Journal article
RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model
Proceedings of the National Academy of Sciences - PNAS, Vol.102(16), pp.5820-5825
From the Cover
04/19/2005
DOI: 10.1073/pnas.0501507102
PMCID: PMC556303
PMID: 15811941
Abstract
Huntington's disease (HD) is a fatal, dominant neurogenetic disorder. HD results from polyglutamine repeat expansion (CAG codon, Q) in exon 1 of
HD
, conferring a toxic gain of function on the protein huntingtin (htt). Currently, no preventative treatment exists for HD. RNA interference (RNAi) has emerged as a potential therapeutic tool for treating dominant diseases by directly reducing disease gene expression. Here, we show that RNAi directed against mutant human htt reduced htt mRNA and protein expression in cell culture and in HD mouse brain. Importantly, htt gene silencing improved behavioral and neuropathological abnormalities associated with HD. Our data provide support for the further development of RNAi for HD therapy.
Details
- Title: Subtitle
- RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model
- Creators
- Scott Q. Harper - National Heart Lung and Blood InstitutePatrick D. Staber - National Heart Lung and Blood InstituteXiaohua He - National Heart Lung and Blood InstituteSteven L. Eliason - University of IowaInês H. Martins - University of IowaQinwen Mao - University of IowaLinda Yang - National Heart Lung and Blood InstituteRobert M. Kotin - National Heart Lung and Blood InstituteHenry L. Paulson - National Heart Lung and Blood InstituteBeverly L. Davidson - National Heart Lung and Blood Institute
- Resource Type
- Journal article
- Publication Details
- Proceedings of the National Academy of Sciences - PNAS, Vol.102(16), pp.5820-5825
- Publisher
- National Academy of Sciences
- Series
- From the Cover
- DOI
- 10.1073/pnas.0501507102
- PMID
- 15811941
- PMCID
- PMC556303
- ISSN
- 0027-8424
- eISSN
- 1091-6490
- Language
- English
- Date published
- 04/19/2005
- Academic Unit
- Anatomy and Cell Biology; Internal Medicine
- Record Identifier
- 9984622056902771
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