Molecular BiologybioRxivPreprint
Reversal of molecular pathology by RNA-targeting Cas9 in a myotonic dystrophy mouse model
Ranjan Batra, David A. Nelles, Florian Krach…·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.
4 citations