Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits

Huntington's disease (HD) is a neurodegenerative disorder caused by expansion of a CAG repeat in the huntingtin (Htt) gene. HD is autosomal dominant and, in theory, amenable to therapeutic RNA silencing. We introduced cholesterol-conjugated small interfering RNA duplexes (cc-siRNA) targeting hu...

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Main Authors: DiFiglia, M., Sena-Esteves, M., Chase, K., Sapp, E., Pfister, E., Sass, M., Yoder, J., Reeves, P., Pandey, R. K., Rajeev, K. G., Manoharan, M., Sah, D. W. Y., Zamore, P. D., Aronin, N.
格式: Artigo
語言:Inglês
出版: National Academy of Sciences 2007
主題:
Biological Sciences
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC2040405/
https://ncbi.nlm.nih.gov/pubmed/17940007
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0708285104
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