Adeno-associated virus-mediated PKP2 gene therapy confers robust exercise tolerance in a murine model of arrhythmogenic cardiomyopathy.
作者信息
Cerrone Marina, Boyce Sarah, Zhang Mingliang, Gencarelli Manuela, Delmar Mario
机构信息
"Leon Charney" Division of Cardiology, NYU Grossmann School of Medicine, New York, New York.
Windland Smith Rice Sudden Death Genomics Laboratory, Department of Molecular Pharmacology and Experimental Therapeutics, Mayo Clinic, Rochester, Minnesota.
出版信息
Heart Rhythm. 2025 May 10. doi: 10.1016/j.hrthm.2025.05.007.
Abstract
摘要
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本文引用的文献
1
Plakophilin 2 gene therapy prevents and rescues arrhythmogenic right ventricular cardiomyopathy in a mouse model harboring patient genetics.桥粒斑蛋白 2 基因治疗可预防和挽救携带患者遗传基因的小鼠模型中的心律失常性右室心肌病。
Nat Cardiovasc Res. 2023 Dec;2(12):1246-1261. doi: 10.1038/s44161-023-00370-3. Epub 2023 Dec 7.
2
Therapeutic efficacy of AAV-mediated restoration of PKP2 in arrhythmogenic cardiomyopathy.腺相关病毒介导的桥粒斑蛋白2恢复在致心律失常性心肌病中的治疗效果
Nat Cardiovasc Res. 2023;2(12):1262-1276. doi: 10.1038/s44161-023-00378-9. Epub 2023 Dec 7.
3
AAV9:PKP2 improves heart function and survival in a Pkp2-deficient mouse model of arrhythmogenic right ventricular cardiomyopathy.腺相关病毒9型：PKP2改善致心律失常性右室心肌病Pkp2基因缺陷小鼠模型的心脏功能并提高生存率。
Commun Med (Lond). 2024 Mar 18;4(1):38. doi: 10.1038/s43856-024-00450-w.
4
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Circ Genom Precis Med. 2024 Feb;17(1):e004305. doi: 10.1161/CIRCGEN.123.004305. Epub 2024 Jan 30.
5
Role of plakophilin-2 expression on exercise-related progression of arrhythmogenic right ventricular cardiomyopathy: a translational study.桥粒斑蛋白-2 表达在运动相关致心律失常性右室心肌病进展中的作用：一项转化研究。
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