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, Borja Vilaplana‐Martí, Sergi César, Antonio de Molina-Iracheta, Miguel Sena‐Esteves, Georgia Sarquella‐Brugada, 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.
bioRxiv · Feb 13, 2025 · PreprintBioengineering