关键词: CRISPR/Cas cardiovascular disease gene therapy lipid nanoparticles

来  源:   DOI:10.1002/mco2.639   PDF(Pubmed)

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,基因编辑工具的发展一直是生命科学研究的重要领域。这些工具已广泛用于疾病检测和机制研究。在新世纪,它们在解决各种科学挑战和通过基因编辑疗法拯救生命方面显示出潜力,特别是在对抗心血管疾病(CVD)。基因编辑疗法的快速发展为CVD患者提供了乐观。CVD的基因编辑治疗进展是基因编辑技术在临床和基础研究环境中的实际实施的综合反映。以及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的DNA靶向基因编辑工具,特别关注这些工具的应用,特别是成簇的规则间隔短回文重复/CRISPR相关基因(Cas)(CRISPR/Cas)系统,在CVD基因编辑疗法中。它还深入研究了当前基因编辑疗法的挑战和局限性,同时总结正在进行的与CVD相关的研究和临床试验。目的是通过总结基因编辑工具在CVD领域的成功应用,以促进相关研究人员的进一步探索。
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