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Article

Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Apr 2014

Dufour Brett D, Smith Catherine A, Clark Randall L, Walker Timothy R, McBride Jodi L

Abstract excerpt

Huntington's disease (HD) is a fatal neurological disorder caused by a CAG repeat expansion in the HTT gene, which encodes a mutant huntingtin protein (mHTT). The mutation confers a toxic gain of function on huntingtin, leading to widespread neurodegeneration and inclusion formation in many brain regions. Although the hallmark symptom of HD is hyperkinesia stemming from striatal degeneration, several other brain...

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Identifiers

PMID: 24390280
DOI: 10.1038/mt.2013.289
PMCID: PMC3982495

Genes

HTT

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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