Researchers used base editing to correct a point mutation in the lamin A (LMNA) gene in mice, preventing skeletal and cardiac abnormalities and extending mice lifespans.
Point mutations in the lamin A (LMNA) gene cause several human diseases, ranging from congenital muscular dystrophy to premature aging, collectively known as laminopathies, which affect skeletal muscles and the heart.
Base-editing methods have recently emerged as tools for correcting point mutations without creating double-stranded breaks in DNA, unlike other gene editing methods, leading many to consider base editing a safer therapeutic strategy.
Author's summary: Base editing corrects cardiac disease mutations in mice.