Genome Editing and Pathological Cardiac Hypertrophy
Genome Editing and Pathological Cardiac Hypertrophy
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基因组编辑与病理性心脏肥大
DOI：
10.1007/978-981-19-5642-3_6
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发表时间：
2023
期刊：
影响因子：
--
通讯作者：
Takao Kato
中科院分区：
文献类型：
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作者：
Adachi Y;Ueda K;Ito K;Takimoto E;Komuro I.;Takao Kato
三种主要的基因组编辑工具，转录激活因子样效应核酸酶（TALEN），锌指核酸酶（ZFN）和成簇规则间隔短回文重复序列（CRISPR）系统，是用于遗传性心肌疾病研究和治疗的越来越重要的技术。生殖细胞基因组编辑和修改可以永久消除受影响家庭后代和下一代的单基因心血管疾病，尽管在伦理上存在争议。体细胞基因组编辑可能是治疗遗传性心肌病的一种有前途的方法，基因敲除对这些疾病有利，也可以治疗已经患病的人，尽管目前存在一些技术挑战。本章描述了基因组编辑在肥厚型心肌病以及其他心肌病的实验研究和治疗中的应用。
Three major genome editing tools, transcription activator-like effector nucleases (TALENs), zinc finger nucleases (ZFNs), and clustered regularly interspaced short palindromic repeat (CRISPR) systems, are increasingly important technologies used in the study and treatment of hereditary myocardial diseases. Germ cell genome editing and modification can permanently eliminate monogenic cardiovascular disease from the offspring of affected families and the next generation, although ethically controversial. Somatic genome editing may be a promising method for the treatment of hereditary cardiomyopathy various diseases for which gene knockout is favorable and can also treat people who are already ill, although there are currently some technical challenges. This chapter describes the application of genome editing in the experimental studies and treatment of hypertrophic cardiomyopathy as well as other cardiomyopathies.