RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model.
basic_science · Level V
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- Record sourced from PubMed, PMID 15811941.
- Also identified by PMC identifier 556303.
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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.
Medical subject headings
- Gene Expression Regulation
- Huntington Disease
- Nerve Tissue Proteins
- Nuclear Proteins
- RNA Interference