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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Bibliografske podrobnosti
izdano v: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
Jezik:Inglês
Izdano: Nature Publishing Group 2014
Teme:
Original Article
Online dostop: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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