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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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書目詳細資料
發表在: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
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2014
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在線閱讀: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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