Recent advances in CRISPR-based genome editing technology and its applications in cardiovascular research.
Recent advances in CRISPR-based genome editing technology and its applications in cardiovascular research.
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基于CRISPR的基因组编辑技术及其在心血管研究中的应用
DOI：
10.1186/s40779-023-00447-x
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发表时间：
2023-03-10
影响因子：
21.1
通讯作者：
Yang, Xiao
中科院分区：
文献类型：
--
作者：
Li, Zhen-Hua;Wang, Jun;Xu, Jing-Ping;Wang, Jian;Yang, Xiao
关键词：
基因组编辑技术的快速发展为生命科学和医学领域带来了重大突破。近年来，基于成簇规则间隔短回文重复序列（CRISPR）的基因组编辑工具箱已经得到了极大的扩展，不仅有新兴的CRISPR相关蛋白（Cas）核酸酶，而且还有通过与不同效应物组合的新应用。最近，转座子相关的可编程RNA引导的基因组编辑系统已经被发现，为基因组编辑工具箱增加了无数潜在的新工具。基于CRISPR的基因组编辑技术也彻底改变了心血管研究。本文首先综述了新发现的Cas直向同源物、工程变体和新型基因组编辑系统的研究进展，然后讨论了CRISPR-Cas系统在精确基因组编辑（如碱基编辑和引物编辑）中的应用。我们还强调了使用基于CRISPR的基因组编辑技术在心血管研究方面取得的最新进展，包括心血管疾病（CVD）的转基因体外和动物模型的生成，以及在治疗不同类型的CVD中的应用。最后，讨论了基因组编辑技术的局限性和未来前景。
The rapid development of genome editing technology has brought major breakthroughs in the fields of life science and medicine. In recent years, the clustered regularly interspaced short palindromic repeats (CRISPR)-based genome editing toolbox has been greatly expanded, not only with emerging CRISPR-associated protein (Cas) nucleases, but also novel applications through combination with diverse effectors. Recently, transposon-associated programmable RNA-guided genome editing systems have been uncovered, adding myriads of potential new tools to the genome editing toolbox. CRISPR-based genome editing technology has also revolutionized cardiovascular research. Here we first summarize the advances involving newly identified Cas orthologs, engineered variants and novel genome editing systems, and then discuss the applications of the CRISPR-Cas systems in precise genome editing, such as base editing and prime editing. We also highlight recent progress in cardiovascular research using CRISPR-based genome editing technologies, including the generation of genetically modified in vitro and animal models of cardiovascular diseases (CVD) as well as the applications in treating different types of CVD. Finally, the current limitations and future prospects of genome editing technologies are discussed.
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