Reversal of molecular pathology by RNA-targeting Cas9 in a myotonic dystrophy mouse model
Ranjan Batra, David A. Nelles, Florian Krach, James D. Thomas, Lukasz Snjader, Steven M. Blue, Stefan Aigner, Maurice S. Swanson, Gene Wei-Ming Yeo
RNA-targeting Cas9 reverses molecular pathology in a myotonic dystrophy mouse model by eliminating toxic CUG RNA and restoring normal cellular functions.
bioRxiv · Sep 4, 2017 · PreprintMolecular Biology