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


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… (More)
DOI: 10.1093/hmg/ddv219


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