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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...

詳細記述

保存先:
書誌詳細
出版年:Mol Ther
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: Nature Publishing Group 2014
主題:
オンライン・アクセス: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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