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I. Hernández

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Open access Aug 2026

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

These results demonstrate that ABE can effectively target the LMNA c.745C>T mutation but also reveal the significant impact of bystander edits on cellular physiology, underscoring the necessity of precise editing technologies to ensure both efficacy and safety in future clinical translation.

M. Santafé, I. Hernández, D. Mazzeo et al. · bioRxiv · 0 citations