LAMA1 and Congenital muscular dystrophy type 1A: In comparison, Kemaledawi et al. either used (i) a single rAAV vector expressing SadCas9-VP64 and one sgRNA or (ii) two separate AAVs, the first encoding SadCas9-VP64 and the second expressing three sgRNAs, to activate the Lama1 gene in a mouse model of congenital muscular dystrophy type 1A (MDC1A).