Molecular BiologybioRxivPreprint
CRISPR-Cas9 mediated endogenous utrophin upregulation improves Duchenne Muscular Dystrophy
Simon Guiraud, Sumitava Dastidar, Fetta Mazed…·Mario Amendola
This study uses CRISPR-Cas9 to disrupt miR binding sites and upregulate utrophin in DMD models, showing functional improvements and therapeutic potential.