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Article

AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 May 2008

Franich Nicholas R, Fitzsimons Helen L, Fong Dahna M, Klugmann Matthias, During Matthew J, Young Deborah

Abstract excerpt

We report the characterization of a new rapid-onset model of Huntington's disease (HD) generated by adeno-associated virus (AAV) vector-mediated gene transfer of N-terminal huntingtin (htt) constructs into the rat striatum. Expression of exon 1 of mutant htt containing 70 CAG repeats rapidly led to neuropathological features associated with HD. In addition, we report novel data relating to neuronal transduction...

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Source

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Identifiers

PMID: 18388917
DOI: 10.1038/mt.2008.50
PMCID: PMC3793641

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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