* Glybera recommended for lipoprotein lipase deficiency

* First (OTC BB: FSTC.OB - news) gene therapy drug to reach market in West

* Green light follows three previous rejections

* Decision is victory for private Dutch biotech firm uniQure (Adds EMA comment, uniQure investors, more background on gene therapy)

LONDON, July 20 (Reuters) - European regulators have recommended approval of the Western world's first gene therapy drug -- after rejecting it on three previous occasions -- in a significant advance for the novel medical technology.

More than 20 years since the first experiments with the ground-breaking method for fixing faulty genes, scientists and drug companies are still struggling to apply gene therapy in practice.

Friday's decision by the European Medicines Agency (EMA) is a win for the drug's maker, the small Dutch biotech company uniQure, and a potential lifeline for patients with the ultra rare genetic disorder lipoprotein lipase deficiency (LPLD).

It comes too late, however, for investors in the previous listed firm Amsterdam Molecular Therapeutics (AMT (Taiwan OTC: 8271.TWO - news) ).

After the earlier rebuffs for its Glybera medicine, AMT was taken private by newly created uniQure in April because it could no longer fund itself in the public markets.

Patients with LPLD are unable to handle fat particles in their blood plasma and are afraid of eating a normal meal because it can lead to acute inflammation of the pancreas.

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UPDATE 1-European regulators back first gene therapy drug

Recommendation and review posted by Bethany Smith

AMSTERDAM, The Netherlands, July 20, 2012 /PRNewswire/ --

uniQure announced today that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances. LPLD is a very rare, inherited disease. Patients with the disease are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain and pancreatitis.

The European Commission (EC) generally follows the recommendations of the CHMP. "We expect final approval from the EC within 3 months after the CHMP decision," says Jrn Aldag, CEO of uniQure. "After today's positive recommendation, Glybera is poised to become the first in a class of gene therapy products approved in Europe to treat orphan diseases, rare conditions with a very high unmet medical need." Marketing authorization covers all 27 European Union member states.

Mr. Aldag continued: "Patients with LPLD are afraid of eating a normal meal because it can lead to acute and extremely painful inflammation of the pancreas, often resulting in a visit to intensive care. Now, for the first time, a treatment exists for these patients that not only reduces this risk of getting severely sick, but also has a multi-year beneficial effect after just a single injection. The positive recommendation from the CHMP for Glybera therefore represents a major breakthrough for both LPLD patients and for medicine as a whole. Restoring the body's natural ability to break down fat particles in the blood in order to prevent pancreatitis and excruciating abdominal pain suffered by patients, is what gene therapy is all about: curing disease at the genetic level."

"At uniQure we are developing treatments for a number of other rare diseases as well, such as acute intermittent porphyria and Sanfilippo B. But the potential of gene therapy stretches far beyond rare diseases. As shown recently in a publication in the New England Journal of Medicine (N Engl J Med 2011; 365:2357-2365, December 22, 2011), hemophilia patients treated with our proprietary gene are showing a sustained clinical effect over several years, which has allowed prophylaxis treatment to be stopped. In addition, we are advancing programs in degenerative diseases such as Parkinson's. We believe that just like antibodies, gene therapy will one day be a mainstay in clinical practice," Mr Aldag added.

As part of the approval, treatment with Glybera will be offered through dedicated centers of excellence with expertise in treating LPLD and by specially trained doctors to ensure ongoing safety of this novel treatment paradigm. uniQure has also committed to building a patient registry for continued understanding of this devastating, under-researched disease. The Company is now preparing to apply for regulatory approval in the US, Canada, and other markets.

Glybera has been tested in three interventional clinical studies conducted in the Netherlands and in Canada, in which a total of 27 LPLD patients participated. In all three clinical trials, Glybera was well tolerated, with no relevant safety issues observed. Data from these clinical trials indicate that a single dose administration of Glybera resulted in a long-term biological activity of the LPL protein.

About Glybera

uniQure has developed Glybera as a therapy for patients with the genetic disorder lipoprotein lipase deficiency. LPLD is an orphan disease for which no treatment exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients. This protein is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD. Glybera has orphan drug designation in the EU and US. LPL Deficiency affects 1-2 persons per million. For further information on LPLD visit http://www.lpldeficiency.com.

About uniQure

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First Gene Therapy in Western World Receives Positive Opinion in Europe from CHMP

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Researchers at Imperial College London have developed an affordable technology that could allow millions of people suffering from ailments like Parkinsons, muscular dystrophy, or spinal cord injury to interact with computers--using just their eyes. The finding brings new hope to many patients that computing--and the many improvements to quality of life the computing brings--could soon be relatively simple and affordable for those who are paralyzed or otherwise disabled.

Its anyones nightmare--to suffer an injury or be diagnosed with a disease that could lead to locked-in syndrome. One feature of locked-in syndrome, though, is that occasionally mobility remains in one part of the body--the eyes. Famously, the French author of The Diving Bell and the Butterfly dictated his memoirs solely through eye-movements--one letter at a time, and with the help of an assistant.

That wont work for everyone, obviously--and nor would the expensive eye-tracking technology of years past. But the Imperial College eye-tracking technology was created with off-the-shelf materials, bringing the cost of the system down to just 40.

"We have built a 3D eye tracking system hundreds of times cheaper than commercial systems and used it to build a real-time brain machine interface that allows patients to interact more smoothly and more quickly than existing invasive technologies that are tens of thousands of times more expensive, Dr. Aldo Faisal, one of the researchers, said of the project. This is frugal innovation; developing smarter software and piggy-backing existing hardware to create devices that can help people worldwide independent of their healthcare circumstances."

The researchers demonstrated how people could play the game of Pong using just eye movements. (The video has an oddly downbeat ending, don't you think, for such a hopeful technology?)

So how does it work? The device is made up of two video game console cameras, which are attached to a pair of glasses, just outside the line of vision. That data can be transmitted over Wi-Fi or USB into a Windows or Linux computer. The device also pairs up with a bit of software that help infer just where the eyes are looking. As the video indicates, the cameras are able to discern just where the pupil is pointing; from this, it can be inferred just where on the screen a users looking. In fact, it even allows you to infer more than that--using a set of detailed calibrations, researchers can even determine how far in 3-D space the user is looking. The researchers speculate novel uses for such technology: for instance, an eye-controlled wheelchair that can determine where you want to go, just by looking.

The Imperial College team is not the only one to have tried its hands at this sort of technology, of course; among others, the University of Minnesota has been at this for some time. Back in January, Tobii Technology presented some gaze interaction tech that was actually aimed at consumers. Tobii mentioned some medical applications, but actually had in mind not patients but rather medical technicians who could use the tech to rapidly scan through photographs, scans, or X-rays, reported the LA Times.

Heres a case where consumer technology and medical technology are involving in tandem and influencing each other. Wherever it catches on first, its good news for everyone--and particularly for the millions of patients for whom this could open up new ways of interacting with the world.

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Controlling a Computer with Your Eyes

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Public release date: 18-Jul-2012 [ | E-mail | Share ]

Contact: David Eve cellmedicinect@gmail.com Cell Transplantation Center of Excellence for Aging and Brain Repair

Tampa, Fla. (July. 18 , 2012) Two studies published in a recent issue of Cell Medicine [2(2)] report on the therapeutic efficacy of stem cell transplantation in animal models of amyotrophic lateral sclerosis (ALS) and spinal cord injury (SCI). Cell Medicine is freely available on-line at http://www.ingentaconnect.com/content/cog/cm.

Mensenchymal stem cell transplantation in spinal cord injured rats promotes functional recovery

Transplantation of mesenchymal stem cells (MSCs), multipotent stem cells with the ability to differentiate into a variety of cell types with renewal capacities, has been found to enhance laboratory animal function after induced spinal cord injury. However, the biological mechanism of the functional enhancement has not been clearly defined.

In an attempt to gain a clearer picture of the mechanism, a team of Korean researchers transplanted MSCs derived from human umbilical cord blood into the tail veins of laboratory rats immediately after spinal cord injury. The intravenous route was selected because the researchers felt that injection into the damaged site could further traumatize the injured spinal cord, although intravenously injected MSCs risk being eliminated by the host immune system.

"We found that MSCs express immunomodulatory effects during the acute phase following SCI," said study corresponding author Dr. Sung-Rae Cho of the Yonsei University College of Medicine in Seoul, Korea. "In our study, MSCs suppressed activated micoglia and inflammatory cytokines, increased anti-inflammatory cytokines and, consequently, promoted functional recovery in SCI rats." They reported "modest but significant improvement" in a number of functional test scores in the rats subjected to transplantation when compared with control group animals not subjected to cell transplantation. The researchers suggested that their study not only confirmed the established link between microglial activation and inflammatory cytokines, but also demonstrated that functional recovery might be attributed to immunomodulatory effects rather than cell replacement. They also recommended that autologous (self-donated) MSCs, rather than human-derived MSCs, should be used in subsequent studies to "suppress undesirable immune response."

Contact: Dr. Sung-Rae Cho, Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, 134 Shinchon-dong, Seodaemun-gu, Seoul, Korea 120-752 Tel: +82 2 2228-3715 Fax: +82 2 363-2795 Email: srcho918@yuhs.ac

Citation: Seo, J. H.; Jang, I. K.; Kim, H.; Yang, M. S.; Lee, J. E.; Kim, H. E.; Eom, Y-W.; Lee, D-H.; Yu, J. H.; Kim, J. Y.; Kim, H. O.; Cho, S-R. Early immunomodulation by intravenously transplanted mesenchymal stem cells promotes functional recovery in spinal cord injured rats. Cell Med. 2(2):55-67; 2011.

Bone marrow cell transplantation coupled with stimulating factor offers neuroprotective and angiogenic effects in ALS animal models

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New studies show spinal cord injury and ALS respond to cell transplantation

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Richard Knox/NPR

Timothy Ray Brown, widely known in research circles as the Berlin patient, was cured of his HIV infection by a bone marrow transplant, doctors say. His story inspired scientists to look for new ways to vanquish the disease in other patients.

Ask AIDS researchers why they think a cure to the disease is possible and the first response is "the Berlin patient."

That patient is a wiry, 46-year-old American from Seattle named Timothy Ray Brown. He got a bone marrow transplant five years ago when he was living in Berlin.

Brown, who now lives in San Francisco, is something of a rock star in the AIDS community. He has made himself endlessly available to researchers, who regularly bleed and biopsy him to learn as much as possible about his amazing cure.

"I have sort of a guilt feeling about being the only person in the world who's been cured so far," Brown said in an interview with NPR. "I'd like to dispel that guilt feeling by making sure that other people are cured."

The transplant was to cure leukemia unrelated to his HIV infection. The German doctors gave Brown a new immune system from a bone marrow donor who is immune to HIV by virtue of a genetic mutation shared by 1 percent of Caucasians.

Brown stopped taking his HIV drugs at the time of the transplant. Five years later, he's still free of HIV drugs and apparently free of HIV. And he's still the only person to be cured of HIV, doctors say, although everyone acknowledges that bone marrow transplantation is not something that could be used routinely for this purpose.

Dr. Steven Deeks at San Francisco General Hospital is following Timothy Brown closely. He's an organizer of a two-day symposium on curing HIV this week in advance of the International AIDS Conference in Washington, D.C.

Until recently, Deeks says, it was virtually taboo to use "HIV" and "cure" in the same sentence.

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HIV Cure Is Closer As Patient's Full Recovery Inspires New Research

Recommendation and review posted by Bethany Smith

We like to think of the Olympics as a level playing field thats why doping is banned. But scientific research complicates this view: There are numerous genetic factors known to confer advantages in athletic contests, from mutations that increase the oxygen carrying capacity of blood to gene variants that confer an incredible increase in endurance, and these mutations appear to be especially common in Olympic athletes. In other words, we may want an egalitarian Olympic games, but it probably isnt in the cards.

In the latest issue of the journal Nature, Juan Enriquez and Steve Gullans, a duo of forward-thinking biotech leaders at the firm Excel Venture Management in Boston, propose an alternative: Push the limits even further. They understand this may be unpalatable to the average fan, but they argue that the games are already full of biological competitive advantages.

They write that almost every male Olympic sprinter and power athlete ever tested carries the 577R allele a version of a gene that enhances performance. And thats just the beginning. There are endurance-related genetic variants in some athletes that have been shown to be far more likely to occur in those who successfully summit high mountains, and less likely to occur in those who fail to. These genes, they argue, are quite common, and athletes probably need a subset of them to achieve elite status.

There are also spectacular examples of extreme abilities that are conferred from genetic mutations. In the 1960s, the Finnish skier Eero Mntyranta won seven Olympic medals in cross-country skiing. Tests later revealed that he had a mutation in his EPOR gene, which improved his bloods oxygen-carrying capacity by somewhere between 25% and 50%. This almost certainly contributed to his remarkable streak of medals; taking supplements that mimic his mutation is strictly banned as doping.

In the face of all these variants, Enriquez and Gullans write that we are already watching a cohort of the genetically elite when we tune in to the Olympics: a showcase of athletes born with genetic advantages, they write. But, of course, some athletes' genetics confer more advantage than others.

In the future, they argue, there are only two ways the playing field could ever truly be level. One would be to handicap individual athletes based on their genetics, a la Kurt Vonneguts story Harrison Bergeron. But the approach that is favored by the authors would be to allow for what is called gene doping to allow athletes who did not win the genetic lottery to upgrade through gene therapy.

In gene therapy, genes are generally ferreted into cells by way of viruses, which allows them to integrate into a persons own DNA and begin producing new proteins. In the case of endurance athletes, one might introduce the coveted variant of the EPOR gene to improve oxygen-carrying capacity.

Given the clear genetic advantages of many athletes, they argue, removing relative genetic disabilities just may be the only way to finally make the Olympics fair while still keeping them fun to watch.

After all, they write, we watch the Games today to marvel at athletes who are faster, higher, stronger whether man or woman, amateur or professional, disabled or not.

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Is gene doping coming to the Olympics?

Recommendation and review posted by Bethany Smith

Public release date: 19-Jul-2012 [ | E-mail | Share ]

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

New Rochelle, NY, July 19, 2012To enable greater reliance on renewable biomass resources for power generation, combination approaches such as co-firing of high percentages of biomass with coal offer unique advantages, but also significant challenges. A comprehensive review of the strategies currently available and in development to improve the characteristics of biomass is presented in Industrial Biotechnology, a peer-reviewed journal from Mary Ann Liebert, Inc.. The article is available free online at the Industrial Biotechnology website.

"Comprehensive and precise characterization of biomass feedstock is important for the development of biotechnology approaches to bioenergy and bioproducts development," says Larry Walker, PhD, Co-Editor-in-Chief and Professor, Biological & Environmental Engineering, Cornell University, Ithaca, NY.

Jaya ShankarTumuluru and colleagues, Idaho National Laboratory (Idaho Falls), describe and compare formulation, pretreatment, and densification options intended to overcome issues related to physical and chemical composition and storage of biomass and the logistics for successful co-firing of <40% mixtures with coal. The Review is entitled "Formulation, Pretreatment, and Densification Options to Improve Biomass Specifications for Co-Firing High Percentages with Coal."

The current issue of Industrial Biotechnology also features a Commentary by Rina Singh, PhD, Senior Policy Director in the Industrial Biotechnology section at the Biotechnology Industry Organization (BIO) on "The National Bioeconomy Blueprint: Meeting Grand Challenges," followed by highlights of the Blueprint.

Gauri Dhavan, Irene Hudson, and J. Peter Fasse, attorneys at Fish & Richardson PC (Boston, MA and New York, NY) take an in-depth look at the impact for the industrial biotechnology community of the Supreme Court's recent decision on patent eligibility of method claims in the Mayo Collaborative Services v. Prometheus Laboratories, Inc. case in the article "Patent Eligibility of Method Claims: What Is the Impact of the Supreme Court's Prometheus Decision?"

A new feature in Industrial Biotechnology, entitled Catalyzing Innovation, in this issue focuses on the application of zinc finger nuclease technology in the aquaculture food industry. Xavier Lauth, John Buchanan, and Keith Hansen, Center for Aquaculture Technologies (San Diego, CA) and Sigma Life Science (St. Louis, MO), describe this innovative technology in the article "Efficient Targeted Genome Editing for Finfish Aquaculture and Other Industries."

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About the Journal

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Strategies to improve renewable energy feedstocks

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Public release date: 19-Jul-2012 [ | E-mail | Share ]

Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center

STANFORD, Calif. The entire genomes of 91 human sperm from one man have been sequenced by Stanford University researchers. The results provide a fascinating glimpse into naturally occurring genetic variation in one individual, and are the first to report the whole-genome sequence of a human gamete the only cells that become a child and through which parents pass on physical traits.

"This represents the culmination of nearly a decade of work in my lab," said Stephen Quake, PhD, the Lee Otterson Professor in the School of Engineering and professor of bioengineering and of applied physics. "We now have devices that will allow us to routinely amplify and sequence to a high degree of accuracy the entire genomes of single cells, which has far-ranging implications for the study of cancer, infertility and many other disorders."

Quake is the senior author of the research, which will be published July 20 in Cell. Graduate student Jianbin Wang and former graduate student H. Christina Fan, PhD, now a senior scientist at ImmuMetrix, share first authorship of the paper.

Sequencing sperm cells is particularly interesting because of a natural process called recombination that ensures that a baby is a blend of DNA from all four of his or her grandparents. Until now, scientists had to rely on genetic studies of populations to estimate how frequently recombination had occurred in individual sperm and egg cells, and how much genetic mixing that entailed.

"Single-sperm sequencing will allow us to chart and understand how recombination differs between individuals at the finest scales. This is an important proof of principle that will allow us to study both fundamental dynamics of recombination in humans and whether it is involved in issues relating to male infertility," said Gilean McVean, PhD, professor of statistical genetics at the Wellcome Trust Centre for Human Genetics. McVean was not involved in the research.

The Stanford study showed that the previous, population-based estimates were, for the most part, surprisingly accurate: on average, the sperm in the sample had each undergone about 23 recombinations, or mixing events. However, individual sperm varied greatly in the degree of genetic mixing and in the number and severity of spontaneously arising genetic mutations. Two sperm were missing entire chromosomes. The study has long-ranging implication for infertility doctors and researchers.

"For the first time, we were able to generate an individual recombination map and mutation rate for each of several sperm from one person," said study co-author Barry Behr, PhD, HCLD, professor of obstetrics and gynecology and director of Stanford's in vitro fertilization laboratory. "Now we can look at a particular individual, make some calls about what they would likely contribute genetically to an embryo and perhaps even diagnose or detect potential problems."

Most cells in the human body have two copies of each of 23 chromosomes, and are known as "diploid" cells. Recombination occurs during a process called meiosis, which partitions a single copy of each chromosome into a sperm (in a man) or egg (in a woman) cell. When a sperm and an egg join, the resulting fertilized egg again has a full complement of DNA.

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Stanford researchers first to determine entire genetic sequence of individual human sperm

Recommendation and review posted by Bethany Smith

New approach sorts cancer driver mutations from abundant but irrelevant passengers

Newswise HOUSTON Its been a burning question in melanoma research: Tumor cells are full of ultraviolet (UV)-induced genetic damage caused by sunlight exposure, but which mutations drive this cancer?

None have been conclusively tied to melanoma. The sheer abundance of these passenger mutations has obscured the search for genetic driver mutations that actually matter in melanoma development and progression.

By creating a method to spot the drivers in a sea of passengers, scientists at the Broad Institute of MIT and Harvard, the Dana-Farber Cancer Institute and The University of Texas MD Anderson Cancer Center have identified six genes with driving mutations in melanoma, three of which have recurrent hotspot mutations as a result of damage inflicted by UV light. Their findings are reported in the July 20 issue of the journal Cell.

Those three mutations are the first smoking gun genomic evidence directly linking damage from UV light to melanoma, said co-senior author Lynda Chin, M.D., Professor and Chair of MD Andersons Department of Genomic Medicine. Until now, that link has been based on epidemiological evidence and experimental data.

This study also is exciting because many of the recent large-scale genomic studies have not discovered new cancer genes with recurrent hot-spot mutations, a pattern strongly indicative of biological importance, said Chin, who also is scientific director of MD Andersons Institute for Applied Cancer Science.

The six new melanoma genes identified by the team are all significantly mutated and provide potential targets for new treatments.

Puzzle has thousands of potential pieces, but only requires a few dozen A number of important mutations had previously been identified as melanoma drivers. These include BRAF (V600) mutations, present in half of all melanomas, and NRAS (Q61) mutations. However, the vast majority of these mutations do not appear to be caused by direct damage from UV light exposure.

Those known mutations are important, but dont tell the whole story. Melanoma, the authors note, has higher genetic mutation rates than most other types of solid tumors. The majority are attributable to passenger mutations caused by UV light damage resulting in a DNA alteration called a cytidine (C) to thymidine (T) transition.

Chin together with Levi A. Garraway M.D, Ph.D., associate professor at Dana-Farber Cancer Institute and Harvard Medical School and senior associate member at the Broad Institute, sequenced the exons active portions of DNA involved in protein synthesis in 121 melanoma samples paired with normal DNA and found 86,813 coding mutations. The resulting mutation rate was higher than that ever reported in any other tumor type.

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Scientists Discover Melanoma-Driving Genetic Changes Caused by Sun Damage

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More than 200 researchers investigating colon cancer tumors have found genetic vulnerabilities that could lead to powerful new treatments. The hope is that drugs designed to strike these weak spots will eventually stop a cancer that is now almost inevitably fatal once it has spread.

Scientists increasingly see cancer as a genetic disease defined not so much by where it starts colon, liver, brain, breast but by genetic aberrations that are its Achilles' heel. And with a detailed understanding of which genetic changes make a cancer grow and thrive, they say they can figure out how best to mount an attack. They caution that most of the drugs needed to target the cancer mutations have yet to be developed.

The colon cancer study, published Wednesday in Nature, is the first part of a sweeping effort that is expected to produce a flood of discoveries for a wide range of cancers.

"There are so many different ways that you can attack this tumor type," said Raju Kucherlapati, the principal investigator for the colon cancer project and a professor of genetics and of medicine at Harvard Medical School. "We have an opportunity to completely change the landscape."

Researchers have studied colon cancer before and have identified mutations that seemed critical, but their work lacked the scope of the new project and provided more limited information on genetic changes, said Dr. Sanford Markowitz, a colon cancer and genomics expert at Case Western Reserve University.

He, like nearly every other leading scientist in colon cancer genomics, is an author of the study.

About 150,000 Americans receive a diagnosis of colon or rectal cancer each year, and about 50,000 die annually from the disease.

The hope now is that the genetic alterations driving tumors are operating through only a limited number of genetic pathways that can be targeted by a more manageable number of drugs.

Those drugs have yet to be developed, said Dr. S. Gail Eckhardt, the head of the division of medical oncology at the University of Colorado and another author of the study. But, she added, the work "confirms where some of the drug development should be going."

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Colon cancer researchers find genetic vulnerabilities

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An Otago University genetics student wants to have his genetic coding mapped and put onto a disk.

Steve Anderson approached genetics service New Zealand Genomics, in the hope of being able to study his DNA and genes as he ages.

Mr Anderson is a student in his 50s at Otago University, and has asked New Zealand Genomics to map his DNA for him.

He says there are over four and a half thousand genetic disorders.

"I'm sure most people in the future may be five- 10 years down the track will have their genome sequenced. it will be part of their medical records and then they'll be able to follow through with any disorders or various things that they find."

But he says there are numerous ethical and privacy issues which surround this - for example family members will have similar coding, which could reveal certain genetic disorders.

"Also I think there's issues with insurance companies and various things like this. If you have a problem coming up in the future and you yourself have been able to work that out then what's the chances of insurance and can they discriminate against you?"

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Otago University student wants a copy of his genetic coding

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In a lecture at the Royal Institution in London, Nobel Prize-winning biologist Sir Paul Nurse said that, on the whole, biologists tend to leave grand ideas and theories to physicists and concentrate more on details. "What we like to do is count things and list things," Nurse said, adding that a 19th century biologist might count the numbers of hairs on a beetle's leg, while an ecologist might count and list the number and type of species in a habitat and a molecular biologist would sequence genes and count the numbers of proteins and RNAs in a cell. But biology also has great ideas, Nurse said. His lecture, presented in a series of videos at The Guardian, concentrates on five of these ideas the cell, the gene, natural selection, life as chemistry, and biology as an organized system, the latter of which, he added, has "yet to be fully formulated."

In his lecture on the gene, Nurse started with Gregor Mendel and his peas and traced how that one idea gave rise to heredity, James Watson and Francis Crick's discovery of the double helix, and to what is now known about the genome. The idea of the gene as the basis of heredity can explain the link between genotype and phenotype, he said, adding, "It has tremendous implications for what we are."

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Genetics' 'Tremendous Implications'

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SAN MARINO, Calif.--(BUSINESS WIRE)--

The first patient has now been enrolled into the Phase 1 clinical trial sponsored by Viral Genetics (OTC Pink: VRAL) and supported by a donation from Scott and White Foundation. The trial will study Metabolic Disruption Technology (MDT) compounds in combination with an existing cancer therapy to treat drug-resistant ovarian cancer. A total of up to 24 patients will receive combination treatment of hydroxychloroquine and sorafenib (marketed as Nexavar) under primary investigator, Tyler Curiel, M.D., MPH, a medical oncologist affiliated with The Cancer Therapy and Research Center (CTRC) of The University of Texas Health Science Center at San Antonio. This clinical trial is the first sponsored by Viral Genetics based on the licensed research of Dr. M. Karen Newell-Rogers, the Companys Chief Scientist, and represents a milestone in the transition of the Company from preclinical- to clinical-stage. Patient enrollment is also expected to commence at Scott and White Hospital as soon as internal review procedures there are finalized.

Patient enrollment marks the formal beginning of a clinical trial and so we are quite happy to get underway after much preparation and hard work by our team members, and Dr. Curiels group, said Haig Keledjian, President of Viral Genetics. I want to emphasize that we would not have proceeded with this choice of one MDT compound if we and our advisors were not confident that it held real promise for patients, but one should also appreciate the severity of the illnesses we are attempting to treat in this study. We advise optimistic but cautious and restrained expectations.

Because of the staggered nature of patient enrollment which calls for a few patients to be enrolled and treated at low doses prior to enrolling additional patients at higher doses, full enrollment of the study could take up to a few months. Follow up and patient observation will continue post-treatment for up to 12 months. The study can be stopped at any time for safety reasons.

Study Design

Title: A Phase I Dose-Escalation Trial of Oral Hydroxychloroquine Plus Oral Sorafenib to Treat Epithelial Ovarian Cancer FIGO Stage III or Stage IV, or Extraovarian Peritoneal Carcinoma, or Fallopian Tube Carcinoma Failing or Ineligible for First-Line Therapy (HSO1)

The HSO1 study will examine the safety and efficacy of one of the MDT compounds, hydroxychloroquine (HCQ), in combination with an existing cancer drug, sorafenib (marketed as Nexavar) in the treatment of resistant or otherwise untreatable Stage III or IV ovarian cancer including related carcinomas. Viral Genetics is sponsoring the HSO1 study, which is taking place at the Cancer Therapy and Research Center and, eventually at Scott and White Hospital system.

The primary endpoints of the HSO1 study are related to safety and reflect the dose-escalation design of the trial, which is intended to find a maximum tolerated dose of different combinations of HCQ and sorafenib in patients while being alert to any potential toxicities. The study can be stopped in the event of significant adverse events, including toxicity. Both HCQ and sorafenib have separately been extensively studied in humans for safety and toxicity, and they have fairly well-understood individual safety profiles. This will be the first study to examine them in combination using the MDT research suggesting that such a dual-pronged approach would significantly enhance results of single agents alone.

Efficacy will also be evaluated through secondary endpoints in three ways: objective tumor responses (defined under the global Response Evaluation Criteria in Solid Tumors standard), progression-free survival, and certain immune system blood markers. A positive response would generally be characterized by shrinkage in tumor, lesion, or lymph nodes and/or patient survival without advancement of their existing cancer. Additional signs of efficacy include improvement or normalization in certain blood markers (including CA-125 and immunological tests) that are linked to successful treatment responses in ovarian cancer, although this would not generally be considered as a successful outcome on its own without other improvements as well.

There will be up to four separate groups or cohorts in the HSO1 study, each of which will have up to 6 patients. Each cohort will be given a combination of doses of HCQ and sorafenib over a treatment cycle of 28 days. Presuming low levels of toxicity following dosing, additional patients will be added and higher doses of the combination will be given. Depending on the outcome of the primary, and to a lesser extent, secondary endpoints, the study will enroll between 2 and 24 patients in total. Treatment will generally continue for as long as toxicity results remain within acceptable limits and patients experience clinical benefit. Results will be available throughout the course of each 28 day cycle, and will be reviewed by a Data Safety Monitoring Board.

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Viral Genetics Begins Phase 1 Clinical Trial for Ovarian Cancer

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(Phys.org) -- New research at the Hebrew University of Jerusalem sheds light on pluripotencythe ability of embryonic stem cells to renew themselves indefinitely and to differentiate into all types of mature cells. Solving this problem, which is a major challenge in modern biology, could expedite the use of embryonic stem cells in cell therapy and regenerative medicine. If scientists can replicate the mechanisms that make pluripotency possible, they could create cells in the laboratory which could be implanted in humans to cure diseases characterized by cell death, such as Alzheimer's, Parkinson's, diabetes and other degenerative diseases.

To shed light on these processes, researchers in the lab of Dr. Eran Meshorer, in the Department of Genetics at the Hebrew Universitys Alexander Silberman Institute of Life Sciences, are combining molecular, microscopic and genomic approaches. Meshorer's team is focusing on epigenetic pathwayswhich cause biological changes without a corresponding change in the DNA sequencethat are specific to embryonic stem cells.

The molecular basis for epigenetic mechanisms is chromatin, which is comprised of a cell's DNA and structural and regulatory proteins. In groundbreaking research performed by Shai Melcer, a PhD student in the Meshorer lab, the mechanisms which support an open chromatin conformation in embryonic stem cells were examined. The researchers found that chromatin is less condensed in embryonic stem cells, allowing them the flexibility or "functional plasticity" to turn into any kind of cell.

A distinct pattern of chemical modifications of chromatin structural proteins (referred to as the acetylation and methylation of histones) enables a looser chromatin configuration in embryonic stem cells. During the early stages of differentiation, this pattern changes to facilitate chromatin compaction.

But even more interestingly, the authors found that a nuclear lamina protein, lamin A, is also a part of the secret. In all differentiated cell types, lamin A binds compacted domains of chromatin and anchors them to the cells nuclear envelope. Lamin A is absent from embryonic stem cells and this may enable the freer, more dynamic chromatin state in the cell nucleus. The authors believe that chromatin plasticity is tantamount to functional plasticity since chromatin is made up of DNA that includes all genes and codes for all proteins in any living cell. Understanding the mechanisms that regulate chromatin function will enable intelligent manipulations of embryonic stem cells in the future.

"If we can apply this new understanding about the mechanisms that give embryonic stem cells their plasticity, then we can increase or decrease the dynamics of the proteins that bind DNA and thereby increase or decrease the cells differentiation potential," concludes Dr. Meshorer. This could expedite the use of embryonic stem cells in cell therapy and regenerative medicine, by enabling the creation of cells in the laboratory which could be implanted in humans to cure diseases characterized by cell death, such as Alzheimer's, Parkinson's, diabetes and other degenerative diseases.

More information: The research appears in the journal Nature Communications as Melcer et al., Histone modifications and lamin A regulate chromatin protein dynamics in early embryonic stem cell differentiation. go.nature.com/9B33Ue

Journal reference: Nature Communications

Provided by Hebrew University of Jerusalem

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Researchers identify mechanisms that allow embryonic stem cells to become any cell in the human body

Recommendation and review posted by simmons

Friends and family of a Lincoln County man who suffered a spinal cord injury Saturday in Lake Norman say he is making slow but steady recovery and might be moved out of intensive care Wednesday.

Adam Dancoff, 21, a Lincolnton firefighters for more than a year, suffered a broken vertebra when he dived from a boat into the lake. Dancoff apparently didnt realize the water was shallow in that area, and he lost the ability to move his legs.

Dancoff was airlifted to Carolinas Medical Center. According to information posted by family and friends on a special Facebook page, he underwent surgery Sunday and has remained in intensive care.

On the Facebook page, family members said doctors have little if any hope that Dancoff will regain movement in the lower part of his body.

However, he has been working to regain mobility in the rest of his body. With the help of nurses, he was able to sit in his hospital bed Tuesday.

Family and friends say they hope that Dancoff can be moved to a rehabilitation center later this week.

You can follow Dancoffs progress on the Facebook page.

See the original post here:
Firefighter battles spinal cord injury

Recommendation and review posted by sam

Researchers at Georgia Institute of Technology have developed a novel device called the Mobile Music Touch -- a wireless glove intended to improve motor skills in people who have suffered a paralyzing spinal cord injury.

The gadget, which looks like a workout glove with a small box on its back, is meant to be used in combination with a piano. The device vibrates the wearers fingers, identifying which keys they are meant to play.

The glove was tested on people who had tetraplegia -- partial paralysis in their limbs. The patients had sustained their injuries a year prior to the study. During this time frame after receiving a paralyzing injury, people in rehab rarely see significant improvement in their limb movement for the rest of their lives.

However, while learning to play piano with the MMT, several patients saw improvement in their fingers motility.

After our preliminary work in 2011, we suspected that the glove would have positive results for people with SCI, Tanya Markow, a PhD graduate of Georgia Tech as well as the projects leader, said in a release.

But we were surprised by how much improvement they made in our study. For example, after using the glove, some participants were able to feel the texture of their bed sheets and clothes for the first time since their injury.

Markow worked with patients with SCI over the course of eight weeks, requiring them to practice piano for 30 minutes, three times a week. Half of them wore the glove, the other half did not. Hooked up to a computer or other MP3 playing device, the MMT played a song such as Ode to Joy while the corresponding notes on the keyboard became illuminated. The glove would then vibrant the corresponding finger, signaling the wearer to tap the illuminated key.

Not only did participants practice piano with the MMT glove, but they used it in day-to-day activities as well. For two hours a day, five days a week, participants wore the glove, only feeling the vibrations a technique that was revealed to help them learn to play piano faster.

To test the effectiveness of the glove, patients were required to perform various grabbing and sensing tests at the studys completion. Those who used the glove performed significantly better than those who did not use the glove.

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Wireless glove teaches music, helps those with spinal cord injuries

Recommendation and review posted by sam

Parkinson's is a horrible degenerative disorder of the central nervous system which is sadly incurable. But now a team of scientists from Johns Hopkins has been able to grow the brain cells which are usually destroyed by the disease from skin stem cellsand they're confident it will help them develop new treatments.

In fact, their experiments have already been using the lab-grown brain cells to test the effectiveness of drugs currently in development to treat Parkinson's. More exciting, they explain in their report, published in Science Translational Medicine, that the ability to test in the lab should massively speed up the search for new drugs to treat the condition. Ted M. Dawson explains another possibility of the development:

"Our study suggests that some failed drugs should actually work if they were used earlier, and especially if we could diagnose Parkinson's before tremors and other symptoms first appear."

While scientists have in the past been able to halt the disease in mice, none of the compounds used to do so have translated effectively to humans. That suggests that the disease works differently in humans to animals, and makes the new finding all the more useful.

The current thinking is that Parkinson's damages the mitochondria of dopamine neurons in the brain, in effect cutting off their energy supply. The next step for the Johns Hopkins researchers, then, is to investigate how they can slow that damage in the lab-grown cells. [Science Translational Medicine via John Hopkins]

Image by Lasse Kristensen/Shutterstock

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Scientists Use Skin To Replace Brain Cells Destroyed By Parkinson's [Science]

Recommendation and review posted by Bethany Smith

(Phys.org) -- New research at the Hebrew University of Jerusalem sheds light on pluripotencythe ability of embryonic stem cells to renew themselves indefinitely and to differentiate into all types of mature cells. Solving this problem, which is a major challenge in modern biology, could expedite the use of embryonic stem cells in cell therapy and regenerative medicine. If scientists can replicate the mechanisms that make pluripotency possible, they could create cells in the laboratory which could be implanted in humans to cure diseases characterized by cell death, such as Alzheimer's, Parkinson's, diabetes and other degenerative diseases.

To shed light on these processes, researchers in the lab of Dr. Eran Meshorer, in the Department of Genetics at the Hebrew Universitys Alexander Silberman Institute of Life Sciences, are combining molecular, microscopic and genomic approaches. Meshorer's team is focusing on epigenetic pathwayswhich cause biological changes without a corresponding change in the DNA sequencethat are specific to embryonic stem cells.

The molecular basis for epigenetic mechanisms is chromatin, which is comprised of a cell's DNA and structural and regulatory proteins. In groundbreaking research performed by Shai Melcer, a PhD student in the Meshorer lab, the mechanisms which support an open chromatin conformation in embryonic stem cells were examined. The researchers found that chromatin is less condensed in embryonic stem cells, allowing them the flexibility or "functional plasticity" to turn into any kind of cell.

A distinct pattern of chemical modifications of chromatin structural proteins (referred to as the acetylation and methylation of histones) enables a looser chromatin configuration in embryonic stem cells. During the early stages of differentiation, this pattern changes to facilitate chromatin compaction.

But even more interestingly, the authors found that a nuclear lamina protein, lamin A, is also a part of the secret. In all differentiated cell types, lamin A binds compacted domains of chromatin and anchors them to the cells nuclear envelope. Lamin A is absent from embryonic stem cells and this may enable the freer, more dynamic chromatin state in the cell nucleus. The authors believe that chromatin plasticity is tantamount to functional plasticity since chromatin is made up of DNA that includes all genes and codes for all proteins in any living cell. Understanding the mechanisms that regulate chromatin function will enable intelligent manipulations of embryonic stem cells in the future.

"If we can apply this new understanding about the mechanisms that give embryonic stem cells their plasticity, then we can increase or decrease the dynamics of the proteins that bind DNA and thereby increase or decrease the cells differentiation potential," concludes Dr. Meshorer. This could expedite the use of embryonic stem cells in cell therapy and regenerative medicine, by enabling the creation of cells in the laboratory which could be implanted in humans to cure diseases characterized by cell death, such as Alzheimer's, Parkinson's, diabetes and other degenerative diseases.

More information: The research appears in the journal Nature Communications as Melcer et al., Histone modifications and lamin A regulate chromatin protein dynamics in early embryonic stem cell differentiation. go.nature.com/9B33Ue

Journal reference: Nature Communications

Provided by Hebrew University of Jerusalem

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Researchers identify mechanisms that allow embryonic stem cells to become any cell in the human body

Recommendation and review posted by Bethany Smith

DNA microarrays allow researchers to analyse the expression of a huge number of genes simultaneously.

A. Nantel/Shutterstock

Purvesh Khatri sits in front of an oversized computer screen, trawling for treasure in a sea of genetic data. Entering the search term breast cancer into a public repository called the Gene Expression Omnibus (GEO), the postdoctoral researcher retrieves a list of 1,170 experiments, representing nearly 33,000 samples and a hoard of gene-expression data that could reveal previously unseen patterns.

That is exactly the kind of search that led Khatris boss, Atul Butte, a bioinformatician at the Stanford School of Medicine in California, to identify a new drug target for diabetes. After downloading data from 130 gene-expression studies in mice, rats and humans, Butte looked for genes that were expressed at higher levels in disease samples than in controls. One gene was strikingly consistent: CD44, which encodes a protein found on the surface of white blood cells, was differentially expressed in 60% of the studies (K. Kodama et al. Proc. Natl Acad. Sci. USA 109, 70497054; 2012). The CD44 protein is not widely investigated as a drug target for diabetes, but Buttes team found that treating obese mice with an antibody against it caused their blood glucose levels to drop.

Butte and his team are now using publicly available data to answer a diverse range of questions Khatri, for instance, hopes to discover secrets behind kidney-transplant rejection. We dont do wet lab experiments for discovery, he says. Those are for validating hypotheses. The beauty of analysing data from multiple experiments is that biases and artefacts should cancel out between data sets, helping true relationships to stand out, Butte says. There is safety in numbers.

And those numbers are rising rapidly. Since 2002, many scientific journals have required that data from gene-expression studies be deposited in public databases such as GEO, which is maintained by the National Center for Biotechnology Information in Bethesda, Maryland, and ArrayExpress, a large gene-expression repository at the European Bioinformatics Institute (EBI) in Hinxton, UK. Some time in the next few weeks, the number of deposited data sets will top one million (see Data dump).

The result is an unprecedented resource that promises to drive down costs and speed up progress in understanding disease. Gene-sequence data are already shared extensively, but expression data are more complex and can reveal which genes are the most active in, say, liver versus brain cells, or in diseased versus healthy tissue. And because studies often look at many genes, researchers can repurpose the data sets, asking questions other than those posed by the original researchers.

Sources: NIH, EBI

It is easy to track how many data sets are being deposited much harder is working out how they are being used. Heather Piwowar, who studies data reuse with the National Evolutionary Synthesis Center from the University of British Columbia in Vancouver, Canada, found that 20% of data sets deposited in GEO in 2005 and 17% of those in 2007 had been cited by the end of 2010. But those rates are certainly underestimates, she says. The PubMed Central repository, which her study relied on, holds only about one-third of the relevant papers, and her algorithms identify reuse only when researchers cite database accession numbers, which many dont do. More studies are reusing data every year, she says. We have every reason to believe it is game-changing.

Having access to such data is immensely valuable, agrees Enrico Petretto, a genomicist at Imperial College London. We would never be in a position to look across multiple tissues and species with the money we have. But he cautions that using other peoples data can be tricky. If data sets give contradictory outcomes, it is unclear whether that is because the underlying data contradict each other or because something went wrong with the analysis. Thats why people sometimes dont trust this, he says.

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Gene data to hit milestone

Recommendation and review posted by Bethany Smith

Public release date: 18-Jul-2012 [ | E-mail | Share ]

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

New Rochelle, NY, July 18, 2012 Approximately 1 in 3 Americans suffers from chronic sleep deprivation and another 10-15% of the population has chronic insomnia. Sleep disorders can profoundly affect a person's whole life and have been linked to a range of diseases, including obesity, depression, anxiety, and inflammatory disorders. Over-the-counter herbal remedies are often used to treat insomnia, but surprisingly, very little research has been done to study their efficacy, according to an article in Alternative and Complementary Therapies, published by Mary Ann Liebert, Inc., publishers. The article is available free on the Alternative and Complementary Therapies website at http://www.liebertpub.com/act.

People need many hours of sound, restorative sleep every night to maintain an optimal state of physiological and psychological health, but many factors can disrupt sleep schedules and compromise the quality of sleep. In the article, "SleepNaturally: A Review of the Efficacy of Herbal Remedies for Managing Insomnia," the authors conducted a search of the Internet and electronic databases to identify literature on herbal remedies that are commonly used to manage insomnia, including valerian, hops, kava-kava, chamomile, and St. John's wort. They found that few scientific studies had been published that reported on the therapeutic potential and safety of these herbal remedies and the results were either inconclusive or contradictory.

The authors concluded that, considering the benefits that a natural management strategy could offer patients with insomnia, additional research is required to assess the effectiveness and safety of herbal remedies as therapeutic agents.

###

About the Journal

Alternative and Complementary Therapies is a bimonthly journal that publishes original articles, reviews, and commentaries evaluating alternative therapies and how they can be integrated into clinical practice. Topics include botanical medicine, vitamins and supplements, nutrition and diet, mind-body medicine, acupuncture and traditional Chinese medicine, ayurveda, indigenous medicine systems, homeopathy, naturopathy, yoga and meditation, manual therapies, energy medicine, and spirituality and health. Complete tables of content and a sample issue may be viewed on the Alternative and Complementary Therapies website at http://www.liebertpub.com/act.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including The Journal of Alternative and Complementary Medicine, Medical Acupuncture, and Journal of Medicinal Food. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc. website at http://www.liebertpub.com.

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Efficacy of herbal remedies for managing insomnia

Recommendation and review posted by Bethany Smith

Lintao Zhangy/Getty Images

That seems to be the question behind a new opinion piece in this week's journal Nature. As scientists uncover the genes that help people become world-class sprinters or record-breaking skiers, the idea that medals are won with just hard work, sweat and tears begins to feel outdated, according to the authors.

"When you start sequencing [the genes] of lots and lots of human beings, what we're going to find out is that we're more different than people had realized," said Steve Gullans, a managing director of Excel Venture Management in Boston, who co-wrote the piece with his colleague Juan Enriquez.

Already, Gullans said, DNA tests have shown that some Olympic athletes have distinct advantages. Finnish cross-country skier and seven-time Olympic medalist Eero Mntyranta, for example, carried a mutation in his EPOR gene that meant he produced up to 25 percent more red blood cells than the norm. That mutation gave Mntyranta an edge because his blood carried more oxygen than the blood of people without the mutation, Gullans told LiveScience. And that raises the question of whether "gene doping," or gene therapy to improve performance, should be banned.

"If someone else is carrying the EPOR receptor that I don't have, why shouldn't I be able to give it to myself to play on an equal playing field?" Gullans said. [7 Amazing Superhuman Feats]

The genome and the Olympics

Gene doping has been banned by the International Olympic Committee since 2003, though the actual therapies that could boost athletic performance remain largely theoretical. Nevertheless, gene therapy is becoming more common, raising new questions, Gullans said. Suppose scientists invented a gene-therapy procedure to cure sickle-cell anemia in babies, he said. Would a child who received the treatment forever be banned from the Olympics?

As the rules are written today, they likely would, Gullans said. The World Anti-Doping Agency rules prohibit "the transfer of nucleic acids or nucleic acid sequences" and "the use of normal or genetically modified cells" if those methods have "the potential to enhance sport performance."

This prohibition is much broader than the ban on drugs, which are split into performance-enhancing and allowed categories, Gullans said. It's likely that officials will have to grapple with a number of ethical gray areas as genetic manipulation advances.

Another example: Imagine that a genetic treatment could slow aging, so that people stayed healthy and youthful until after they were 100 years old, Gullans said. Would Olympic athletes be the only people forced to abstain?

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Scientist Suggests That Genetic Engineering Will Kill The Olympics

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/563f2h/china_genetic_engi) has announced the addition of the "China Genetic Engineering Drug Industry Report, 2011-2012" report to their offering.

At present, China has at least one hundred enterprises involved in genetic engineering drugs. In recent years, the compound growth rate of genetic engineering drug market in China is as high as 49%, with an average gross margin of more than 80%.

However, the technology strength and efficacy of locally produced genetic engineering drugs are relatively weak. In particular, the pegylated recombinant human granulocyte colony stimulating factor (PEG-rhG-CSF) for injection of CSPC Pharmaceutical Group Limited that approved for marketing in March 2012 is the only homemade long-acting protein product. Still, due to the impetus of huge market capacity as well as a package of preferential policies, many domestic enterprises, including GeneScience Pharmaceuticals, Amoytop and Anhui Anke Biotechnology, are accelerating the industrialized research of long-acting protein drugs.

Although China lags behind in terms of the overall level of genetic engineering drugs, the industry is now rich R&D and industrialization experience as well as capital reserves. Thus, with a host of genetic engineering drug patents to become due, Chinese enterprises, such as Walvax, are committed to the industrialization research of monoclonal antibody, long-acting recombinant protein drugs and other generic drugs with high technical barriers.

Key Topics Covered:

1 Profile of Biopharmaceutical Industry

1.1 Definition and Classification

1.2 Genetic Engineering Drugs

2 Overview of China Genetic Engineering Drug Industry

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Research and Markets: China Genetic Engineering Drug Industry Report, 2011-2012

Recommendation and review posted by Bethany Smith

LEUVEN, BELGIUM--(Marketwire -07/17/12)- TiGenix (EURONEXT:TIG), the European leader in cell therapy, announced today that after obtaining national reimbursement for ChondroCelect in the Netherlands last month, the company has now contracted with four major hospitals to make its innovative cartilage repair therapy available to their patients on a routine basis: University Medical Center Utrecht, University Hospital Maastricht, Martini Hospital Groningen, and, most recently, the Elisabeth Hospital Tilburg. Discussions with other Cartilage Expert Centers are ongoing. Reimbursement for ChondroCelect in the Netherlands is retroactive per January 1, 2011.

"Our close collaboration with the Dutch hospitals is key to making ChondroCelect available to patients in the Netherlands," said Eduardo Bravo, CEO of TiGenix. "Dutch scientists and clinicians have made important contributions to the development of this innovative cartilage repair therapy. Patients who suffer from cartilage lesions in the knee that cause recurrent pain and can be incapacitating can now be routinely treated and literally find their footing again. We expect to soon expand the number of hospitals in the Netherlands where ChondroCelect is available."

Damage to the articular cartilage in the knee can be caused by sports or professional activities in which the knee is repeatedly and forcefully impacted. It is a condition that predominantly occurs in young adults, who as a result suffer from recurrent pain, locking or limited range of motion, and risk being incapacitated. TiGenix has developed ChondroCelect as a therapy to help patients regain their mobility and fully active lives by effectively repairing the damaged cartilage in the knee.

About ChondroCelect ChondroCelect for cartilage regeneration in the knee is an implantation suspension of characterized viable autologous (from the patient her- or himself) cartilage cells. The product is administered to patients in an autologous chondrocyte implantation procedure known as Characterized Chondrocyte Implantation (CCI), a surgical procedure to treat cartilage defects, in conjunction with debridement (preparation of the defect bed), a physical seal of the lesion (placement of a biological membrane, preferentially a collagen membrane) and rehabilitation.

Cartilage defects of the knee are very common and the spontaneous healing capacity of cartilage is limited. Currently, roughly 2 million cases of articular cartilage defects of the knee are diagnosed worldwide every year. TiGenix estimates that in Europe and the United States around 130,000 patients are eligible for treatment with cartilage regeneration products such as ChondroCelect.

ChondroCelect is the first cell-based product to successfully complete the entire development track from research to clinical development, and was approved by the European Medicines Agency as an Advanced Medicinal Therapy Product in October 2009. ChondroCelect is to date the only EMA approved cartilage repair therapy, and is commercially available in Belgium, the Netherlands, Luxemburg, Germany, the United Kingdom, Finland, and Spain.

About TiGenixTiGenix NV (EURONEXT:TIG) is a leading European cell therapy company with a marketed cell therapy product for cartilage repair, ChondroCelect, and a strong pipeline with clinical stage allogeneic adult stem cell programs for the treatment of autoimmune and inflammatory diseases. TiGenix is based out of Leuven (Belgium) and has operations in Madrid (Spain), and Sittard-Geleen (the Netherlands). For more information please visit http://www.tigenix.com.

Forward-looking informationThis document may contain forward-looking statements and estimates with respect to the anticipated future performance of TiGenix and the market in which it operates. Certain of these statements, forecasts and estimates can be recognized by the use of words such as, without limitation, "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will" and "continue" and similar expressions. They include all matters that are not historical facts. Such statements, forecasts and estimates are based on various assumptions and assessments of known and unknown risks, uncertainties and other factors, which were deemed reasonable when made but may or may not prove to be correct. Actual events are difficult to predict and may depend upon factors that are beyond TiGenix' control. Therefore, actual results, the financial condition, performance or achievements of TiGenix, or industry results, may turn out to be materially different from any future results, performance or achievements expressed or implied by such statements, forecasts and estimates. Given these uncertainties, no representations are made as to the accuracy or fairness of such forward-looking statements, forecasts and estimates. Furthermore, forward-looking statements, forecasts and estimates only speak as of the date of the publication of this document. TiGenix disclaims any obligation to update any such forward-looking statement, forecast or estimates to reflect any change in TiGenix' expectations with regard thereto, or any change in events, conditions or circumstances on which any such statement, forecast or estimate is based, except to the extent required by Belgian law.

Continued here:
TiGenix Signs Up 4th Major Hospital in the Netherlands for Innovative Cartilage Repair Therapy

Recommendation and review posted by simmons

Newswise Georgia Tech researchers have created a wireless, musical glove that may improve sensation and motor skills for people with paralyzing spinal cord injury (SCI).

The gadget was successfully used by individuals with limited feeling or movement in their hands due to tetraplegia. These individuals had sustained their injury more than a year before the study, a time frame when most rehab patients see very little improvement for the remainder of their lives. Remarkably, the device was primarily used while the participants were going about their daily routines.

The device is called Mobile Music Touch (MMT). The glove, which looks like a workout glove with a small box on the back, is used with a piano keyboard and vibrates a persons fingers to indicate which keys to play. While learning to play the instrument, several people with SCI experienced improved sensation in their fingers.

Researchers at Georgia Tech and Atlantas Shepherd Center recently completed a study focusing on people with weakness and sensory loss due to SCI.

After our preliminary work in 2011, we suspected that the glove would have positive results for people with SCI, said Ph.D. graduate Tanya Markow, the projects leader. But we were surprised by how much improvement they made in our study. For example, after using the glove, some participants were able to feel the texture of their bed sheets and clothes for the first time since their injury.

Markow worked with individuals with SCI who had limited feeling or movement in their hands. Each suffered a spinal injury more than a year prior to the study. The eight-week project required study participants to practice playing the piano for 30 minutes, three times a week. Half used the MMT glove to practice; half did not.

The MMT system works with a computer, MP3 player or smart phone. A song, such as Ode to Joy, is programmed into a device, which is wirelessly linked to the glove. As the musical notes are illuminated on the correct keys on the piano keyboard, the gadget sends vibrations to tap the corresponding fingers. The participants play along, gradually memorizing the keys and learning additional songs.

However, these active learning sessions with MMT were not the primary focus of the study. The participants also wore the glove at home for two hours a day, five days a week, feeling only the vibration (and not playing the piano). Previous studies showed that wearing the MMT system passively in this manner helped participants learn songs faster and retain them better. The researchers hoped that the passive wearing of the device would also have rehabilitative effects.

At the end of the study, participants performed a variety of common grasping and sensation tests to measure their improvement. Those who used the MMT system performed significantly better than those who just learned the piano normally.

Some people were able to pick up objects more easily, said Markow. Another said he could immediately feel the heat from a cup of coffee, rather than after a delay.

See original here:
Musical Glove Improves Sensation, Mobility for People with Spinal Cord Injury

Recommendation and review posted by sam

ALAMEDA, Calif.--(BUSINESS WIRE)--

BioTime, Inc. (NYSE MKT: BTX), a biotechnology company that develops and markets products in the field of regenerative medicine, today announced the signing of an exclusive sublicense agreement and a supply agreement with Jade Therapeutics, LLC, a developer of an ophthalmological therapeutic sustained-release drug delivery platform. Under the agreements, BioTime will provide Jade with clinical-grade HyStem hydrogels and certain patented technology for use by Jade Therapeutics in the development of new pharmaceutical products for ophthalmologic use. Jade plans to utilize the hydrogels to facilitate time-release topical delivery of recombinant human growth hormone to help heal lesions on the ocular surface. Jade Therapeutics will retain rights to market their product upon completion of development and obtaining marketing approval. Financial terms of the transaction were not disclosed.

William P. Tew, Ph.D., BioTimes Chief Commercialization Officer, stated that Numerous published scientific reports have established the efficacy of HyStem to facilitate cell transplantation in animal models, and we currently plan on a near-term approval to market one HyStem-related product, ReneviaTM, in the EU for reconstructive and cosmetic surgery. We believe our HyStem technology may also be useful as a device for the slow, timed release of therapeutic agents such as those being developed by Jade Therapeutics, as well as for the controlled release of proteins secreted from BioTimes stem cell lines.

The HyStem product line has potential utility in a wide array of human therapeutic products, said Michael West, Ph.D., BioTimes CEO. We intend to seek additional industry partners for applications that are not core to our own therapeutic product development.

BioTime's HyStem hydrogels are proprietary biocompatible hydrogels that mimic the human extracellular matrix (ECM), a web of molecules surrounding cells that is essential to cellular function. When cells lacking the ECM (or an ECM substitute) are introduced into the body, they typically die or fail to function correctly after transplantation. BioTime's HyStem hydrogels are currently being used by researchers at a number of leading medical schools in studies of stem cell therapies for facilitating wound healing and for the treatment of ischemic stroke, brain cancer, vocal fold scarring, and cardiac infarct.

About Jade Therapeutics

Jade Therapeutics, LLC, a privately-held company headquartered in Park City, Utah, focuses on the development of locally administered, sustained-release therapeutics that improve corneal healing following damage from disease or injury, thus improving visual function and quality of life. The Companys initial product is designed to deliver recombinant human growth hormone, a well characterized biologic that has already been demonstrated to have significant healing properties. Jade recently secured a prestigious Utah Science Technology and Research (USTAR) grant to continue to conduct preclinical and market research and is in negotiation with several prominent academic and military affiliates to further product development. Examples of ocular disorders addressed by the Companys technology includes persistent corneal epithelial defects and corneal damage due to dry eye disease.

About BioTime, Inc.

BioTime, headquartered in Alameda, California, is a biotechnology company focused on regenerative medicine and blood plasma volume expanders. Its broad platform of stem cell technologies is enhanced through subsidiaries focused on specific fields of application. BioTime develops and markets research products in the field of stem cells and regenerative medicine, including a wide array of proprietary ACTCellerateTM cell lines, HyStem hydrogels, culture media, and differentiation kits. BioTime is developing ReneviaTM (formerly known as HyStem-Rx), a biocompatible, implantable hyaluronan and collagen-based matrix for cell delivery in human clinical applications. BioTime's therapeutic product development strategy is pursued through subsidiaries that focus on specific organ systems and related diseases for which there is a high unmet medical need. BioTime's majority-owned subsidiary Cell Cure Neurosciences, Ltd. is developing therapeutic products derived from stem cells for the treatment of retinal and neural degenerative diseases. BioTime's subsidiary OrthoCyte Corporation is developing therapeutic applications of stem cells to treat orthopedic diseases and injuries. Another subsidiary, OncoCyte Corporation, focuses on the diagnostic and therapeutic applications of stem cell technology in cancer, including the diagnostic product PanC-DxTM currently being developed for the detection of cancer in blood samples. ReCyte Therapeutics, Inc. is developing applications of BioTime's proprietary induced pluripotent stem cell technology to reverse the developmental aging of human cells to treat cardiovascular and blood cell diseases. BioTime's subsidiary LifeMap Sciences, Inc. markets GeneCards, the leading human gene database, and is developing an integrated database suite to complement GeneCards that will also include the LifeMapTM database of embryonic development, stem cell research, and regenerative medicine, and MalaCards, the human disease database. LifeMap will also market BioTime research products. BioTime's lead product, Hextend, is a blood plasma volume expander manufactured and distributed in the U.S. by Hospira, Inc. and in South Korea by CJ CheilJedang Corporation under exclusive licensing agreements. Additional information about BioTime can be found on the web at http://www.biotimeinc.com.

Forward-Looking Statements

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BioTime Signs Agreements with Jade Therapeutics for Ophthalmological Drug Delivery Applications of HyStem® Technology

Recommendation and review posted by sam