Article
AAV9-mediated gene supplementation therapy prevents and rescues arrhythmogenic cardiomyopathy in Pnpla2-mutated mice.
Molecular therapy : the journal of the American Society of Gene Therapy - 3 Sept 2025
Zhang Xiulin, Wang Congrui, Chang Yuan, Jia Hao, Zhang Yue, Wang Yifan, Wang Weiteng, Han Han, Hu Yuhong, Shao Xijia, Wen Shuang, Tan Siyu, Zhang Ningning, Hua Xiumeng, Cui Hao, Chen Xiao, Song Jiangping
Abstract excerpt
Arrhythmogenic cardiomyopathy (ACM) is an inherited disorder involving ventricular arrhythmias, cardiac dysfunction, and fibrofatty myocardial replacement. Current treatments are largely palliative, with heart transplantation as the only definitive option for advanced ACM. Here, we show that, building upon our previous identification of a patient with a PNPLA2c.G245A/c.G245A mutation, we developed a murine model...
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- Animals
- Dependovirus
- Genetic Therapy
- Mice
- Lipase