of CTS-Cre and targeting of the enzyme into the early core-scaffold of the T4 procapsid in vivo. Procapsid assembly and maturation-specific viral protease stabilize the procapsid, remove most of the scaffolding core as peptides, and remove the CTS peptide from Cre. Mutations in the viral terminase block DNA packaging and allow a mature but DNA-empty large Cre-containing procapsid to be highly purified from viral-infected bacteria. (B) In vitro packaging into the mature capsid of plasmid DNA containing mCherry driven by a CMV promoter and two loxP sites flanking an SfiI restriction enzyme site that allow the linearization required for packaging. The DNA is packaged into the procapsid by the ATP-driven terminase motor protein (gp17) with high efficiency. (C) The packaged Cre enzyme recircularizes the packaged linear plasmid DNA between the two loxP sites. The DNA-containing capsid is taken up by eukaryotic cells, here without displaying a specific peptide target, or into eukaryotic cells specifically using Soc and Hoc displayed peptides that have high affinity for the RP1 and RP2 receptors, respectively. Credit: Liu JL, et al. (Published online before print August 26, 2014) Viral nanoparticle-encapsidated enzyme and restructured DNA for cell delivery and gene expression. Credit: Liu JL, et al. (2014) Viral nanoparticle-encapsidated enzyme and restructured DNA for cell delivery and gene expression. Proc Natl Acad Sci USA Published online before print August 26, 2014. doi:10.1073/pnas.1321940111

(Phys.org) By loading any specific protein and nucleic acid into an icosahedral phage T4 capsid-based nanoparticle, the resulting cell delivery vehicle's ligands can bind to the surface of specific target tissues to deliver the protein/DNA cargo. (Icosahedral viral nanoparticles are evolutionary protein shells assembled in a hierarchical order that results in a stable protein layer and an inner space for accommodating nucleic acids and proteins; a capsid is the protein shell of a virus.) The technique has drug- and gene-delivery applications in human diseases, diagnostic and cellular imaging, and other medical areas. Recently, scientists at US Naval Research Laboratory, Washington, DC and University of Maryland at Baltimore packaged T4 nanoparticles in vivo with active cyclic recombination, or Cre, recombinase (a genetic recombination enzyme used to manipulate genome structure and control gene expression) and in vitro with fluorescent mCherry (a fluorescent protein used as a marker when tagged to molecules and cell components) expression plasmid DNA, and delivered these nanoparticles into cancer cells: When released into cells in the presence of both DNA and protein, the recombinase enhances mCherry expression by circularization (that is, changing the packaged linear DNA into a circular loop). The researchers state that this efficient and specific packaging into capsids and the unpackaging of both DNA and protein with release of the enzymatically altered protein/DNA complexes from the nanoparticles into cells have potential in numerous downstream applications such as genetic and cancer therapeutics.

Dr. Jinny L. Liu discussed the paper that she, Prof. Lindsay W. Black and their co-authors published in Proceedings of the National Academy of Sciences USA. "Icosahedral viral nanoparticles are essentially 100 nm by 80 nm nanocontainers that allow exogenous genetic material to be packaged in vitro through nucleic acid machinery that generally only allows linear DNA/RNA to be packaged through a portal channel," Liu tells Phys.org. "However, in vitro protein packaging is generally impossible, because for most viral nanoparticles there is no protein packaging machinery comparable to nucleic acid packaging machinery." While protein may be chemically cross-linked to the capsid inner surface, this is expected to lead to protein denaturation and loss of enzymatic activity.

That being said, nature has evolved solutions to this protein packaging conundrum. During in vivo viral capsid assembly, Liu explains, some bacterial viruses, or bacteriophages, target proteins within the procapsids before the nucleic acid is packaged so as to eject the proteins with the nucleic acid, thereby facilitating infection in conjunction with the nucleic acid. (A procapsid, or prohead, is an immature viral capsid structure formed in the early stages of self-assembly of some bacteriophages. Production and assembly of stable proheads is an essential precursor to bacteriophage genome packaging.) Only a few phages have well-characterized in vivo protein packaging systems, and phage T4 is the best characterized. "Prof. Black's lab at UMB and my lab at NRL have demonstrated that not only can a specific foreign enzyme cyclic recombination (Cre) recombinase be packaged into the capsid in vivo, but also that it is active within the capsid." This activity was demonstrated by showing the religation (the rejoining of two DNA strands or other molecules by a phosphate ester linkage) of packaged linear DNA flanked with two Cre recombination sites.

The paper shows that the substantial space within a T4 nanocontainer accommodates the active Cre enzyme along with exogenous DNA. "For potential applications, T4 can package up to 50 kb exogenous linear DNA containing full-length desired genes along with recombinases, either Cre or -red proteins, for specific homologous recombination within the chromosome," Liu notes. (Homologous recombination is a type of genetic recombination in which nucleotide sequences are exchanged between two similar or identical molecules of DNA.) "We expect that the cas9 enzyme could be encapsidated in a comparable way and in fact, at least eight different proteins have been encapsidated in this manner. Through homologous recombination, our system can allow the corrected gene to replace the mutated gene in its original location within the chromosome or by precisely knocking out the overactive genes in stem cells." Liu points out that the T4 delivery vector is safer and better controlled than other viral delivery gene therapy, such as those delivering genes using infectious animal viral vectors to randomly insert the gene within the chromosome.

In their paper, the authors report that the T4 capsid NP gene expression and protein delivery system may be complementary to or used in conjunction with gene therapy based on RNA Cas and taran nuclease. (Cas genes code for proteins related to DNA loci containing short repetitions of base sequences known as Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPRs.) "The T4 nanoparticle expression system can easily complement Cas9 and taran nuclease-based recombination by packaging the linear cas9, target-sgRNA plasmid DNA, and Cre recombinase or even ligase, an enzyme that facilitates the joining of DNA strands and deliver the resulting T4 nanoparticles into the recipient eukaryotic cells with high specificity employing SOC and HOC," Liu tells Phys.org. (SOC and HOC are dispensable T4 capsid proteins.) "By displaying the targeting ligands (binding molecules) onto the surface, the T4 capsid gene expression and protein system will be able to efficiently deliver the Cas9 and sgRNA plasmids together into the desired recipient cells. Relevant enzymatically-active proteins Cas9, lambda exonuclease, lambda beta protein and others can be delivered directly at the same time from the T4 nanoparticle."

Liu adds that her lab has also been studying cell imaging and drug/gene delivery to eukaryotic cells using T4 tailless nanoparticles, which the researchers demonstrated can enter the eukaryotic cells without causing cell death.

A specific example of potential downstream drug and gene therapeutic applications resulting from the new approach is delivery of the toxic protein and linear plasmid that produces neutralizing peptides or antibodies into targeted cancer cells displaying specific cancer markers using high affinity SOC + HOC marker binding proteins on the surface of the capsids, while another example is to use the system for HIV gene therapy.

Liu adds that there are several pathways to use this system for gene therapy:

In addition to diagnostic and cellular imaging, the T4 nanoparticle gene-protein system can deliver repaired genes to correct human genetic diseases for example, reversing adenosine deaminase (ADA) deficiency by introducing the protein-DNA complex to express ADA in stem cells. Other broad areas of research impacted by gene therapy technologies, such as genetic defects, cancer, neurological diseases in adults, and aging itself, may also benefit from this study.

Link:
Nanocontainers for nanocargo: Delivering genes and proteins for cellular imaging, genetic medicine and cancer therapy

Recommendation and review posted by Bethany Smith

TORONTO Twenty-five years ago this month, the medical world was turned on its ear with the isolation of the gene that causes cystic fibrosis, a devastating inherited disease that usually killed children by their late teens.

At the helm of the research was Lap-Chee Tsui, who led the team at Torontos Hospital for Sick Children that made the seminal discovery in collaboration with scientists at the University of Michigan.

The science of human genetics was still in its infancy at that time. Pinpointing the mutated CFTR gene came about through painstaking mapping of bits of DNA to locate the root of CF symptoms thick, sticky mucus that clogs the lungs and gums up the gastrointestinal tract, requiring patients to take scores of digestive enzymes a day so they could digest food.

The cystic fibrosis defect is really a very subtle defect, Tsui (pronounced Choy), 63, said Monday during an event at Sick Kids to mark the 1989 discovery. It didnt kill the patients (right away), but the problems accumulated slowly, and at the end the patients succumbed to infection.

Using the same analogy as he used in 1989 to explain CFTRs location on chromosome 7, Tsui said researchers first narrowed it down to somewhere between Halifax and Vancouver, then further pinpointed it in Toronto, and finally zeroed in to a certain street and then the actual house that represented the defective gene.

In the ensuing years, researchers have determined there is not only one mutation in the CF gene, but about 1,900 different defects that cause greater or lesser severity of symptoms in individual patients a scientific process Tsui likened to going into the house and turning on all the lights and taps to see which ones are faulty.

The celebrated geneticist, who left Toronto 12 years ago to become vice-chancellor and president of the University of Hong Kong, from which he just retired, called progress in understanding and treating CF since the gene was isolated very exciting.

Within two years of that discovery, other Sick Kids researchers had determined that a protein that keeps epithelial cells lining the lungs, airways and digestive system nice and moist was faulty, causing the buildup of mucus that clogs the lungs and disables the digestive system.

I think the expectation when the gene was first discovered was that it would be easy to fix because the disease was caused by a single gene, and if you replaced that gene through gene therapy, then you would be able to completely reverse the consequences of the disease, said senior scientist Christine Bear, who led that team.

And it may be that gene therapy will be part of that future therapy in CF, but right now we havent developed safe ways to do that.

Originally posted here:
Canadian researchers mark 25 years since CF gene found

Recommendation and review posted by Bethany Smith

Scientists could soon better predict a man's risk of getting prostate cancer after a worldwide team of researchers carried out the largest-ever analysis of the cancer's genetic biomarkers, reported in Nature Genetics today.

QUT Institute of Health and Biomedical Innovation's Dr Jyotsna Batra and Distinguished Professor Judith Clements, who led the Australian researchers in the large consortia of research hubs around the world, said the teams analysed more than 10 million genetic markers in 80,000 men.

"It's the largest analysis of genetic biomarkers ever done. We found another 23 new prostate cancer risk loci (sites) on the genome in addition to the 76 identified previously," Dr Batra said.

"We now have 100 genetic regions and no other cancer has had this many loci identified to be associated with it. What we are looking for is the combination effect of how these loci work together and how much they can explain the heritability of prostate cancer.

"The indications are that these genetic variants explain 33 per cent of the familial risk of the disease.

"These are low-risk gene variants but what we have learnt is you can't rely on just one gene to predict risk. You have to look at the total of the 100.

"The top one per cent of men with these variants have a 5.7-fold relative risk compared with the population average." Dr Batra said that, in addition to family history, incorporating information regarding carrier status of these 100 risk variants could be valuable in defining risk levels in targeted screening and prevention programs for prostate cancer.

She said the multi-ethnic analysis of 80,000 individuals with prostate cancer found some risk variants were more common in different ethnic populations.

"The aggressive form is prevalent in Africa and we found some risk genes specific to African populations," she said.

"Of the 23 new variants we found 15 were in men of European ancestry and seven in the multi-ethnic analyses."

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New gene research helps pinpoint prostate cancer risk

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A DNA sequencer the size of a cell phone could change where, and how, gene research occurs.

The DNA sequencer built by Oxford Nanopore draws power from a computers USB port.

One day in 1989, biophysicist David Deamer pulled his car off Californias Interstate 5 to hurriedly scribble down an idea. In a mental flash, he had pictured a strand of DNA threading its way through a microscopic pore. Grabbing a pen and a yellow pad, he sketched out a radical new way to study the molecule of life.

Twenty-five years later, his idea is now being commercialized as a gene sequencing machine thats no larger than a smartphone, and whose effects might eventually be similarly transformative.

Early versions of the instrument, called the MinION, have been reaching scientific labs over the past few months after long delays (see 10 Breakthrough Technologies 2012: Nanopore Sequencing). Its built by a U.K. company, Oxford Nanopore, that has raised $292 million and spent 10 years developing Deamers idea into a DNA sequencer unlike any other now available. It is four inches long and gets its power from a USB port on a computer. Unlike other commercial sequencing machines, which can be the size of a refrigerator and require jugs of pricey chemicals, this one measures DNA directly as the molecule is drawn through a tiny pore suspended in a membrane. Changes in electrical current are used to read off the chain of genetic letters, A, G, C, and T.

Scientists with early access to prototypes of the first commercial nanopore sequencer say its glitchy and error-prone but may still be the way scientists study DNA in the future. After testing it, Mick Watson, a bioinformatics researcher at the Roslin Institute, in Scotland, says nanopore sequencing is a disruptive technology that could, potentially, dominate the sequencing market for years to come.

Although researchers say the device is still desperately inaccurate, it can already carry out some unheard-of scientific feats. And then theres its size. A sequencer this small might one day let police read off a genome from a spot of blood at a crime scene, or permit doctors to pinpoint viruses in the midst of an epidemic. One scientist this month tweeted a picture of the sequencer on his dining room table, decoding DNA.

The MinION is the result of some very high-stakes R&D by Oxford, a 200-person company thats long has had its eye on the expanding market for high-speed DNA sequencers. Cracking that market wont be easy. About 90 percent of DNA data is produced on sequencing machines from a single company, Illumina of San Diego (see 50 Smartest Companies: Illumina). Its sequencers are so good that most of its competitors have ended up in Chapter 11 or retreated in ignominy.

David Deamer made this sketch in 1989 when the idea for nanopore sequencing came to him.

But now some big companies are betting that nanopores could be the technology to break Illuminas lucrative monopoly. Roche, which made a failed attempt to acquire Illumina in 2012, this year spent $125 million to buy Genia Technologies, a small nanopore company based in California, and invested in another, Stratos Genomics. Hitachi is also working on nanopore technology, as are startups like Electronic Biosciences.

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Radical New DNA Sequencer Finally Gets into Researchers Hands

Recommendation and review posted by Bethany Smith

MONDAY, Sept. 15, 2014 (HealthDay News) -- An international team of scientists has identified 23 new genetic variants linked to a greater risk for prostate cancer.

Although more research is needed, the researchers said their findings, which bring the total number of common gene variants associated with prostate cancer to 100, could help doctors diagnose the disease earlier and could lead to the development of new treatments.

"Our study tells us more about the effect of the genetic hand that men are dealt on their risk of prostate cancer. We know that there are a few major genes that are rare and significantly affect prostate cancer risk, but what we are now learning is that there are many other common genetic variants that individually have only a small effect on risk, but collectively can be very important," Ros Eeles, a professor of oncogenetics at The Institute of Cancer Research in London, explained in an institute news release.

Eeles added, "To use the playing cards analogy again, sometimes multiple low cards can combine to form a high-risk score. We can now explain a third of the inherited risk of prostate cancer, and will shortly be conducting a clinical trial to find out whether testing for genetic variants in men can successfully pick up the disease early, and help direct targeted interventions for patients."

The team of scientists, from The Institute of Cancer Research, the University of Cambridge and the University of Southern California, examined the genetic information of over 87,000 men of European, African, Japanese, and Latino heritage. The investigators compiled genetic population studies of 43,303 men with prostate cancer and 43,737 "controls" (without prostate cancer) to increase the likelihood they would identify new variants.

The researchers found 16 new genetic indicators associated with prostate cancer risk in European men, including a variant linked with increased risk of early onset disease. They also identified seven genetic indicators in men of mixed heritage. As a result, the study's authors said they could now explain 33 percent of the inherited origins of prostate cancer in European men.

Among the European men assessed for the 100 common variants, the 10 percent at greatest risk are nearly three times more likely to develop prostate cancer, according to the study published Sept. 14 in Nature Genetics. The researchers also found the top 1 percent are nearly six times more likely to develop the disease.

Looking ahead, the researchers plan to investigate if these new findings could improve on prostate cancer screening tests. In particular, they are examining whether genetic testing could help diagnose more men at risk for aggressive forms of prostate cancer that need immediate treatment.

"The results of this study could take us a step closer to targeted screening by allowing us to identify those most at risk of the disease based on the genes that they possess," Dr. Matthew Hobbs, deputy director of research at Prostate Cancer UK, said in the news release.

"However, this is not the end of the story, and the challenge now lies in translating this knowledge into a reliable test that can be used on a large scale through the NHS [National Health Service] to find those men at highest risk," Hobbs added.

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New Gene Variants for Prostate Cancer Identified

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