Article
In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Dec 2014
Southwell Amber L, Skotte Niels H, Kordasiewicz Holly B, Østergaard Michael E, Watt Andrew T, Carroll Jeffrey B, Doty Crystal N, Villanueva Erika B, Petoukhov Eugenia, Vaid Kuljeet, Xie Yuanyun, Freier Susan M, Swayze Eric E, Seth Punit P, Bennett Clarence Frank, Hayden Michael R
Abstract excerpt
Huntington disease (HD) is a dominant, genetic neurodegenerative disease characterized by progressive loss of voluntary motor control, psychiatric disturbance, and cognitive decline, for which there is currently no disease-modifying therapy. HD is caused by the expansion of a CAG tract in the huntingtin (HTT) gene. The mutant HTT protein (muHTT) acquires toxic functions, and there is significant evidence that...
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