Article
The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1.
Nature biomedical engineering - 1 Feb 2021
Batra Ranjan, Nelles David A, Roth Daniela M, Krach Florian, Nutter Curtis A, Tadokoro Takahiro, Thomas James D, Sznajder Łukasz J, Blue Steven M, Gutierrez Haydee L, Liu Patrick, Aigner Stefan, Platoshyn Oleksandr, Miyanohara Atsushi, Marsala Martin, Swanson Maurice S, Yeo Gene W
Abstract excerpt
Myotonic dystrophy type I (DM1) is a multisystemic autosomal-dominant inherited human disorder that is caused by CTG microsatellite repeat expansions (MREs) in the 3' untranslated region of DMPK. Toxic RNAs expressed from such repetitive sequences can be eliminated using CRISPR-mediated RNA targeting, yet evidence of its in vivo efficacy and durability is lacking. Here, using adult and neonatal mouse models of...
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