The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1

Ranjan Batra, David A. Nelles, Daniela Roth, Florian Krach, Curtis A. Nutter, Takahiro Tadokoro, James D. Thomas, Łukasz J. Sznajder, Steven M. Blue, Haydee L. Gutierrez, Patrick Liu, Stefan Aigner, Oleksandr Platoshyn, Atsushi Miyanohara, Martin Maršala, Maurice S. Swanson, G Yeo