Article
Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Dec 2011
Carroll Jeffrey B, Warby Simon C, Southwell Amber L, Doty Crystal N, Greenlee Sarah, Skotte Niels, Hung Gene, Bennett C Frank, Freier Susan M, Hayden Michael R
Abstract excerpt
Huntington disease (HD) is an autosomal dominant neurodegenerative disorder caused by CAG-expansion in the huntingtin gene (HTT) that results in a toxic gain of function in the mutant huntingtin protein (mHTT). Reducing the expression of mHTT is therefore an attractive therapy for HD. However, wild-type HTT protein is essential for development and has critical roles in maintaining neuronal health. Therapies for...
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