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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2014
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4160241/
https://www.ncbi.nlm.nih.gov/pubmed/25207939
http://dx.doi.org/10.1371/journal.pone.0107434

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