Genome medicine2026ResearchNon-viral Gene Delivery
1. 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
Rovina D, Milasi S, Di Giuseppe G, Pioner Jm, Langione M, Giammarino L, Et Al.
Duchenne muscular dystrophy (DMD) is a X-linked disease affecting skeletal and cardiac muscle and is caused by mutations in the dystrophin gene (DMD). Patient-derived induced pluripotent stem cells (iPSCs) serve as reliable in vitro disease models. Their genetic correction by CRISPR/Cas9 allows the generation of…
- CRISPR/Cas9
- Duchenne muscular dystrophy
- Dystrophin
- cardiomyocytes, stromal cells, engineered heart tissue
- iPSC