CRISPR-Cas9 mediated endogenous utrophin upregulation improves Duchenne Muscular Dystrophy
Simon Guiraud, Sumitava Dastidar, Fetta Mazed, Fatima Amor, Maëlle Ralu, Anne De Cian, Isabelle Richard, Giuseppe Ronzitti, Francesco Saverio Tedesco, Mario Amendola
This study uses CRISPR-Cas9 to disrupt miR binding sites and upregulate utrophin in DMD models, showing functional improvements and therapeutic potential.
bioRxiv · Apr 18, 2023 · PreprintMolecular Biology