Base Editing Corrects Mutations for Cardiac Disease in Mice

Base Editing Corrects Mutations for Cardiac Disease in Mice

Researchers used base editing to correct a point mutation in the lamin A (LMNA) gene in mice, preventing skeletal and cardiac abnormalities and extending their lifespans.

Point mutations in the lamin A (LMNA) gene cause several human diseases, including congenital muscular dystrophy and premature aging, collectively known as laminopathies, which affect skeletal muscles and the heart.

While existing therapies can mitigate the symptoms of these conditions, there is no cure. Base-editing methods have recently emerged as tools for correcting point mutations without creating double-stranded breaks in DNA, making them a safer therapeutic strategy.

Base editing of LMNA-associated mutations that cause cardiac disease has remained unexplored.

Author's summary: Base editing corrects cardiac disease mutations in mice.

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The Scientist The Scientist — 2025-10-14

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