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心血管疾病的基因编辑疗法

Gene editing therapy for cardiovascular diseases.

作者信息

Wu Xinyu, Yang Jie, Zhang Jiayao, Song Yuning

机构信息

State Key Laboratory for Diagnosis and Treatment of Severe Zoonotic Infectious Diseases Key Laboratory for Zoonosis Research of the Ministry of Education and College of Veterinary Medicine Jilin University Changchun China.

出版信息

MedComm (2020). 2024 Jul 5;5(7):e639. doi: 10.1002/mco2.639. eCollection 2024 Jul.

DOI:10.1002/mco2.639
PMID:38974714
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11224995/
Abstract

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.

摘要

近30年来,基因编辑工具的开发一直是生命科学领域的一个重要研究方向。这些工具已广泛应用于疾病检测和机制研究。在新世纪,它们通过基因编辑疗法在应对各种科学挑战和拯救生命方面展现出潜力,尤其是在对抗心血管疾病(CVD)方面。基因编辑疗法的迅速发展为CVD患者带来了希望。CVD基因编辑疗法的进展全面反映了基因编辑技术在临床和基础研究中的实际应用情况,以及CVD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,特别关注这些工具,尤其是成簇规律间隔短回文重复序列/CRISPR相关基因(Cas)(CRISPR/Cas)系统在CVD基因编辑疗法中的应用。它还深入探讨了当前基因编辑疗法的挑战和局限性,同时总结了与CVD相关的正在进行的研究和临床试验。目的是通过总结基因编辑工具在CVD领域的成功应用,促进相关研究人员的进一步探索。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d187/11224995/2a18ce87731e/MCO2-5-e639-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d187/11224995/1631e47f0692/MCO2-5-e639-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d187/11224995/2a18ce87731e/MCO2-5-e639-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d187/11224995/1631e47f0692/MCO2-5-e639-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d187/11224995/2a18ce87731e/MCO2-5-e639-g002.jpg

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