Base editing therapy corrects long QT syndrome in mice

Gene therapy involving adenine base editing can correct a pathogenic variant in the Scn5a gene and alleviate arrhythmia phenotypes in a mouse model of long QT syndrome type 3 (LQTS3). “This finding provides fundamental support for the future application of gene base editing therapy for hereditary arrhythmias,” say study investigators Shuhong Ma, Xiangbo Chen, Yongming Wang and Feng Lan.

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