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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Bibliographic Details
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
Language:Inglês
Published: Public Library of Science 2014
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Research Article
Online Access: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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