In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides

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 huntin...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: 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
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2014
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4429695/
https://ncbi.nlm.nih.gov/pubmed/25101598
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.153
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