Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients

Huntington disease (HD) is an inherited, fatal neurodegenerative disorder caused by a CAG repeat expansion in the huntingtin gene. The mutant protein causes neuronal dysfunction and degeneration resulting in motor dysfunction, cognitive decline, and psychiatric disturbances. Currently, there is no d...

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Main Authors: Skotte, Niels H., Southwell, Amber L., Østergaard, Michael E., Carroll, Jeffrey B., Warby, Simon C., Doty, Crystal N., Petoukhov, Eugenia, Vaid, Kuljeet, Kordasiewicz, Holly, Watt, Andrew T., Freier, Susan M., Hung, Gene, Seth, Punit P., Bennett, C. Frank, Swayze, Eric E., Hayden, Michael R.
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2014
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4160241/
https://ncbi.nlm.nih.gov/pubmed/25207939
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0107434
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