Primate Model Carrying LMNA Mutation Develops Dilated Cardiomyopathy

Xiang Luo, Hao Jia, Fang Wang,Han Mo,Yu Kang,Ningning Zhang,Lu Zhao,Lizhu Xu, Zhengsheng Yang,Qiaoyan Yang,Yuan Chang,Shulin Li, Ning Bian,Xiumeng Hua,Hao Cui, Yu Cao,Chu Chu, Yuqiang Zeng,Xinglong Chen, Zhigang Chen

JACC: Basic to Translational Science(2024)

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摘要
•For clinical findings, patients with LMD had an earlier stage of disease progression when receiving heart transplantation, and the degree of impairment to heart structure and function was relatively mild.•For the primate model, CRISPR-mediated adenine base editing constructed the first gene-edited primate model of DCM.•The primate model was highly similar to patients with LMD in terms of symptoms, disease progression, histopathology, and electrophysiological and transcriptional features.•Arrhythmia events in patients with LMD and the primate model predated impairment of heart structure and function.
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关键词
CRISPR-mediated adenine base editing,dilated cardiomyopathy,lamin A/ C,primate model
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