Impeding Transcription of Expanded Microsatellite Repeats by Deactivated Cas9 (Molecular Cell)

Pinto, Saxena, et al. demonstrate that deactivated Cas9 efficiently blocks transcription of the expanded microsatellite repeats causing diseases such as myotonic dystrophy types 1 and 2 and C9orf72-ALS/FTD, thereby reversing toxic downstream molecular and cellular effects of repeat expression in vitro and in vivo.