心血管疾病的基因编辑疗法

IF 10.7 Q1 MEDICINE, RESEARCH & EXPERIMENTAL MedComm Pub Date : 2024-07-05 DOI:10.1002/mco2.639
Xinyu Wu, Jie Yang, Jiayao Zhang, Yuning Song
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引用次数: 0

摘要

近 30 年来,基因编辑工具的开发一直是生命科学研究的一个重要领域。这些工具已广泛应用于疾病检测和机理研究。进入新世纪,这些工具在应对各种科学挑战和通过基因编辑疗法拯救生命方面显示出了潜力,尤其是在防治心血管疾病(CVD)方面。基因编辑疗法的快速发展为心血管疾病患者带来了希望。基因编辑疗法治疗心血管疾病的进展是基因编辑技术在临床和基础研究中实际应用的综合体现,也是心血管疾病研究和治疗的稳步推进。本文概述了迄今为止开发的常用 DNA 靶向基因编辑工具,重点介绍了这些工具,尤其是成簇规则间隔短回文重复/CRISPR 相关基因(Cas)(CRISPR/Cas)系统在心血管疾病基因编辑疗法中的应用。报告还深入探讨了当前基因编辑疗法所面临的挑战和局限性,同时总结了正在进行的与心血管疾病相关的研究和临床试验。目的是通过总结基因编辑工具在心血管疾病领域的成功应用,促进相关研究人员的进一步探索。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Gene editing therapy for cardiovascular diseases

The development of gene editing tools has been a significant area of research in the life sciences for nearly 30 years. These tools have been widely utilized in disease detection and mechanism research. In the new century, they have shown potential in addressing various scientific challenges and saving lives through gene editing therapies, particularly in combating cardiovascular disease (CVD). The rapid advancement of gene editing therapies has provided optimism for CVD patients. The progress of gene editing therapy for CVDs is a comprehensive reflection of the practical implementation of gene editing technology in both clinical and basic research settings, as well as the steady advancement of research and treatment of CVDs. This article provides an overview of the commonly utilized DNA-targeted gene editing tools developed thus far, with a specific focus on the application of these tools, particularly the clustered regularly interspaced short palindromic repeat/CRISPR-associated genes (Cas) (CRISPR/Cas) system, in CVD gene editing therapy. It also delves into the challenges and limitations of current gene editing therapies, while summarizing ongoing research and clinical trials related to CVD. The aim is to facilitate further exploration by relevant researchers by summarizing the successful applications of gene editing tools in the field of CVD.

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CiteScore
6.70
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0.00%
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审稿时长
10 weeks
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