Kemaladewey et al. demonstrated in vivo systemic delivery of an adeno-associated virus (AAV) carrying CRISPR/Cas9 genome editing components in a mouse model of congenital muscular dystrophy type 1A (MDC1A) to correct a pathogenic SS mutation in LAMA2 pre-mRNA in order to include exon 2. Here, LAMA2 is linked to Congenital muscular dystrophy type 1A.