基因编辑在未来心血管治疗中的应用

IF 8.6 1区 生物学 Q1 GENETICS & HEREDITY
Tomonori Tadokoro, Eric N Olson, Ning Liu
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引用次数: 0

摘要

尽管在药理学和外科干预方面取得了进展,但心血管疾病仍是全球发病率和死亡率的主要原因。CRISPR-Cas9基因组编辑技术的出现为纠正遗传性心血管疾病的遗传原因和调节与各种心脏病相关的致病性信号通路提供了有希望的方法。然而,在通往可能的临床应用的道路上,关于基因编辑组件的体内递送以及重要的安全性考虑的几个挑战仍有待解决。我们回顾了当前的基因编辑策略,它们在各种心血管疾病背景下的潜在治疗应用,以及它们各自的优点、局限性和监管考虑。这一领域的快速发展,加上利用基因编辑治疗心血管疾病的许多机会,预示着这一变革性技术的未来。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Gene Editing Applications as Future Cardiovascular Therapies.

Cardiovascular disease is the leading cause of global morbidity and mortality, despite advances in pharmacological and surgical interventions. The emergence of CRISPR-Cas9 genome editing technology offers promising approaches for correcting genetic causes of hereditary cardiovascular disorders and modulating pathogenic signaling pathways implicated in various heart diseases. However, several challenges with respect to in vivo delivery of gene editing components, as well as important safety considerations, remain to be addressed in the path toward possible clinical application. We review current gene editing strategies, their potential therapeutic applications in the context of a variety of cardiovascular disorders, and their respective merits, limitations, and regulatory considerations. The rapid advances in this field combined with the many opportunities for deploying gene editing therapies for cardiovascular disorders augur well for the future of this transformative technology.

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来源期刊
Annual review of genetics
Annual review of genetics 生物-遗传学
CiteScore
18.30
自引率
0.90%
发文量
17
期刊介绍: The Annual Review of Genetics, published since 1967, comprehensively covers significant advancements in genetics. It encompasses various areas such as biochemical, behavioral, cell, and developmental genetics, evolutionary and population genetics, chromosome structure and transmission, gene function and expression, mutation and repair, genomics, immunogenetics, and other topics related to the genetics of viruses, bacteria, fungi, plants, animals, and humans.
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