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That study built on previous research by the Columbus-based team, which had successfully used gene therapy to treat rodents with the muscle-wasting disease muscular dystrophy. "We wanted to raise the bar and test a species closely related to humans," says neuroscientist Brian Kaspar, a co-leader of the study published in Science Translational Medicine. (Read "What Can Genetic Tests Tell...
...single gene injected into the monkeys was coded for the naturally occurring protein follistatin, which blocks the function of another protein called myostatin that hinders muscle growth. Past research in mice that were genetically engineered to have an extra copy of the follistatin-producing gene has shown that blocking myostatin, by increasing follistatin, causes muscles to bulk up fast. What Kaspar and his team found was that the same effect could be achieved simply by injecting genes - ferried aboard a small, non-disease-causing virus known as AAV, or adeno-associated virus - into the muscle. They further discovered that once...
...findings got headlines, not least because they immediately raised the possibility of power-hungry athletes someday using gene doping to improve performance - a technique that would be much harder to detect than using performance-enhancing drugs. And while myostatin-blocking drugs are not yet available, the pharmaceutical companies Amgen and Wyeth are currently experimenting with myostatin inhibitors, with encouraging early results, and it's clearly something that antidoping agencies around the world are concerned about. The World Anti-Doping Agency has banned gene doping, and included "agents modifying myostatin function(s)" on its list of prohibited substances...
Scientists do not yet know whether myostatin-related gene therapy will even work in humans. Given the financial and regulatory hurdles to launching a first-phase trial, it could take years and several million dollars before researchers could replicate their animal findings in people. But advances like the muscle trial in monkeys help attract funds - largely from advocacy groups like the Muscular Dystrophy Association and charitable organizations founded by patient families, as well as drug companies and the federal government - to a field that has until now been somewhat better known for its failures. In 2003, for instance, two French...
...despite gene therapy's public-image problem, scientists are optimistic. Many believe that over the next four to five years, they will be able to apply what they have learned from studying gene therapies for rare diseases to the treatment of more common ailments like epilepsy, arthritis and congestive heart failure. "[Gene therapy] still needs one killer app. One clear, unambiguous success," says Greely. "And then the money will flood...