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Category Archives: Gene Medicine

Rice University, IBM Partner to Bring First Blue Gene Supercomputer to Texas

HOUSTON, March 30, 2012 /PRNewswire/ --Rice University and IBM (NYSE: IBM) today announced a partnership to build the first award-winning IBM Blue Gene supercomputer in Texas. Rice also announced a related collaboration agreement with the University of Sao Paulo (USP) in Brazil to initiate the shared administration and use of the Blue Gene supercomputer, which allows both institutions to share the benefits of the new computing resource.

(Logo: http://photos.prnewswire.com/prnh/20090416/IBMLOGO )

Rice faculty will use the Blue Gene to further their own research and to collaborate with academic and industry partners on a broad range of science and engineering questions related to energy, geophysics, basic life sciences, cancer research, personalized medicine and more.

The collaborative agreement securing Brazil's share of time on Rice's Blue Gene was signed in Sao Paulo March 27 by a delegation that included Rice President David Leebron and USP President Joao Grandino Rodas. Leebron is traveling with a delegation led by Houston Mayor Annise Parker. The delegation includes Rice Provost George McLendon, Greater Houston Partnership (GHP) President and CEO Jeff Moseley and other GHP members.

"Collaboration and partnership have a unique place in Rice's history as a pre-eminent research university, and it is fitting that Rice begins its second century with two innovative partnerships that highlight the university's commitments to expanding our international reach, strengthening our research and building stronger ties with our home city," Leebron said.

USP is Brazil's largest institution of higher education and research, and Rodas said the agreement represents an important bond between Rice and USP. "The joint utilization of the supercomputer by Rice University and USP, much more than a simple sharing of high-tech equipment, means the strength of an effective partnership between both universities," he said.

Mayor Parker, a 1978 Rice alumna, said, "When I was at Rice, it looked inward. Today it looks outward through this agreement. It strengthens not only Rice University but also the city of Houston."

Rice's new Blue Gene supercomputer, which has yet to be named, is slated to become operational in May. It is based on IBM's POWER processor technology, which was developed in part at the company's Austin, Texas labs. Rice and IBM shared the cost of the system.

"High-performance computers like the IBM Blue Gene/P are critical in virtually every discipline of science and engineering, and we are grateful for IBM's help in bringing this resource to Rice," McLendon said. "For individual faculty, the supercomputer will open the door to new areas of research. The Blue Gene also opens doors for Rice as the university seeks to establish institutional relationships both in our home city and with critical international partners like USP."

Unlike the typical desktop or laptop computer, which have a single microprocessor, supercomputers typically contain thousands of processors. This makes them ideal for scientists who study complex problems, because jobs can be divided among all the processors and run in a matter of seconds rather than weeks or months. Supercomputers are used to simulate things that cannot be reproduced in a laboratory -- like Earth's climate or the collision of galaxies -- and to examine vast databases like those used to map underground oil reservoirs or to develop personalized medical treatments.

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Rice University, IBM Partner to Bring First Blue Gene Supercomputer to Texas

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Single Gene Could Play Role in Obesity

(Ivanhoe Newswire)-- A ravenous appetite may be what causes many overweight people to be obese. Researchers have found a mutation in a single gene is responsible for the inability of neurons to effectively send out appetite suppressing signals from the body to the correct area of the brain.

A study suggests that there might be a way to stimulate expression of that gene to treat obesity caused by uncontrolled eating.

Researchers found that a mutation in the brain-derived neurotrophic factor (Bdnf) gene in mice does not allow brain neurons to adequately pass leptin and insulin chemical signals through the brain. In humans, these hormones are designed to "tell" the body to stop eating. But if the signals fail to reach correct locations in the hypothalamus, the area in the brain that signals satiety, eating continues.

"This is the first time protein synthesis in dendrites, tree-like extensions of neurons, has been found to be critical for control of weight," Baoji Xu, Ph.D., study's senior investigator, an associate professor of pharmacology and physiology at Georgetown, was quoted as saying.

"This discovery may open up novel strategies to help the brain control body weight," he said.

Xu has long investigated the Bdnf gene. He has found that the gene produces a growth factor that controls communication between neurons.

Xu also found that the mice with the same Bdnf mutation grew to be severely obese.

Other researchers began to look at the Bdnf gene in humans, and large-scale genome-wide association studies showed Bdnf gene variants are, were also linked to obesity.

However, until this study, no one has been able to describe exactly how BDNF controls body weight.

Xu's data shows that both leptin and insulin stimulate synthesis of BDNF in neuronal dendrites in order to move their chemical message from one neuron to another through synapses. The intent is to keep the leptin and insulin chemical signals moving along the neuronal pathway to the correct brain locations, where the hormones will turn on a program that suppresses appetite.

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Gene genie out of the bottle

Worried about cybertheft? Thats so 20th century. Tonights NOVA (8 p.m., PBS) explores a brave new world thats now upon us: the era of the genetic code and the powerful and potentially dangerous information it can reveal.

Our genetic blueprints can become the starting point for aggressive preventive medicine, allowing doctors to understand a cancers genome and learn how to attack it. We may soon be able to pinpoint medications to particular genes and diseases, methods that will make the medical carpet bombing of chemotherapy seem crude by comparison. If were going to switch to prevention, then your own genome sequence may be one of the most critical tools you could imagine, says Francis Collins, director of the National Institutes of Health.

But knowledge of peoples genetic makeup and defects could lead to a kind of DNA-based discrimination. Will insurance companies and potential employers start to shun people whose DNA pinpoints expensive maladies? Will it lead to a master-race-breeding mentality, affecting how individuals choose a potential spouse? Will political candidates be compelled to reveal their genetic code the way they now must release their tax statements?

Sequencing an individuals human genome used to be the stuff of science fiction. And until quite recently its prohibitive price tag (upward of $350,000) kept it out of reach. Now companies can do it for less than $1,000 and the price continues to fall, bringing this powerful information within reach of those who could help you, or spy upon your innermost vulnerabilities.

Whitechapel (9 p.m., BBC America), the stylish British detective series where criminals always hearken back to the murderous misdeeds of history, returns for a third season.

Tonights other highlights

An expired license trips up a trip down the aisle on the season finale of Whitney (7 p.m., NBC).

Two hours of live performances on American Idol (7 p.m., Fox).

Americas Next Top Model: British Invasion (8 p.m., CW) travels to Canada. Banality without borders.

Woodys career path could turn on the determination of a victims cause of death on Psych (9 p.m., USA).

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Gene genie out of the bottle

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Innovaro Announces Completion of Licensing Agreement between Inven2 and Oxford Gene Technology

TAMPA, Fla.--(BUSINESS WIRE)--

Innovaro, Inc (NYSE Amex: INV), The Innovation Solutions Company, is pleased to announce that its client Inven2, the technology transfer office at Oslo University Hospital and University of Oslo, has entered into an exclusive licensing agreement with Oxford Gene Technology (OGT) for 12 highly promising colorectal cancer tissue biomarkers through Innovaros Pharmalicensing Partnering Search & Profiling division.

The exclusive license allows OGT to commercialize any resulting test developed using these biomarkers and to sublicense the markers to other parties. The DNA methylation biomarkers were developed in the laboratory of Professor Ragnhild A. Lothe, in the department of Cancer Prevention, the Norwegian Radium Hospital, part of the Oslo University Hospital.

OGT has validated the results obtained in Professor Lothes laboratory showing sensitivity of 93% and specificity of 90% when using tissue biopsies. Further work investigating the efficacy of these biomarkers in blood and fecal samples is ongoing.

We fully support the collaboration with Oxford Gene Technology to develop a new method of detecting colorectal cancer using these biomarkers. This agreement demonstrates the importance of industry and academic collaboration in turning scientific excellence into products that address medical needs, commented Benedicte Bakke, Business Development Manager at Inven2. The Innovaro Pharmalicensing Profiling service was able to bring this high quality potential partner to our attention that we may not otherwise have met.

This licensing agreement gives OGT exclusive access to genetic markers which are associated with colorectal cancer, stated Mike Evans, CEO of OGT. We believe that developing tests that include these genetic markers will permit the earlier identification of patients at risk of this disease and allow for more timely diagnosis and clinical interventions. He added, The higher specificity of this new panel of markers could prove a more robust screening tool than the tests currently used, while eventually lowering overall costs, which would be of significant benefit for both patients and the clinicians using them.

We are delighted that Inven2 was able to identify Oxford Gene Technology as an appropriate candidate partner, using Innovaro Pharmalicensings Profiling service, clearly leading to this important licensing agreement, confirmed Mark McBride, Senior VP Fulfilment Services, Innovaro, Inc. This agreement also demonstrates the effectiveness of Innovaros Pharmalicensing Profiling service for the life sciences alongside our already well recognized proficiency in Partnering Search services.

About Inven2

Inven2 is the Technology Transfer Office for the University of Oslo and Oslo University Hospital, Norway's largest and leading university and hospital representing pioneering research. Inven2 is the largest contributor in Norway within the field of commercialization of research across Life Sciences. For more information on Inven2, please visit its website at http://www.inven2.com.

About Oxford Gene Technology

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Innovaro Announces Completion of Licensing Agreement between Inven2 and Oxford Gene Technology

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New gene therapy approach developed for red blood cell disorders

ScienceDaily (Mar. 27, 2012) A team of researchers led by scientists at Weill Cornell Medical College has designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment.

This study's findings, published in PLoS ONE, represents a new approach to treating these related, and serious, red blood cells disorders, say the investigators.

"This gene therapy technique has the potential to cure many patients, especially if we prescreen them to predict their response using just a few of their cells in a test tube," says the study's lead investigator, Dr. Stefano Rivella, Ph.D., an associate professor of genetic medicine at Weill Cornell Medical College. He led a team of 17 researchers in three countries.

Dr. Rivella says this is the first time investigators have been able to correlate the outcome of transferring a healthy beta-globin gene into diseased cells with increased production of normal hemoglobin -- which has long been a barrier to effective treatment of these disease.

So far, only one patient in France has been treated with gene therapy for beta thalassemia, and Dr. Rivella and his colleagues believe the new treatment they developed will be a significant improvement. No known patient has received gene therapy yet to treat sickle cell anemia.

A Fresh Approach to Gene Therapy

Beta-thalassemia is an inherited disease caused by defects in the beta-globin gene. This gene produces an essential part of the hemoglobin protein, which, in the form of red blood cells, carries life-sustaining oxygen throughout the body.

The new gene transfer technique developed by Dr. Rivella and his colleagues ensures that the beta-globin gene that is delivered will be active, and that it will also provide more curative beta-globin protein. "Since the defect in thalassemia is lack of production of beta-globin protein in red blood cells, this is very important," Dr. Rivella says.

The researchers achieved this advance by hooking an "ankyrin insulator" to the beta-globin gene that is carried by a lentivirus vector. During the gene transfer, this vector would be inserted into bone marrow stem cells taken from patients, and then delivered back via a bone marrow transplant. The stem cells would then produce healthy beta-globin protein and hemoglobin.

This ankyrin insulator achieves two goals. First, it protects delivery of the normal beta-globin gene. "In many gene therapy applications, a curative gene is introduced into the cells of patients in an indiscriminate fashion," Dr. Rivella explains. "The gene lands randomly in the genome of the patient, but where it lands is very important because not all regions of the genome are the same." For example, some therapeutic genes may land in an area of the genome that is normally silenced -- meaning the genes in this area are not expressed. "The role of ankyrin insulator is to create an active area in the genome where the new gene can work efficiently no matter where it lands," Dr. Rivella says. He adds that the small insulator used in his vector should eliminate the kind of side effects seen in the French patient treated with beta-thalassemia gene therapy.

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Sanofi-Regeneron drug lowers cholesterol up to 72 percent in study

By Bloomberg News

An experimental drug from Sanofi and Regeneron Pharmaceuticals Inc. lowered patients so-called bad cholesterol by as much as 72 percent on top of Lipitor in a company-funded study.

(Last year, French drug maker Sanofi bought Genzyme Corp. of Cambridge for $20.1 billion.)

The medicine, one in a class of drugs targeting the PCSK9 gene, reduced patients average LDL cholesterol levels to as little as 34 milligrams per deciliter after 12 weeks in the mid- stage study, presented today at the American College of Cardiology meeting in Chicago. Less than 100 mg/dL is considered optimal for LDL, according to the Mayo Clinic.

Its a wow study, said James McKenney, the trials lead investigator and chief executive officer of National Clinical Research, a Virginia-based company that runs studies. Instantly youre seeing all those patients you could not get to the level youd like for them to be at, now you have a medicine that unquestionably can.

About 25 percent of US adults 45 and over take cholesterol-lowering statins, according to the National Center for Health Statistics. Statins include pills such as Pfizer Inc.s Lipitor, the worlds best-selling medicine in 2010 with revenue of $10.7 billion. Almost a third of statin users dont get enough of a benefit, leaving them more vulnerable to heart disease and in need of additional therapy, McKenney said in a telephone interview.

The 183-patient trial, funded by Paris-based Sanofi and Tarrytown, N.Y.-based Regeneron, produced results reminiscent of the cholesterol-lowering advances achieved by statins two to three decades ago, McKenney said. Patients for whom statins work can see their cholesterol reduced by as much as 50 percent, lowering heart risks, he said.

Amgen Inc., in Thousand Oaks, Calif., New York-based Pfizer, and Whitehouse Station, N.J.-based Merck & Co. are also developing treatments targeting PCSK9, said Chris Raymond, an analyst with Robert W. Baird & Co. in Chicago. The gene creates a protein that disrupts the ability of liver cells to remove bad cholesterol from blood, enabling it to accumulate.

The study presented today evaluated five doses of Regeneron and Sanofis medicine, dubbed REGN727 or SAR236553, compared with a placebo. It found that 50 milligrams of the drug injected once every two weeks lowered LDL cholesterol by 40 percent, while 300 milligrams dosed every four weeks reduced LDL by 48 percent. The most-effective dose was 150 milligrams injected every two weeks, shown to lower LDL by 72 percent.

Patients took Lipitor at doses of 10, 20, or 40 milligrams for at least six weeks before starting the study, and still had LDL levels higher than 100 mg/dL. All groups continued taking Lipitor throughout the trial. Those on placebo -- Lipitor with no additional therapy -- had an LDL decline of 5 percent.

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