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Article

Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy.

Human molecular genetics - 1 Sept 2015

Bisset Darren R, Stepniak-Konieczna Ewa A, Zavaljevski Maja, Wei Jessica, Carter Gregory T, Weiss Michael D, Chamberlain Joel R

Abstract excerpt

RNA interference (RNAi) offers a promising therapeutic approach for dominant genetic disorders that involve gain-of-function mechanisms. One candidate disease for RNAi therapy application is myotonic dystrophy type 1 (DM1), which results from toxicity of a mutant mRNA. DM1 is caused by expansion of a CTG repeat in the 3' UTR of the DMPK gene. The expression of DMPK mRNA containing an expanded CUG repeat...

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Source

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Identifiers

PMID: 26082468
DOI: 10.1093/hmg/ddv219
PMCID: PMC4527493

Genes

DMPK

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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