State Key Laboratory of Proteomics, Beijing Proteome Research Center, National Center for Protein Sciences, Beijing Institute of Lifeomics, Beijing, 100071, China.
Yaneng BIOScience (Shenzhen) Co., Ltd., Shenzhen, 518102, Guangdong, China.
Mil Med Res. 2023 Mar 10;10(1):12. doi: 10.1186/s40779-023-00447-x.
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.
基因组编辑技术的快速发展在生命科学和医学领域带来了重大突破。近年来，基于成簇规律间隔短回文重复序列（CRISPR）的基因组编辑工具包得到了极大扩展，不仅出现了新兴的 CRISPR 相关蛋白（Cas）核酸酶，而且通过与各种效应物结合还产生了新的应用。最近，还发现了转座子相关可编程 RNA 引导的基因组编辑系统，为基因组编辑工具包增添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。在这里，我们首先总结了新鉴定的 Cas 直系同源物、工程化变体和新型基因组编辑系统的进展，然后讨论了 CRISPR-Cas 系统在精确基因组编辑中的应用，例如碱基编辑和先导编辑。我们还重点介绍了基于 CRISPR 的基因组编辑技术在心血管研究中的最新进展，包括生成用于心血管疾病（CVD）的基因修饰体外和动物模型以及在治疗不同类型 CVD 中的应用。最后，讨论了基因组编辑技术的当前局限性和未来前景。
