When Lama1 was upregulated using CRISPR and a catalytically inactive Cas9 in a mouse model of congenital muscular dystrophy type 1A, apparent hindlimb paralysis, muscle fibrosis and nerve myelination defects were ameliorated in symptomatic mice.
- Dwi U. Kemaladewi
- Prabhpreet S. Bassi
- Ronald D. Cohn