USING gene therapy to switch off genes instead of adding new ones could slow down or prevent the fatal brain disorder Huntington’s disease. The method, which exploits a mechanism called RNA interference, might also help treat a wide range of other inherited diseases.
“When I first heard of this work, it just took my breath away,” says Nancy Wexler of Columbia University Medical School, who is president of the Hereditary Disease Foundation in New York. Though the gene-silencing technique has yet to be tried in people, she says it is the most promising potential treatment so far for Huntington’s.
It…
