Article
Precise gene editing of pathogenic Lamin A mutations corrects cardiac disease.
Proceedings of the National Academy of Sciences of the United States of America - 28 Oct 2025
Caravia Xurde M, Hayashi Brian, Li Hui, Gan Peiheng, Alzhanov Damir, Tan Wei, Chen Kenian, McAnally John R, Xu Lin, Liu Ning, Olson Eric N
Abstract excerpt
Mutations in the Lamin A (LMNA) gene, which encodes the Lamin A and C proteins, cause severe human diseases collectively known as laminopathies. These conditions are often devastating and lack effective therapies. In this study, we developed precise base editing (BE) strategies targeting the human LMNA gene variants L35P and R249Q, which cause congenital muscular dystrophy (CMD) and dilated cardiomyopathy with...
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