Emerging CRISPR Therapies for Precision Gene Editing and Modulation in the Cardiovascular Clinic

IF 3.1 3区医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Current Cardiology Reports Pub Date : 2024-09-17 DOI:10.1007/s11886-024-02125-3

Nicholas J. Legere, J. Travis Hinson

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Abstract

Purpose of Review

Outline the growing suite of novel genome editing tools powered by CRISPR-Cas9 technology that are rapidly advancing towards the clinic for the treatment of cardiovascular disorders.

Recent Findings

A diversity of new genome editors and modulators are being developed for therapies across myriad human diseases. Recent breakthroughs have improved the efficacy, safety, specificity, and delivery of CRISPR-mediated therapies that could impact heart disease in the next decade, though several challenges remain.

Summary

Many iterations of the original CRISPR system have been developed seeking to leverage its vast therapeutic potential. As examples, nuclease-free editing, precision single-nucleotide editing, gene expression regulation, and epigenomic modifications are now feasible with the current CRISPR-mediated suite of enzymes. These emerging tools will be indispensable for the development of novel cardiovascular therapeutics as demonstrated by recent successes in both basic research laboratories and pre-clinical models. Here, we provide an overview of current and emerging CRISPR-mediated technologies as they pertain to the cardiovascular system, highlighting successful implementations and future challenges.

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心血管临床中用于精准基因编辑和调控的新兴 CRISPR 疗法

综述目的概述由 CRISPR-Cas9 技术驱动的新型基因组编辑工具日益增多，这些工具正迅速走向临床，用于治疗心血管疾病。最近的研究结果目前正在开发多种新型基因组编辑器和调节器，用于治疗各种人类疾病。最近的突破提高了 CRISPR 介导疗法的疗效、安全性、特异性和给药方式，这些疗法可能在未来十年内对心脏病产生影响，但仍存在一些挑战。摘要原始 CRISPR 系统的多次迭代开发旨在利用其巨大的治疗潜力。举例来说，无核酸酶编辑、精确单核苷酸编辑、基因表达调控和表观基因组修饰现在都可以通过目前 CRISPR 介导的一系列酶来实现。最近在基础研究实验室和临床前模型中取得的成功表明，这些新兴工具将成为开发新型心血管治疗药物不可或缺的工具。在此，我们概述了与心血管系统有关的当前和新兴 CRISPR 介导技术，重点介绍了成功的应用和未来的挑战。

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来源期刊

Current Cardiology Reports CARDIAC & CARDIOVASCULAR SYSTEMS-

CiteScore

6.20

自引率

2.70%

发文量

209

期刊介绍： The aim of this journal is to provide timely perspectives from experts on current advances in cardiovascular medicine. We also seek to provide reviews that highlight the most important recently published papers selected from the wealth of available cardiovascular literature. We accomplish this aim by appointing key authorities in major subject areas across the discipline. Section editors select topics to be reviewed by leading experts who emphasize recent developments and highlight important papers published over the past year. An Editorial Board of internationally diverse members suggests topics of special interest to their country/region and ensures that topics are current and include emerging research. We also provide commentaries from well-known figures in the field.