Genetic therapy reverses nervous system damage in animal model of inherited hum...( g in the bloodstream the researchers i...)

Genetic therapy reverses nervous system damage in animal model of inherited human disease

g in the bloodstream, the researchers injected the virus directly into the brain.

"The brain of the cat is much closer in size to the human infant brain compared to mice. Thus we think that it may be possible to achieve similar results in humans with as few as 20 to 30 injections in each of the two hemispheres of the brain," Wolfe said. "We believe, however, we can further limit the number of injections by the use of strong promoters that could increase the amount of enzyme that comes from corrected cells."

Although encouraged by their findings, the researchers note that any clinical trials in humans might be years in the future.

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Contact: Greg Lester
glester@pobox.upenn.edu
215-573-6604
University of Pennsylvania
9-Mar-2005

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