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August 01, 2004
Another Article On The Breakthrough
One of the best article,s yet, this one from The Independent, on the genetic therapy success out of the University of Iowa. Here's the highlights from the article:
In a groundbreaking study, scientists have shown for the first time that it is possible to stop a progressive brain disease in mice with a genetic technique known as RNA interference (RNAi).
Dr Davidson said that the findings, published in this month's Nature Medicine, were among the most important results of her career because they demonstrated the possibility of directly attacking the faulty gene responsible for Huntington's disease. "I'm extremely excited about the potential of RNAi and cautiously optimistic about its possible use in human medicine," Dr Davidson said.
"This is the first example of targeted gene silencing of a disease gene in the brains of live animals and it suggests that this approach may eventually be useful for human therapies," Dr Davidson said. "We have had success in tissue culture, but translating those ideas to animal models of disease has been a barrier. We seem to have broken through that barrier," she said.
Nancy Wexler of Columbia University in New York, a world authority on Huntington's disease, said RNAi offers the most promising potential treatment for the disease she has seen. "When I first heard of this work, it just took my breath away. Its everything you ever wanted to hear and more," said Professor Wexler, president of the Hereditary Disease Foundation in New York and a member of the team that originally discovered the Huntington's gene.
Phillip Sharp, a Nobel laureate from the Massachusetts Institute of Technology in Boston, said Dr Davidson's findings were "striking" because they demonstrated that RNAi may work for human patients suffering from a range of debilitating brain diseases. "It shows that, in the context of the biology, it's possible to do. This is a significant step, there's no doubt about it," Professor Sharp said.
The big question was whether this ability to silence genes could be "delivered" to all the cells of the body that needed it. Beverly Davidson's work shows that in a mammal the delivery of RNAi can work, even across the notoriously difficult biological barrier that protects the brain. She used a harmless carrier vehicle called adeno-associated virus to take the RNAi molecule into the diseased cells of the brain where the defective Huntington gene needed to be silenced. It worked.
"The broader science of RNAi is spectacular. Its just absolutely spectacular," Professor Sharp said.
"This is not hype. The biggest science prizes in the world will fall to RNAi."
Posted by Dave at August 1, 2004 09:45 PM
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