Phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons. (link.springer.com)

Authors: Rovina D … Maione AS Journal: Genome Medicine Published: 2026-07-31 DOI: https://doi.org/10.1186/s13073-026-01738-3