Article
Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts.
Human gene therapy - 1 Jul 2008
van Bilsen P H J, Jaspers L, Lombardi M S, Odekerken J C E, Burright E N, Kaemmerer W F
Abstract excerpt
Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder caused by the expression of mutant huntingtin protein (Htt). Suppression of Htt expression, using RNA interference, might be an effective therapy. However, if reduction of wild-type protein is not well tolerated in the brain, it may be necessary to suppress just the product of the mutant allele. We present a small interfering RNA...
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