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TOKYO: Shinya Yamanaka, fresh from the Nobel Prize for medicine, states that science and ethics must go hand in hand. Interviewed by the Mainichi Shimbun after the award, he said: "I would like to invite ethical experts as teachers at my laboratory and work to guide iPS [induced pluripotent stem] cell research from that direction as well. The work of a scientific researcher is just one part of the equation. "

Yamanaka, 50, found that adult cells can be transformed into cells in their infancy, stem cells (iPS), which are, so to speak, the raw material for the reconstruction of tissue irreparably damaged by disease. For regenerative medicine the implications of Yamanaka's discovery are obvious. Adult skin cells can for example be reprogrammed and transformed into any other cell that is desired: from the skin to the brain, from the skin to the heart, from the skin to elements that produce insulin.

"Their discovery - says the statement of the jury that awarded him the Nobel Prize on October 8 - has revolutionized our understanding of how cells and organisms develop. Through the programming of human cells, scientists have created new opportunities for the study of diseases and development of methods for the diagnosis and therapy ".

These "opportunities" are not only "scientific", but also "ethical". Much of the scientific research and global investment is in fact launched to design and produce stem cells from embryos, arriving at the point of manipulating and destroying them, facing scientists with enormous ethical problems.

" Ethics are really difficult - Yamanaka explainsto Mainichi - In the United States I began work on mouse experiments, and when I returned to Japan I learned that human embryonic stem cells had been created. I was happy that they would contribute to medical science, but I faced an ethical issue. I started iPS cell research as a way to do good things as a researcher, and I wanted to do what I could to expand the merits of embryonic stem cells. If we make sperm or eggs from iPS cells, however, it leads to the creation of new life, so the work I did on iPS cells led to an ethical problem. If we don't prepare debates for ethical problems in advance, technology will proceed ahead faster than we think.. "

The "ethical question" Yamanaka pushed to find a way to "not keep destroying embryos for our research."

Speaking with his co-workers at the University of Kyoto, immediately after receiving the award, Yamanaka showed dedication and modesty.

"Now - he said - I strongly feel a sense of gratitude and responsibility" gratitude for family and friends who have supported him in a demanding journey of discovery that lasted decades; responsibility for a discovery that gives hope to millions of patients. Now iPS cells can grow into any tissue of the human body allowing regeneration of parts so far irretrievably lost due to illness.

His modesty also led him to warn against excessive hopes. To a journalist who asked him for a message to patients and young researchers awaiting the results of his research heresponded: "The iPS cells are also known as versatile cells, and the technology may be giving the false impression to patients that they could be cured any day now. It will still take five or 10 years of research before the technology is feasible. There are over 200 researchers at my laboratory, and I want patients to not give up hope"

"Dozens of times - he continued - I tried to get some results and I have often failed in the experiments .... Many times I was tempted to give up or cry. Without the support of my family, I could not have continued this search. From now on I will be facing the moment of truth. I would like to return to my laboratory as quickly as possible. "

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Stem-cell transplant claims debunked

Hisashi Moriguchi presented his work at the New York Stem Cell Foundation meeting this week.

AP/Press Association

From the beginning, it seemed too good to be true. Days after Kyoto University biologist Shinya Yamanaka won a Nobel prize for his 2006 discovery of induced pluripotent stem (iPS) cells (see 'Cell rewind wins medicine Nobel'), Hisashi Moriguchi a visiting researcher at the University of Tokyo claimed to have modified that technology to treat a person with terminal heart failure. Eight months after surgical treatment in February, said a front-page splash in the Japanese newspaper Yomiuri Shimbun yesterday, the patient was healthy.

But after being alerted to the story by Nature, Harvard Medical School and Massachusetts General Hospital (MGH), where Moriguchi claimed to have done the work, denied that the procedure had taken place. No clinical trials related to Dr Moriguchi's work have been approved by institutional review boards at either Harvard University or MGH, wrote David Cameron, a spokesman for Harvard Medical School in Boston, Massachusetts. The work he is reporting was not done at MGH, says Ryan Donovan, a public-affairs official at MGH, also in Boston.

A video clip posted online by the Nippon News Network and subsequently removed showed Moriguchi presenting his research at the New York Stem Cell Foundation meeting this week.

If true, Moriguchis feat would have catapulted iPS cells into use in a wide range of clinical situations, years ahead of most specialists' predictions. I hope this therapy is realized in Japan as soon as possible, the head of a Tokyo-based organization devoted to helping children with heart problems told Yomiuri Shimbun.

But there were reasons to be suspicious. Moriguchi said he had invented a method to reprogram cells using just two chemicals: microRNA-145 inhibitor and TGF- ligand1. But Hiromitsu Nakauchi, a stem-cell researcher at the University of Tokyo, says that he has never heard of success with that method. He adds that he had also never heard of Moriguchi before this week.

Moriguchi also said that the cells could be differentiated into cardiac cells using a 'supercooling' method that he had invented. Thats another weird thing, says Nakauchi.

The article in which Moriguchi presented his two-chemical method, published in a book1 describing advances in stem-cell research, includes paragraphs copied almost verbatim from other papers. The section headed 2.3 Western blotting, for example, is identical to a passage from a 2007 paper by Yamanaka2. Section 2.1.1, in which Moriguchi describes human liver biopsies, matches the number of patients and timing of specimen extractions described in an earlier article3, although the name of the institution has been changed.

When contacted by Nature, Moriguchi stood by his publication. We are all doing similar things so it makes sense that wed use similar words, he says. He did admit to using other papers as reference.

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Regenevéda Opens Flagship Stem Cell Therapy Clinic in Beverly Hills

Regenevda recently opened its brand new flagship facility in Beverly Hills, specializing in cutting edge anti-aging treatments such as Stem Cell Therapy.

Beverly Hills, CA (PRWEB) October 12, 2012

Dr. Thom Lobe is an internationally respected surgeon and has been in practice for over 30 years. Consistently pioneering advances in medicine, Dr. Lobe was one of the first doctors to ever separate conjoined twins. Consistently working to help make advances in medicine, Dr. Lobe also has over 200 publications to his credit.

Overseeing the business aspect of Regenevda is Lindsey Combs. She is responsible for sales, staff, accounting, facility management, and business development. A graduate of the University of California, Los Angeles, Ms. Combs has been working in the anti-aging field for over 10 years and has been a California Licensed Esthetician since 2003.

Being one of the very few physicians in the country to hold the most advanced board certification (FAARM), Dr. Lobe is able to offer Stem Cell Therapy at the Regenevda clinic. Inside each persons own body, there are special cells in nearly every organ and tissue that have the ability to help heal damage. These special cells are called Stem Cells and this therapy works by harvesting these cells from a persons own blood, bone marrow, or fat and can help with different conditions. Some examples of procedures that use Stem Cell Therapy are: Stem Cell Facelifts, Stem Cell Breast Augmentation, and Stem Cell Joint Therapy. Stem Cell treatments are safe, non-invasive, and are done under local anesthesia.

Intravenous Nutrition Therapy (or IV Vitamin Therapy) is another anti-aging and rejuvenation treatment that can also help patients prevent migraines, lose weight, fight chronic infections like hepatitis, candida, lyme disease, as well as fight acute infections like the flu and mono. IV Therapy works by using intravenous solutions to deliver vitamins and minerals directly to the body cells. This bypasses the digestive system and provides a more direct method of delivery, which ensures that all of the nutrients required are delivered, allowing the patient to feel an improvement in condition almost immediately.

Human Growth Hormone (HGH) Therapy is another advanced treatment offered at Regenevda. HGH is secreted by the Pituitary gland and fuels cell growth and reproduction. This production peaks at adolescence. Over time, due to the effect of aging, the production of HGH slows down dramatically. As production declines, it makes it more difficult for the body to recover from physical and mental exertion. HGH Therapy acts as a supplement for HGH deficient adults to lessen body fat, boost lipid lineament, improve memory, promote bone density, as well as decrease risk factors that involve cardio-vascular conditions. If used at the onset of the decrease in HGH production, HGH Therapy can help curtail early aging and even be used as preventive measure against osteoporosis. A complete analysis of the patients sex hormones, evaluation of glucose regulation and functions of the adrenal gland, thyroid gland, and pancreas are performed before the treatment is administered for optimal results.

Combining decades of medical experience with the most cutting edge advances in medical technology, the Regenevda clinic looks to pave the way for the future of anti-aging treatments. The Regenevda Beverly Hills Institute of Cellular Therapy is located at 50 North La Cienega Boulevard. For any inquiries, they can be reached at 855-734-3638, or visit http://www.regeneveda.com.

About Regenevda :

Regeneveda, home of The Beverly Hills Institute of Cellular Therapy, provides state-of-the-art Stem Cell Therapy. Stem Cell Therapy is an effective treatment for chronic conditions such as Arthritis, Diabetes, Chronic Sports Injuries, and Chronic Pain, but is also revolutionizing anti-aging treatments such as Breast Enhancement, Erectile Dysfunction, and Facial Aging.

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Regenevéda Opens Flagship Stem Cell Therapy Clinic in Beverly Hills

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Global Autologous Cell Therapy Market worth $2.2 Billion by 2017

DALLAS, October 12, 2012 /PRNewswire/ --

The report "Autologous Cell Therapy Market(2012 - 2017)", published by MarketsandMarkets (http://www.marketsandmarkets.com), would be the first global and exclusive report on ACT market. It also gives clear information about the complete industry, approved products and potential market size; it also identifies driving and restraining factors for the global ACT market with analysis of trends, opportunities and challenges. The market is segmented and revenue is forecasted on the basis of major regions such as USA, Europe and Rest of the World (ROW). Further, market is segmented and revenues are forecasted on the basis of potential application areas of ACT.

Browse ACT market research data tables/figures spread through 111 slides and in-depth TOC on"Autologous Cell Therapy (ACT) Market (2012 - 2017)". http://www.marketsandmarkets.com/Market-Reports/autologous-cell-therapy-market-837.html Early buyers will receive 10% customization on reports.

There is a wide market potential and favorable landscape for adoption across many geographical locations of the world. During the forecast period, these technologies are expected to revolutionize the area of bio-pharma and personalized medicine. High incidence and lack of effective treatment for several diseases will drive the ACT technology in developed and developing nations.

Investment activities, for past five years are actively held in research and developments, attracting interests of cell therapy industry firms, medical centers and academic institutions. ACT potential can be demonstrated by mergers, collaborations, acquisitions and partnerships that happened actively between the ACT technology developing companies in past three years. Development of sophisticated automation devices for cell expansion and culture process for use in the treatment is one of the emerging trends of ACT market.

The global ACT market is valued around $650 million by 2011 with a CAGR of 21%. Several products and technologies of ACT are in pipeline which is expected to hit the market during the forecast period, which will result in increased growth rate.

About MarketsandMarkets

MarketsandMarkets is a global market research and consulting company based in the U.S. We publish strategically analyzed market research reports and serve as a business intelligence partner to Fortune 500 companies across the world. MarketsandMarkets also provides multi-client reports, company profiles, databases, and custom research services.

MarketsandMarkets covers thirteen industry verticals; including advanced materials, automotives and transportation, banking and financial services, biotechnology, chemicals, consumer goods, energy and power, food and beverages, industrial automation, medical devices, pharmaceuticals [ http://www.marketsandmarkets.com/pharmaceutical-market-research-3.html ], semiconductor and electronics, and Telecommunications and IT.

We at MarketsandMarkets are inspired to help our clients grow by providing apt business insight with our huge market intelligence repository. To know more about us and our reports, please visit our website http://www.marketsandmarkets.com.

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Global Autologous Cell Therapy Market worth $2.2 Billion by 2017

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Research and Markets: GTx's Capesaris – A Novel Medical Castration Method

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/vgdk75/gtxs_capesaris) has announced the addition of the "GTx's Capesaris - A novel medical castration method" report to their offering.

Capesaris is an oral selective estrogen receptor modulator (SERM) that GTx is developing as a first-line androgen deprivation therapy (ADT) in advanced prostate cancer. By utilizing this approach, GTx hopes that use will be devoid of many of the adverse effects of historical medical castration products such as diethylstilbestrol (DES, an estrogen replacement therapy), and the luteinizing hormone receptor hormone (LHRH) agonists that form the current standard of care. This report explores the validity of that premise, the probability of phase III success, the regulatory path to approval, and the market into which it will launch, if successful.

Key Topics Covered:

INTRODUCTION AND BACKGROUND

1. Prostate cancer

- Epidemiology

- Clinical presentation, diagnosis, and staging

- Current treatments

2. GTX-758 (CAPESARIS)

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Research and Markets: GTx's Capesaris - A Novel Medical Castration Method

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Single Gene Variant in Donors May Affect Survival of Transplanted Kidneys

Variant linked to increased risk for long-term organ failure

Highlights In the largest study of its kind, a variant within the multidrug resistance 1 (MDR-1) gene in kidney transplant donors was linked to a 69% increased risk for long-term failure of transplanted organs. This variant affects the expression of the protein that the MDR-1 gene encodes, which pumps drugs out of cells. (Immunosuppressant drugs are critical for preventing organ rejection but are also toxic to the kidneys.)

Newswise Washington, DC (October 11, 2012) A single genetic variant in kidney donors cells may help determine whether their transplanted organs will survive long term, according to a study appearing in an upcoming issue of the Journal of the American Society of Nephrology (JASN). The findings provide new information that might be used to improve transplant longevity by revealing that the genetic make-up of kidney transplant donors affects the survival of transplanted organs.

A transplant recipient must take lifelong immunosuppressive drugs to prevent rejection of the new organ, but these drugs can have serious side effects, including kidney damage. So, ironically, the very drugs needed to prevent kidney rejection can also be toxic to the kidneys. Research suggests that how well certain proteins pump these drugs out of kidney cells may influence the drugs kidney toxicity.

Richard Borrows, MB (Queen Elizabeth Hospital Birmingham, in the UK) and his colleagues looked to see if variants in the genes that encode such pumps might influence the health of transplanted kidneys. They investigated the links between donor and recipient gene variants with kidney outcome among 811 immunosuppressant-treated kidney transplant recipients.

Among the major findings: One particular variant within the multidrug resistance 1 (MDR-1) gene in donors was linked to a 69% increased risk for long-term failure of transplanted organs. The researchers validated the link in another 3,660 donors, making this the largest study of its kind. This variant affects the expression of the protein that the MDR-1 gene encodes, the drug transporter P-glycoprotein. No other genetic variants in donors or recipients were linked with organ survival or failure.

The study of donor, as opposed to recipient, gene variation is relatively uncommon in the field of transplantation, and it certainly warrants more attention, said Dr. Borrows. He added that a single genetic variant probably has limited effect on its own, but when combined, multiple genetic variants may play an important role in transplant longevity.

Study co-authors include Jason Moore, MBBS, Amy Jayne McKnight, PhD, Bernd Dhler, PhD, Matthew Simmonds, PhD, Aisling Courtney, PhD, Oliver Brand, PhD, David Briggs, PhD, Simon Ball, PhD, Paul Cockwell, PhD, Christopher Patterson, PhD, Alexander Maxwell, PhD, Stephen Gough, PhD, and Gerhard Opelz, PhD.

Disclosures: The authors reported no financial disclosures.

The article, entitled Donor ABCB1 Variant Associates with Increased Risk for Kidney Allograft Failure, will appear online at http://jasn.asnjournals.org/ on October 11, 2012, doi: 10.1681/ASN.2012030260.

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Single Gene Variant in Donors May Affect Survival of Transplanted Kidneys

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Single gene variant may affect transplanted kidney survival

When a patient in need of a kidney transplant finally receives their new organ, their health problems may not necessarily be over. For some kidney recipients, their bodies may ultimately reject the foreign kidney leading to the organs removal and another long wait for a new donor.

However, new research out of the Queen Elizabeth Hospital Birmingham in the U.K. may help prevent this kind of painful rejection in the future. Scientists have discovered a single gene variant in kidney transplant donors that may predict whether or not the transplanted kidney will survive in the recipient.

The gene- the multidrug resistance 1 (MDR-1) gene was originally identified by the researcher of having a potential impact on kidney rejection and survival, because the protein the gene encodes helps to pump drugs out of cells.

In order for a transplanted kidney to successfully adapt and adhere to a new bodys system, the kidney recipient must take a number of immunosuppressive drugs to prevent rejection often including a class of drugs called calcineurin inhibitors (CNIs). Unfortunately, these kinds of medications can also come with serious side effects.

We wanted to look at a link between the genotype of the donor and the risk of transplant failure to CNI toxicity, Dr. Richard Borrows, in the department of nephrology and kidney transplantation at University Hospital Birmingham and the studys lead author, told FoxNews.com. Although they have revolutionized kidney transplantation, they are inherently toxic to the transplanted kidney. Theres a train of thought that that toxicity in turn leads to transplant failure.

Borrows and his team examined three different cohorts of pairs of kidney recipients and their donors. In the first group of 811 individuals, they looked for 52 gene variants in both the donors and the recipients. Only one gene appeared to be associated with kidney failure a particular variant of MDR-1.

Overall, the variant was linked with a 69 percent increased risk for long-term transplant failure. Two additional groups of a combined 3,660 donors confirmed the results.

The MDR-1 gene variant affects the expression of the protein that the gene encodes the drug transporter P-glycoprotein. Donor kidney cells with this kind of variant ultimately have more P-glycoprotein. Originally, the researchers believed that less P-glycoprotein would increase CNI toxicity and thus increase the chances of organ failure but instead the opposite was found to be true.

Borrows and his team have a few ideas as to why the increase of P-glycoprotein would potentially lead to kidney transplant failure, but did not test them.

The presence of P-glycoprotein seems to promote cellular damage by cholesterol and cholesterol esters, Borrows said. Also, in animal models, the presence of P-glycoprotein is associated with an increased likelihood of kidney damage when blood supply to the kidney is interrupted. So on one hand, this protein seems to be involved in the transport of CNI inhibitors, and on the other hand it seems to be involved in many other biological processes as well. So we need to tease each of these out.

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Single gene variant may affect transplanted kidney survival

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DKFZ and Genetic Immunity Sign Collaboration Agreement to Develop HPV Therapeutic Vaccine

HEIDELBERG, GERMANY and BUDAPEST, HUNGARY--(Marketwire - Oct 12, 2012) - Genetic Immunity ( OTCBB : PWRV ), a leader in immunotherapy technology product development, and DKFZ (German Cancer Research Center, Heidelberg, Germany) signed a collaborative agreement to develop a DNA-based vaccine for the treatment of Human Papilloma Virus (HPV) infection that causes cervical cancer and other cancers of the anus, penis, vulva, vagina, and oropharynx. Present HPV vaccines (Cervarix, Gardasil) have no therapeutic effect on HPV-related diseases, so they will not treat existing diseases or conditions caused by HPV.

The Division of Genome Modifications and Carcinogenesis led by Prof. Dr. Lutz Gissmann will initiate a preclinical research program to evaluate the therapeutic efficacy of the DKFZ's HPV-specific plasmid DNA using Genetic Immunity's nanomedicine formulation and Langerhans cell-targeting administration technologies. DKFZ is a world leading research center in tumor virology. Harald zur Hausen was awarded the Nobel Medicine Prize for his work on HPV-caused cancer of the cervix. Zur Hausen, former Scientific Director of the German Cancer Research Center, is recognized for finding that cervical cancer is caused by viral infections. His research made it possible to develop a vaccine against one of the most frequent cancers in women. Zur Hausen shared the Nobel Prize for Medicine with Franoise Barr-Sinoussi and Luc Montagnier for discovering HIV, the virus that causes AIDS.

"There is a huge unmet medical need for such cancer vaccine, because vaccines we have developed earlier do not provide protection against cancer when used for treatment of existing conditions caused by HPV. Our goal is to provide protection against cancer for patients after the onset of sexual activity, after they might be exposed to HPV," said Dr. Julianna Lisziewicz, CEO of Genetic Immunity.

Genetic Immunity has successfully tested in clinical trials DermaVir, a candidate immunotherapy for the cure of HIV. This new collaboration is using the clinically proven technology expanding the pipeline to another deadly viral disease that causes cancer. The partners will test whether HPV-specific memory T cells induced by Genetic Immunity's nanomedicine products could protect against cancer after infection has occurred.

"We found that Genetic Immunity technology is unique to target the vaccine DNA into the nucleus of the Langerhans cells. We believe that it will provide a breakthrough in cancer immunotherapy. We pioneered HPV prophylactic vaccines with new innovations and we would like to expand this tradition to therapeutic setting," said Dr. Prof. Gissmann of DKFZ.

Genetic Immunity is a wholly owned subsidiary of Power of the Dream Ventures, Inc. ( OTCBB : PWRV ).

About Genetic Immunity

Genetic Immunity, part of Power of the Dream Ventures, Inc. (PWRV), is a clinical stage technology company committed to discovering, developing, manufacturing and commercializing a new class of immunotherapeutic biologic drugs for the treatment of viral infections, cancer and allergies. Our Langerhans cell-targeting nanomedicines are exceptional in both safety and immune modulating activity boosting specific Th1-type central memory T cells. These are essential to eliminate infected cells or cancerous cells, and balance the immune reactivity in response to allergens.

In 1988 Drs. Lisziewicz and Lori founded Genetic Immunity in the US after they described the 1st patient whose immune system was boosted to control HIV after treatment interruption (Lisziewicz et al. New England Journal of Medicine 1999) that lead to the invention of DermaVir. The Company's innovative technology team directed by Dr. Lisziewicz, a champion of immune boosting therapies, is now headquartered in Budapest, Hungary. She has been invited into the Scientific Advisory Board of the HIV Cure Initiative led by Francoise Barre-Sinoussi Nobel Prize Laureate for her HIV research in 2009. For more information please visit http://www.geneticimmunity.com

About DKFZ

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DKFZ and Genetic Immunity Sign Collaboration Agreement to Develop HPV Therapeutic Vaccine

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New gene test flags risk of serious complications in sarcoidosis

ScienceDaily (Oct. 11, 2012) Researchers at the University of Illinois Hospital & Health Sciences System have identified a genetic signature that distinguishes patients with complicated sarcoidosis, an inflammatory lung disease that can be fatal, from patients with a more benign form of the disease. The gene signature could become the basis for a simple blood test.

Their findings are reported online in the journal PLOS ONE.

In sarcoidosis, tiny clumps of abnormal tissue form in organs of the body. These clusters of immune cells, called granulomas, cause inflammation. Sarcoidosis can occur in the lymph nodes, liver, eyes, skin or other tissues, but almost always also in the lungs. The cause of the disease is unknown. African Americans are at higher risk for the disease and for more severe cases.

"One of the perplexing aspects of this disease is that two thirds of the people who get sarcoidosis get better with only minimal therapy," says Dr. Joe G.N. "Skip" Garcia, vice president for health affairs at the University of Illinois and principle investigator on the study.

But one third of patients go on to develop complicated sarcoidosis -- neurologic sarcoidosis, cardiac sarcoidosis and progressive lung disease, Garcia said. Complicated sarcoidosis can leave patients with lung damage, and in a small percentage of cases the disease can be fatal.

The challenge, Garcia says, is that there is no difference in the clinical presentation between patients with simple sarcoidosis and those who will go on to develop more serious disease.

The researchers took blood from patients with simple and complicated sarcoidosis as well as patients without the disease to look for a pattern of gene expression unique to complicated sarcoidosis.

They were able to identify a distinct 20-gene pattern of gene expression that could reliably identify those most likely to progress to complicated sarcoidosis.

A 31-gene expression signature had been identified previously, but a smaller panel of genes makes the new test less expensive and more useful clinically, said Garcia.

"We are dedicated to looking for new insights as well as new therapies for sarcoidosis and hope to someday be able to identify people at risk for it ahead of time," Garcia said.

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New gene test flags risk of serious complications in sarcoidosis

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Scientists discover that shape matters in DNA nanoparticle therapy

Public release date: 12-Oct-2012 [ | E-mail | Share ]

Contact: Megan Fellman fellman@northwestern.edu 847-491-3115 Northwestern University

Researchers from Johns Hopkins and Northwestern universities have discovered how to control the shape of nanoparticles that move DNA through the body and have shown that the shapes of these carriers may make a big difference in how well they work in treating cancer and other diseases.

This study, to be published in the Oct. 12 online edition of the journal Advanced Materials, is also noteworthy because this gene therapy technique does not use a virus to carry DNA into cells. Some gene therapy efforts that rely on viruses have posed health risks.

"These nanoparticles could become a safer and more effective delivery vehicle for gene therapy, targeting genetic diseases, cancer and other illnesses that can be treated with gene medicine," said Hai-Quan Mao, an associate professor of materials science and engineering in Johns Hopkins' Whiting School of Engineering.

Mao, co-corresponding author of the Advanced Materials article, has been developing nonviral nanoparticles for gene therapy for a decade. His approach involves compressing healthy snippets of DNA within protective polymer coatings. The particles are designed to deliver their genetic payload only after they have moved through the bloodstream and entered the target cells. Within the cells, the polymer degrades and releases DNA. Using this DNA as a template, the cells can produce functional proteins that combat disease.

A major advance in this work is that Mao and his colleagues reported that they were able to "tune" these particles in three shapes, resembling rods, worms and spheres, which mimic the shapes and sizes of viral particles. "We could observe these shapes in the lab, but we did not fully understand why they assumed these shapes and how to control the process well," Mao said. These questions were important because the DNA delivery system he envisions may require specific, uniform shapes.

To solve this problem, Mao sought help about three years ago from colleagues at Northwestern. While Mao works in a traditional wet lab, the Northwestern researchers are experts in conducting similar experiments with powerful computer models.

Erik Luijten, associate professor of materials science and engineering and of applied mathematics at Northwestern's McCormick School of Engineering and Applied Science and co-corresponding author of the paper, led the computational analysis of the findings to determine why the nanoparticles formed into different shapes.

"Our computer simulations and theoretical model have provided a mechanistic understanding, identifying what is responsible for this shape change," Luijten said. "We now can predict precisely how to choose the nanoparticle components if one wants to obtain a certain shape."

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Scientists discover that shape matters in DNA nanoparticle therapy

Recommendation and review posted by Bethany Smith

Shape matters in DNA nanoparticle therapy

ScienceDaily (Oct. 12, 2012) Researchers from Johns Hopkins and Northwestern universities have discovered how to control the shape of nanoparticles that move DNA through the body and have shown that the shapes of these carriers may make a big difference in how well they work in treating cancer and other diseases.

This study, published in the Oct. 12 online edition of Advanced Materials, is also noteworthy because this gene therapy technique does not use a virus to carry DNA into cells. Some gene therapy efforts that rely on viruses have posed health risks.

"These nanoparticles could become a safer and more effective delivery vehicle for gene therapy, targeting genetic diseases, cancer and other illnesses that can be treated with gene medicine," said Hai-Quan Mao, an associate professor of materials science and engineering in Johns Hopkins' Whiting School of Engineering.

Mao, co-corresponding author of the Advanced Materials article, has been developing nonviral nanoparticles for gene therapy for a decade. His approach involves compressing healthy snippets of DNA within protective polymer coatings. The particles are designed to deliver their genetic payload only after they have moved through the bloodstream and entered the target cells. Within the cells, the polymer degrades and releases DNA. Using this DNA as a template, the cells can produce functional proteins that combat disease.

A major advance in this work is that Mao and his colleagues reported that they were able to "tune" these particles in three shapes, resembling rods, worms and spheres, which mimic the shapes and sizes of viral particles. "We could observe these shapes in the lab, but we did not fully understand why they assumed these shapes and how to control the process well," Mao said. These questions were important because the DNA delivery system he envisions may require specific, uniform shapes.

To solve this problem, Mao sought help about three years ago from colleagues at Northwestern. While Mao works in a traditional wet lab, the Northwestern researchers are experts in conducting similar experiments with powerful computer models.

Erik Luijten, associate professor of materials science and engineering and of applied mathematics at Northwestern's McCormick School of Engineering and Applied Science and co-corresponding author of the paper, led the computational analysis of the findings to determine why the nanoparticles formed into different shapes.

"Our computer simulations and theoretical model have provided a mechanistic understanding, identifying what is responsible for this shape change," Luijten said. "We now can predict precisely how to choose the nanoparticle components if one wants to obtain a certain shape."

The use of computer models allowed Luijten's team to mimic traditional lab experiments at a far faster pace. These molecular dynamic simulations were performed on Quest, Northwestern's high-performance computing system. The computations were so complex that some of them required 96 computer processors working simultaneously for one month.

In their paper, the researchers also wanted to show the importance of particle shapes in delivering gene therapy. Team members conducted animal tests, all using the same particle materials and the same DNA. The only difference was in the shape of the particles: rods, worms and spheres.

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Shape matters in DNA nanoparticle therapy

Recommendation and review posted by Bethany Smith

Shape matters in DNA nanoparticle therapy: Particles could become a safer, more effective delivery vehicle for gene …

ScienceDaily (Oct. 12, 2012) Researchers from Johns Hopkins and Northwestern universities have discovered how to control the shape of nanoparticles that move DNA through the body and have shown that the shapes of these carriers may make a big difference in how well they work in treating cancer and other diseases.

This study, to be published in the Oct. 12 online edition of the journal Advanced Materials, is also noteworthy because this gene therapy technique does not use a virus to carry DNA into cells. Some gene therapy efforts that rely on viruses have posed health risks.

"These nanoparticles could become a safer and more effective delivery vehicle for gene therapy, targeting genetic diseases, cancer and other illnesses that can be treated with gene medicine," said Hai-Quan Mao, an associate professor of materials science and engineering in Johns Hopkins' Whiting School of Engineering.

Mao, co-corresponding author of the Advanced Materials article, has been developing nonviral nanoparticles for gene therapy for a decade. His approach involves compressing healthy snippets of DNA within protective polymer coatings. The particles are designed to deliver their genetic payload only after they have moved through the bloodstream and entered the target cells. Within the cells, the polymer degrades and releases DNA. Using this DNA as a template, the cells can produce functional proteins that combat disease.

A major advance in this work is that Mao and his colleagues reported that they were able to "tune" these particles in three shapes, resembling rods, worms and spheres, which mimic the shapes and sizes of viral particles. "We could observe these shapes in the lab, but we did not fully understand why they assumed these shapes and how to control the process well," Mao said. These questions were important because the DNA delivery system he envisions may require specific, uniform shapes.

To solve this problem, Mao sought help about three years ago from colleagues at Northwestern. While Mao works in a traditional wet lab, the Northwestern researchers are experts in conducting similar experiments with powerful computer models.

Erik Luijten, associate professor of materials science and engineering and of applied mathematics at Northwestern's McCormick School of Engineering and Applied Science and co-corresponding author of the paper, led the computational analysis of the findings to determine why the nanoparticles formed into different shapes.

"Our computer simulations and theoretical model have provided a mechanistic understanding, identifying what is responsible for this shape change," Luijten said. "We now can predict precisely how to choose the nanoparticle components if one wants to obtain a certain shape."

The use of computer models allowed Luijten's team to mimic traditional lab experiments at a far faster pace. These molecular dynamic simulations were performed on Quest, Northwestern's high-performance computing system. The computations were so complex that some of them required 96 computer processors working simultaneously for one month.

In their paper, the researchers also wanted to show the importance of particle shapes in delivering gene therapy. Team members conducted animal tests, all using the same particle materials and the same DNA. The only difference was in the shape of the particles: rods, worms and spheres.

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Safety results of intra-arterial stem cell clinical trial for stroke presented

ScienceDaily (Oct. 11, 2012) Early results of a Phase II intra-arterial stem cell trial for ischemic stroke showed no adverse events associated with the first 10 patients, allowing investigators to expand the study to a targeted total of 100 patients.

The results were presented October 11 by Sean Savitz, M.D., professor of neurology and director of the Stroke Program at The University of Texas Health Science Center at Houston (UTHealth), at the 8th World Stroke Congress in Brasilia, Brazil.

The trial is the only randomized, double-blind, placebo-controlled intra-arterial clinical trial in the world for ischemic stroke. It is studying the safety and efficacy of a regenerative therapy developed by Aldagen Inc., a wholly-owned subsidiary of Cytomedix, Inc., that uses a patient's own bone marrow stem cells, which can be administered between 13 and 19 days post-stroke.

The therapy, called ALD-401, consists of stem cells that are identified using Aldagen's proprietary technology to isolate cells that express high levels of an enzyme that serves as a marker of stem cells. Pre-clinical studies found that these cells enhance recovery after stroke in mice. The cells are administered into the carotid artery. Patients are followed for 12 months to monitor safety and to assess mental and physical function.

"We have been approved by the Data Safety Monitoring Board (DSMB) to move the study into the next phase, which will allow us to expand the number of sites in order to complete enrollment," said Savitz, senior investigator for the multi-center study. As per the protocol for the trial, the Food and Drug Administration required a review by the DSMB prior to advancing to the next phase.

Preclinical research, including research at the UTHealth Medical School, has suggested that stem cells can promote the repair of the brain after an ischemic stroke, which is caused by a blood clot in the brain. Stroke is a leading cause of disability and the fourth-leading cause of death in the United States, according to 2008 statistics reported by the Centers for Disease Control and Prevention.

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TOKYO: Shinya Yamanaka, fresh from the Nobel Prize for medicine, states that science and ethics must go hand in hand. Interviewed by the Mainichi Shimbun after the award, he said: "I would like to invite ethical experts as teachers at my laboratory and work to guide iPS [induced pluripotent stem] cell research from that direction as well. The work of a scientific researcher is just one part of the equation. "

Yamanaka, 50, found that adult cells can be transformed into cells in their infancy, stem cells (iPS), which are, so to speak, the raw material for the reconstruction of tissue irreparably damaged by disease. For regenerative medicine the implications of Yamanaka's discovery are obvious. Adult skin cells can for example be reprogrammed and transformed into any other cell that is desired: from the skin to the brain, from the skin to the heart, from the skin to elements that produce insulin.

"Their discovery - says the statement of the jury that awarded him the Nobel Prize on October 8 - has revolutionized our understanding of how cells and organisms develop. Through the programming of human cells, scientists have created new opportunities for the study of diseases and development of methods for the diagnosis and therapy ".

These "opportunities" are not only "scientific", but also "ethical". Much of the scientific research and global investment is in fact launched to design and produce stem cells from embryos, arriving at the point of manipulating and destroying them, facing scientists with enormous ethical problems.

" Ethics are really difficult - Yamanaka explainsto Mainichi - In the United States I began work on mouse experiments, and when I returned to Japan I learned that human embryonic stem cells had been created. I was happy that they would contribute to medical science, but I faced an ethical issue. I started iPS cell research as a way to do good things as a researcher, and I wanted to do what I could to expand the merits of embryonic stem cells. If we make sperm or eggs from iPS cells, however, it leads to the creation of new life, so the work I did on iPS cells led to an ethical problem. If we don't prepare debates for ethical problems in advance, technology will proceed ahead faster than we think.. "

The "ethical question" Yamanaka pushed to find a way to "not keep destroying embryos for our research."

Speaking with his co-workers at the University of Kyoto, immediately after receiving the award, Yamanaka showed dedication and modesty.

"Now - he said - I strongly feel a sense of gratitude and responsibility" gratitude for family and friends who have supported him in a demanding journey of discovery that lasted decades; responsibility for a discovery that gives hope to millions of patients. Now iPS cells can grow into any tissue of the human body allowing regeneration of parts so far irretrievably lost due to illness.

His modesty also led him to warn against excessive hopes. To a journalist who asked him for a message to patients and young researchers awaiting the results of his research heresponded: "The iPS cells are also known as versatile cells, and the technology may be giving the false impression to patients that they could be cured any day now. It will still take five or 10 years of research before the technology is feasible. There are over 200 researchers at my laboratory, and I want patients to not give up hope"

"Dozens of times - he continued - I tried to get some results and I have often failed in the experiments .... Many times I was tempted to give up or cry. Without the support of my family, I could not have continued this search. From now on I will be facing the moment of truth. I would like to return to my laboratory as quickly as possible. "

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U.S. marks first iPS clinical applications

Friday, Oct. 12, 2012

NEW YORK A team of researchers has transplanted artificial cardiac muscle cells developed from multipurpose stem cells into six patients in the United States in the world's first clinical application of iPS cells, one of the researchers said Wednesday.

Shinya Yamanaka, who won this year's Nobel Prize in medicine or physiology for his development of iPS cells, declined comment on the transplants, while other experts said details about the medical performance should be carefully evaluated.

The researchers developed the muscle cells from induced pluripotent stem cells produced from the patients' livers and transplanted them to the patients, said Hisashi Moriguchi, a visiting professor at Harvard University.

A 34-year-old American male patient who was the first to receive the transplant in February now has normal heart functions and has been discharged from the hospital, Moriguchi said.

The patient suffered from liver cancer and received a liver transplant in February 2009. He developed ischemic cardiomyopathy this February, prompting the researchers to conduct the heart surgery.

The researchers took cells from the patient's original liver, which was kept after removal for the 2009 transplant, and developed iPS cells by adding protein and other medical agents from which they produced cardiac muscle cells. The muscle cells were placed in 30 locations in the patient's heart.

No rejection or cancer development was found in the heart, and his heart function gradually recovered to normal levels 10 days after the surgery, they said.

"We need to improve the efficacy and safety of such medical treatment . . . and think of ways to reduce economic burden on patients," Moriguchi said.

The researchers used an improved technique to produce iPS cells developed by Yamanaka, the professor from Kyoto University who jointly won this year's Nobel with John Gurdon of Britain. Such cells have the potential to grow into any type of body tissue.

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New tool determines leukemia cells' 'readiness to die,' may guide clinical care

ScienceDaily (Oct. 11, 2012) Researchers at Dana-Farber Cancer Institute have developed a novel method for determining how ready acute myeloid leukemia (AML) cells are to die, a discovery that may help cancer specialists to choose treatments option more effectively for their patients who have AML.

In a study published in the Oct. 12 issue of the journal Cell, the researchers report that their findings may lead to improved tests to predict which patients successfully treated for AML can continue in remission with standard chemotherapy alone, and which patients are likely to relapse despite additional treatment, but might benefit from a bone marrow transplant.

Anthony Letai, MD, PhD, senior author of the paper, said the study's results also help to explain the "therapeutic index" of AML chemo drugs: That is, how a patient's normal blood-forming stem cells can survive chemotherapy doses that kill the leukemia cells. Unlike current predictive tools, the new method determines the degree to which an individual patient's AML cells are "primed to die" by apoptosis, or programmed cell death. Chemotherapy is more effective when the cancer cells are well along the path to self-destruction, while patients with less-primed leukemia cells are more likely to suffer fatal relapse without a bone marrow transplant, said the researchers.

"Our data suggest that applying our assay in addition to conventional indicators yields a much better predictive tool," said Letai. "We plan to confirm this in independent experiments, and then test its performance prospectively in clinical trials to see if we can use it to do a better job of assigning individualized therapy in AML."

According to the American Cancer Society, an estimated 13,780 cases of AML will be diagnosed in the United States this year, and more than 10,000 people are expected to die from AML, making it the most lethal form of leukemia in the U.S.

Currently, clinicians try to predict an AML patient's outcome by assessing the cancer cells' pathological features and whether the cells contain certain mutations that suggest a poorer response. But these indicators do not provide a biological explanation for patients' differing responses to treatment, noted Letai.

The method described in the new study takes a different approach, first described by Letai in 2011 paper. It employs a technique called "BH3 profiling" to measure the readiness of mitochondria -- tiny organelles within the cell -- to unleash chemical compounds that cause the cell to destroy itself. The self-destruction process, called apoptosis, is triggered by "death molecules," whose mission is to eliminate unneeded or dangerously damaged cells from the body. The study's authors called this readiness for apoptotic self-destruction "mitochondrial priming."

BH3 profiling involves exposing cancer cells to BH3 molecules, which mimic the protein death signals in the body. If the cancer cells' mitochondria membrane is rapidly and easily disrupted, then the cells are considered to be highly primed for death. If the mitochondria strongly resist the disruption, the leukemia cells are further from self-destruction and less likely to respond to chemotherapy.

Applying the method to stored AML patient samples, "We found that mitochondrial priming measured by BH3 profiling was a determinant of initial response to induction [initial] chemotherapy, relapse following remission, and requirement for allogeneic bone marrow transplantation," the authors wrote.

Moreover, knowing whether a patient is likely to have a complete response to chemotherapy would be also very useful in personalizing chemotherapy decisions even when bone marrow transplant is not a consideration. "In elderly patients with AML, chemotherapy can be very toxic with an increased risk of fatal complications," said Letai. "You don't want to give chemotherapy unless you know whether it will benefit. Now we can predict who will benefit from it and who won't -- and should receive an alternative treatment."

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Robin Roberts returns home from hospital following bone marrow transplant

Robin Roberts has returned home from the hospital, following a bone marrow transplant she received with stem cells from her sister last month.

"There's no place like home. After 30 days in the hospital I'm home," Roberts Tweeted on October 11. "Praise God from whom all blessings flow. Thank YOU and bless YOU."

The 51-year-old "Good Morning America" anchor was being treated for myelodysplastic syndrome (MDS), a rare blood and bone marrow disorder. Roberts revealed her ailment in June, saying it was caused in part by treatments she had undergone for breast cancer five years ago. Her older sister, Sally-Ann, was her bone marrow donor.

Check out 9 facts about Robin Roberts, her MDS and bone marrow transplant.

Roberts went on medical leave a day early than she had initially planned in late August in order to visit her ailing mother, Lucimarian Tolliver Roberts. Lucimarian died on August 30 at the age of 88 and Robin Roberts made it back just in time to see her mother.

In the recent blog post, Roberts detailed her difficulties with chemotherapy and how her co-workers' visit helped lift her spirits.

"Today is what I like to call 'Thankful Thursday, aka Friday Eve,'" Roberts wrote in a post on October 4. "I have been in the hospital 25 days now. My bone marrow transplant took place exactly two weeks ago. The only numbers that matter are my blood counts and they are... GREAT! My sister Sally-Ann's stem cells apparently feel right at home in my body -- an answer to so many prayers."

"My doctors and rock star nurses are very pleased with my progress and I could not be more thankful for the excellent care I am receiving," she added. "I have had some extremely painful days and it's still difficult for me to eat because of all the chemo."

Roberts also mentioned a visit she had with fellow "Good Morning America" co-workers Josh Elliott and Sam Champion, which can be seen in the photo above, as well as an upcoming visit from a childhood pastor.

"I continue to learn so much on this journey, especially when it comes to true friendship and love. My friends near and far -- like Sam and Josh who came to visit yesterday -- have been lifting my spirits," Roberts wrote. "My childhood pastor (who delivered Momma's eulogy) is coming from down South to see me tomorrow. I am hopeful that I MAY be well enough to continue my recovery at home next week and my sisters plan to come back to NYC for that milestone in my journey."

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Generation of functional thyroid tissue from stem cells

ScienceDaily (Oct. 11, 2012) The generation of functional thyroid tissue from stem cells could allow the treatment of patients, which suffer from thyroid hormone deficiency due to defective function, or abnormal development of the thyroid gland. The team of Sabine Costagliola at the IRIBHM (Universit Libre de Bruxelles) recently developed a protocol that allowed for the first time the efficient generation of functional thyroid tissue from stem cells in mice and published the results of their studies in the scientific journal Nature.

Thyroid hormones are a class of iodide-containing molecules that play a critical role in the regulation of various body function including growth, metabolism and heart function and that are crucial for normal brain development. The thyroid gland, an endocrine organ that has been specialized in trapping iodide, is the only organ where these hormones are produced. It is, however, of note that one out of 3000 human newborns is born with congenital hypothyroidism, a condition characterized by insufficient production of thyroid hormones. In the absence of a medical treatment with thyroid hormones -- initiated during the first days after birth -- the child will be affected by an irreversible mental retardation. Moreover, a life-long hormonal treatment is necessary in order to maintain proper regulation of growth and general metabolism.

By employing a protocol in which two important genes can be transiently induced in undifferentiated stem cells, the researchers at IRIBHM were able to efficiently push the differentiation of stem cells into thyrocytes, the primary cell type responsible for thyroid hormone production in the thyroid gland.

A first exciting finding of these studies was the development of functional thyroid tissue already within the culture dishes. As a next step, the team of Sabine Costagliola transplanted the stem-cell-derived thyrocytes into mice lacking a functional thyroid gland. Four weeks after transplantation, the researchers observed that transplanted mice had re-established normal levels of thyroid hormones in their blood and were rescued from the symptoms associated with thyroid hormone deficiency. These findings have several important implications. First, the cell system employed by the IRIBHM group provides a vital tool to better characterize the molecular processes associated with embryonic thyroid development. Second, the results of the transplantation studies open new avenues for the treatment of thyroid hormone deficiency but also for the replacement of thyroid tissue in patients suffering from thyroid cancer.

The researchers are currently developing a similar protocol based on human stem cells and explore ways to generate functional human thyroid tissue by reprogramming pluripotent stem cells (iPS) derived from skin cells.

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Riken to test iPS cells in human trial

Friday, Oct. 12, 2012

Stem cells derived from a mouse's skin won Shinya Yamanaka the Nobel Prize in physiology or medicine on Monday. Now researchers in Japan are seeking to use his pioneering technology for an even greater prize: restoring sight.

Scientists at the Riken Center for Developmental Biology in Kobe plan to use induced pluripotent stem (iPS) cells in a human trial using patients with macular degeneration, a disease in which the retina becomes damaged and results in loss of vision, Yamanaka, a Kyoto University professor, told reporters the same day in San Francisco.

Companies including Pfizer Inc. are already planning trials of stem cells derived from human embryos, but Riken's will be the first to use a technology that mimics the power of embryonic cells while avoiding the ethical controversy that accompanies them.

"The work in that area looks very encouraging," John B. Gurdon, 79, a professor at the University of Cambridge who shared this year's Nobel Prize with Yamanaka, said in an interview in London.

Yamanaka and Gurdon split the 8 million Swedish kronor (about 94 million) award for experiments 50 years apart demonstrating that mature cells in latent form retain all of the DNA they had as immature stem cells, and that they can be returned to that potent state.

Their findings offer the potential for a new generation of therapies against hard-to-treat diseases like macular degeneration.

In a study published in 1962, Gurdon took a cell from a tadpole's gut, extracted the nucleus and inserted it into the egg cell of an adult frog whose own nucleus had been removed. The reprogrammed egg cell developed into a tadpole with the genetic characteristics of the original tadpole, and subsequent trials yielded adult frogs.

Yamanaka, 50, built on Gurdon's work by adding four genes to a skin cell from a mouse, returning it to its immature state as a stem cell with the potential to become any cell in the body.

He dubbed them induced pluripotent stem cells.

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Zinc fingers: A new tool in the fight against Huntington's disease

ScienceDaily (Oct. 10, 2012) Huntington's disease (HD) is an inherited genetic disorder caused by the multiple repetition of a DNA sequence (the nucleotides CAG) in the gene encoding a protein called "Huntingtin". People who do not suffer from the disease have this sequence repeated 10 to 29 times. But in an affected person, the triplet is present more than 35 times.

Huntingtin protein can be found in various tissues of the human body and is essential for the development and survival of neurons in adults. When the mutant gene is present, an aberrant form of the Hungtingtin protein is produced, causing the symptoms of the disease: involuntary movements, changes in behavior and dementia, among others. Although there are several promising studies, there is currently no cure for HD. There are only palliative treatments of symptoms, and Huntington's patients die about 15 years after the symptoms onset.

Unlike other neurodegenerative diseases (such as Alzheimer or Parkinson), only a single gene is responsible for HD (i.e. the disorders is monogenic), and a therapy based on the inhibition of the gene, will open new perspectives of research for the development of a treatment.

A recently developed tool by scientists around the world is based on the modification of proteins that are found naturally in all living beings. These proteins are called Zinc Finger proteins, and can recognize and bind to specific DNA sequences. This enables the regulation of those genes to which they are attached.

A study conducted by researchers of the Centre for Genomic Regulation (CRG) in Barcelona provides positive results reducing the chromosomal expression of the mutant gene, which would prevent the development of disease. The research is published in Early Edition by the journal Proceedings of the National Academy of Sciences (PNAS).

"We designed specific ZFP that recognize and specifically bind to more than 35 repetitions of CAG triplet, preventing the expression of the gene containing these repeats and reducing the production of the mutant Huntingtin protein. When applying this treatment to a transgenic mouse model carrying the human mutant Huntingtin gene, we observed a delayed onset of the symptoms, "says Mireia Garriga-Canut, first author of the study and researcher at the Gene Network Engineering group at the CRG. Another co-author of the study, Carmen Agustn Pavn, adds that "the next step is to optimize the design for an effective and durable treatment for patients. This would pave the way to find a therapy for Huntington's disease".

The research was funded by the FP7 program of the European Commission and the Ministry of Science and Innovation of Spain.

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US Panel Urges Gene Privacy Protection

It sounds like a scene from a TV show: Someone sends a discarded coffee cup to a laboratory where the unwitting drinker's DNA is decoded, predicting what diseases lurk in his or her future.

A presidential commission found that's legally possible in about half the states and says new protections to ensure the privacy of people's genetic information are critical if the nation is to realize the enormous medical potential of gene-mapping.

Such whole genome sequencing costs too much now for that extreme coffee-cup scenario to be likely. But the report being released Thursday says the price is dropping so rapidly that the technology could become common in doctors' offices very soon and there are lots of ethical issues surrounding how, when and with whom the results may be shared.

Without public trust, people may not be as willing to allow scientists to study their genetic information, key to learning to better fight disease, the report warns.

"If this issue is left unaddressed, we could all feel the effects," said Dr. Amy Gutmann, who chairs the Presidential Commission for the Study of Bioethical Issues.

Mapping entire genomes now is done primarily for research, as scientists piece together which genetic mutations play a role in various diseases. It's different than getting a lab test to see if you carry, say, a single gene known to cause breast cancer.

Gutmann said her commission investigated ahead of an anticipated boom in genome sequencing as the price drops from thousands today to about $1,000, cheaper than running a few individual gene tests.

AP

The sheer amount of information in a whole genome increases the privacy concerns. For example, people may have their genomes sequenced to study one disease that runs in the family, only to learn they're also at risk for something else with implications for relatives who may not have wanted to know.

Thursday's report shows a patchwork of protection. A 2008 federal law prohibits employers or health insurers from discriminating on the basis of genetic information, so that people don't put off a potentially important gene test for fear of losing their job or health coverage. But that law doesn't prevent denial of life insurance or long-term care insurance. Plus, there's little oversight of how securely genetic information is stored electronically, the report found.

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Gene implicated in schizophrenia risk is also associated with risk for cannabis dependence

ScienceDaily (Oct. 11, 2012) A paper by Shizhong Han and colleagues in the current issue of Biological Psychiatry implicates a new gene in the risk for cannabis dependence. This gene, NRG1, codes for the ErbB4 receptor, a protein implicated in synaptic development and function.

The researchers set out to investigate susceptibility genes for cannabis dependence, as research has already shown that it has a strong genetic component.

To do this, they employed a multi-stage design using genetic data from African American and European American families. In the first stage, a linkage analysis, the strongest signal was identified in African Americans on chromosome 8p21. Then using a genome-wide association study dataset, they identified one genetic variant at NRG1 that showed consistent evidence for association in both African Americans and European Americans. Finally, they replicated the association of that same variant in an independent sample of African-Americans.

All together, the findings suggest that NRG1 may be a susceptibility gene for cannabis dependence.

An interesting feature of this paper is that these findings may also suggest a link between the genetics of schizophrenia and the genetics of cannabis dependence. NRG1 emerged into public awareness after a series of genetic studies implicated it in the heritable risk for schizophrenia. Subsequent studies in post-mortem brain tissue also suggested that the regulation of NRG1 was altered in the brains of individuals diagnosed with schizophrenia.

Thus, the current findings may help to explain the already established link between cannabis use and the risk for developing schizophrenia. A number of epidemiologic studies have attributed the association of cannabis use and schizophrenia to the effects of cannabis on the brain rather than a common genetic link between these two conditions.

"The current data provide a potentially important insight into the heritable risk for schizophrenia and raise the possibility that there are some common genetic contributions to these two disorders," commented Dr. John Krystal, Editor of Biological Psychiatry.

However, further research will be necessary to further confirm the role that NRG1 plays in cannabis dependence and the potential link between cannabis use and psychosis.

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Engineered flies spill secret of seizures

ScienceDaily (Oct. 11, 2012) Scientists have observed the neurological mechanism behind temperature-dependent -- febrile -- seizures by genetically engineering fruit flies to harbor a mutation analogous to one that causes epileptic seizures in people. In addition to contributing the insight on epilepsy, their new study also highlights the first use of genetic engineering to swap a human genetic disease mutation into a directly analogous gene in a fly.

In a newly reported set of experiments that show the value of a particularly precise but difficult genetic engineering technique, researchers at Brown University and the University of California-Irvine have created a Drosophila fruit fly model of epilepsy to discern the mechanism by which temperature-dependent seizures happen.

The researchers used a technique called homologous recombination -- a more precise and sophisticated technique than transgenic gene engineering -- to give flies a disease-causing mutation that is a direct analogue of the mutation that leads to febrile epileptic seizures in humans. They observed the temperature-dependent seizures in whole flies and also observed the process in their brains. What they discovered is that the mutation leads to a breakdown in the ability of certain cells that normally inhibit brain overactivity to properly regulate their electrochemical behavior.

In addition to providing insight into the neurology of febrile seizures, said Robert Reenan, professor of biology at Brown and a co-corresponding author of the paper in the Journal of Neuroscience, the study establishes

"This is the first time anyone has introduced a human disease-causing mutation overtly into the same gene that flies possess," Reenan said.

Engineering seizures

Homologous recombination (HR) starts with the transgenic technique of harnessing a transposable element (jumping gene) to insert a specially mutated gene just anywhere into the fly's DNA, but then goes beyond that to ultimately place the mutated gene into exactly the same position as the natural gene on the X chromosome. HR does this by outfitting the gene to be handled by the cell's own DNA repair mechanisms, essentially tricking the cell into putting the mutant copy into exactly the right place. Reenan's success with the technique allowed him to win a special grant from the National Institutes of Health last year.

The new paper is a result of that grant and Reenan's collaboration with neurobiologist Diane O'Dowd at UC-Irvine. Reenan and undergraduate Jeff Gilligan used HR to insert a mutated version of the para gene in fruit flies that is a direct parallel of the mutation in the human gene SCN1A that causes febrile seizures in people.

When the researchers placed flies in tubes and bathed the tubes in 104-degree F water, the mutant fruit flies had seizures after 20 seconds in which their legs would begin twitching followed by wing flapping, abdominal curling, and an inability to remain standing. After that, they remained motionless for as long as half an hour before recovering. Unaltered flies, meanwhile, exhibited no temperature-dependent seizures.

The researchers also found that seizure susceptibility was dose-dependent. Female flies with mutant strains of both copies of the para gene (females have two copies of the X chromosome) were the most susceptible to seizures. Those in whom only one copy of the gene was a mutant were less likely than those with two to seize, but more likely than the controls.

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Citing privacy concerns, US panel urges end to secret DNA testing

They're called discreet DNA samples, and the Elk Grove, California, genetic-testing company easyDNA says it can handle many kinds, from toothpicks to tampons.

Blood stains from bandages and tampons? Ship them in a paper envelope for paternity, ancestry or health testing. EasyDNA also welcomes cigarette butts (two to four), dental floss ("do not touch the floss with your fingers"), razor clippings, gum, toothpicks, licked stamps and used tissues if the more standard cheek swab or tube of saliva isn't obtainable.

If the availability of such services seems like an invitation to mischief or worse - imagine a discarded tissue from a prospective employee being tested to determine whether she's at risk for an expensive disease, for instance - the Presidential Commission for the Study of Bioethical Issues agrees.

On Thursday it released a report on privacy concerns triggered by the advent of whole genome sequencing, determining someone's complete DNA make-up. Although sequencing "holds enormous promise for human health and medicine," commission chairwoman Amy Gutmann told reporters on Wednesday, there is a "potential for misuse of this very personal data."

"In many states someone can pick up your discarded coffee cup and send it for (DNA) testing," said Gutmann, who is the president of the University of Pennsylvania.

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Iverson Genetic Diagnostics, Inc. Announces an Exclusive Licensing Agreement with Johns Hopkins University School of …

BOTHELL, Wash.--(BUSINESS WIRE)--

Iverson Genetic Diagnostics, Inc. announced today an exclusive licensing agreement with Johns Hopkins University School of Medicine under which Iverson receives global exclusive commercialization rights for molecular diagnostics that are designed to help physicians to assess cardiovascular risk in men and women and infertility risk in women. In this new era of personalized medicine, it is now possible to more accurately determine if the healthy cholesterol fraction, HDL, and its partner protein, scavenger receptor class B type I (SR-BI), affect risk for heart disease in men and women as well as hormonal and fertility outcomes in women. Mutations within the SR-BI gene (SCARB1) are common and work by Annabelle Rodriguez-Oquendo, M.D. at John Hopkins University School of Medicine has suggested that variations within theSCARB1gene show associations with heart disease risk in men and women as well as hormonal and fertility problems in women.

Leroy Hood, M.D., Ph.D., co-founder of the Institute for Systems Biology and a member of Iverson Genetic Diagnostics Board of Directors, commented, The importance of finding gene variants that affect the metabolism of cholesterol, especially the healthy fraction, and hormones--hence causing disease--is incredibly important for personalized medicine. This agreement between Iverson and Johns Hopkins is a wonderful example of a diagnostic test that could significantly improve the health of many patients throughout the world.

DeanSproles, CEO of Iverson Genetic Diagnostics, Inc., stated, We are very pleased to collaborate with Johns Hopkins University School of Medicine on this product and look forward to including the new SR-BI test in the Iversons Physicians LogicTMportfolio later this year.

About Iverson Genetic Diagnostics, Inc.

Iverson Genetic Diagnostics, Inc. is a Nevada C corporation with administrative headquarters in Bothell, Washington, and production headquarters in Charleston, South Carolina. Iverson is establishing a recognizable global brand for providing trusted genetic tests and testing services for the emerging market of individualized medicine and genetics-based molecular diagnostics. The companys mission is to improve patient outcomes through personalized care. Iverson is a fully credentialed laboratory service company focused on providing results within 24 hours for hospitals and physicians. Iversons patented technology, Physicians LogicTM, is our healthcare information resource developed to deploy test results to providers and integrate with various electronic medical record systems in a HIPAA-compliant environment.

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