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心力衰竭和心肌病的基因治疗。

Gene therapy for heart failure and cardiomyopathies.

机构信息

Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy.

Division of Cardiovascular Medicine, Department of Medicine, University of California, San Diego, San Diego, CA, United States.

出版信息

Rev Esp Cardiol (Engl Ed). 2023 Dec;76(12):1042-1054. doi: 10.1016/j.rec.2023.06.009. Epub 2023 Jul 26.

Abstract

Gene therapy strategies encompass a range of approaches, including gene replacement and gene editing. Gene replacement involves providing a functional copy of a modified gene, while gene editing allows for the correction of existing genetic mutations. Gene therapy has already received approval for treating genetic disorders like Leber's congenital amaurosis and spinal muscular atrophy. Currently, research is being conducted to explore its potential use in cardiology. This review aims to summarize the mechanisms behind different gene therapy strategies, the available delivery systems, the primary risks associated with gene therapy, ongoing clinical trials, and future targets, with a particular emphasis on cardiomyopathies.

摘要

基因治疗策略包括多种方法,包括基因替换和基因编辑。基因替换涉及提供修饰基因的功能副本,而基因编辑则允许纠正现有的遗传突变。基因治疗已经获得批准,可用于治疗莱伯先天性黑蒙和脊髓性肌萎缩症等遗传疾病。目前,正在进行研究以探索其在心内科的潜在用途。本综述旨在总结不同基因治疗策略的机制、可用的传递系统、基因治疗相关的主要风险、正在进行的临床试验以及未来的目标,特别关注心肌病。

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