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Rare Diseases Blog

By Mary Kugler, R.N., About.com Guide to Rare Diseases since 2000

Gene therapy for Lou Gehrig's disease

Wednesday August 13, 2003
Researchers at the Salk Institute and Johns Hopkins University have developed a gene therapy that fights the destruction caused by amyotrophic lateral sclerosis (Lou Gehrig's disease). Mice with the disorder who received the treatment lived 90% longer than those not treated, and more of the treated mice's muscle mass was preserved. The researchers plan to begin human tests of the gene therapy sometime in the next year or so.

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