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June 10, 2004
More On The Gene Therapy News
The journal Nature has the best writeup yet that I've seen on the University of Iowa's breakthrough. The title of the article is Cure Hoped For Huntington's Sufferers.
Here are excerpts from the article:
"Most gene therapy involves replacing a missing gene sequence. But in dominant disorders it is the mutant sequence itself that causes the problem, so any therapy needs to actively block a sequence rather than just replacing one.
To do this in the mice, Davidson’s team used a technique called RNA interference. The researchers isolated pieces of genetic material that bind to and block the mutant gene. They packaged these into stripped-down virus particles and injected them into the mice. The virus used was an adeno-associated virus that does not cause disease in mice or people.
After the injections, the proteins created by the mutant gene disappeared and the mice seemed to improve, the researchers told the annual meeting of the American Society of Gene Therapy in Minneapolis, Minnesota last week.
....
Davidson says she hopes that her technique will move quickly into clinical trials. “The data are very promising; we hope we will be able to use RNA interference as a therapy for dominant neurodegenerative diseases.”
Posted by Dave at June 10, 2004 04:26 PM
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