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Article

Focused ultrasound for targeted delivery of siRNA and efficient knockdown of Htt expression.

Journal of controlled release : official journal of the Controlled Release Society - 28 Oct 2012

Burgess Alison, Huang Yuexi, Querbes William, Sah Dinah W, Hynynen Kullervo

Abstract excerpt

RNA interference is a promising strategy for the treatment of Huntington's disease (HD) as it can specifically decrease the expression of the mutant Huntingtin protein (Htt). However, siRNA does not cross the blood-brain barrier and therefore delivery to the brain is limited to direct CNS deliver...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 22921802
DOI: 10.1016/j.jconrel.2012.08.012
PMCID: PMC4010143

Genes

FUS

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-22921802-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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