Multiplexed genome editing by CRISPR-Un1Cas12f1 restores dystrophin expression in a mouse model of Duchenne muscular dystrophy.
The compact type V clustered regularly interspaced short palindromic repeats (CRISPR) nuclease Un1Cas12f1 is compatible with adeno-associated virus (AAV)-mediated genome editing, although the protospacer adjacent motif (PAM) requirements and capacity for multiplexed genome editing remain undefined. Here, we show that U...