Initially discovered in bacteria and archaea as adaptive immune strategies against invading nucleic acids, the clustered regularly interspaced short palindromic repeats-associated protein (CRISPR-Cas) system has now been repurposed as a practical tool for genome editing and other applications [1]. Owing to its flexibility, simplicity, efficiency and precision, the application of CRISPR systems in medicine has attracted significant attention and experienced rapid development. Currently, CRISPR systems have been used to screen novel drug targets [2], identify pathogenic genes [3], establish disease research modes [4], develop therapies against various diseases [5, 6] and so forth.

中文翻译：

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CRISPR在医学中的应用和挑战。

最初在细菌和古细菌中发现它是一种针对入侵核酸的适应性免疫策略，这种簇状规则间隔的短回文重复相关蛋白（CRISPR-Cas）系统现已被重新用作基因组编辑和其他应用的实用工具[1]。由于其灵活性，简单性，效率和精确性，CRISPR系统在医学中的应用引起了广泛的关注并经历了快速的发展。目前，CRISPR系统已用于筛选新型药物靶标[2]，鉴定致病基因[3]，建立疾病研究模式[4]，开发针对各种疾病的疗法[5、6]等。