Article
Personalized gene silencing therapeutics for Huntington disease.
Clinical genetics - 1 Jul 2014
Kay C, Skotte N H, Southwell A L, Hayden M R
Abstract excerpt
Gene silencing offers a novel therapeutic strategy for dominant genetic disorders. In specific diseases, selective silencing of only one copy of a gene may be advantageous over non-selective silencing of both copies. Huntington disease (HD) is an autosomal dominant disorder caused by an expanded CAG trinucleotide repeat in the Huntingtin gene (HTT). Silencing both expanded and normal copies of HTT may be...
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