Adenine base editing correction of LMNA c.745C>T (p.R249W) in congenital muscular dystrophy myoblasts improves cellular phenotype while revealing deleterious p.L248P bystander effects
Mario Santafe, Iván Hernández, D. Mazzeo…·Ignacío Pérez de Castro
This study uses adenine base editing to correct the LMNA c.745C>T mutation in myoblasts, showing improved cellular phenotype but also revealing harmful bystander mutations.