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...

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Bibliografske podrobnosti
izdano v:Hum Mol Genet
Main Authors: Bisset, Darren R., Stepniak-Konieczna, Ewa A., Zavaljevski, Maja, Wei, Jessica, Carter, Gregory T., Weiss, Michael D., Chamberlain, Joel R.
Format: Artigo
Jezik:Inglês
Izdano: Oxford University Press 2015
Teme:
Articles
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4527493/
https://ncbi.nlm.nih.gov/pubmed/26082468
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv219
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