BioengineeringbioRxivPreprint
CRISPR/Cas9-mediated elimination of the LMNA c.745C>G pathogenic mutation enhances survival and cardiac function in LMNA-associated congenital muscular dystrophy
Déborah Gómez-Domínguez, Carolina Epifano, Iván Hernández…·Ignacío Pérez de Castro
This study uses CRISPR/Cas9 to target a specific mutation in LMNA-associated congenital muscular dystrophy, improving survival and cardiac function in mice.