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Article

Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Oct 2005

Rodriguez-Lebron Edgardo, Denovan-Wright Eileen M, Nash Kevin, Lewin Alfred S, Mandel Ronald J

Abstract excerpt

Huntington's disease (HD) is a fatal neurodegenerative disorder caused by the presence of an abnormally expanded polyglutamine domain in the N-terminus of huntingtin. We developed a recombinant adeno-associated viral serotype 5 (rAAV5) gene transfer strategy to posttranscriptionally suppress the...

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Identifiers

PMID: 16019264
DOI: 10.1016/j.ymthe.2005.05.006
PMCID: PMC2656966

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-16019264-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 2 Sept 2026.

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