Assistant Director - Animal Modeling Core; Assistant Research Professor - Department of Veterinary Pathobiology; and Comparative Medicine Program Faculty, University of Missouri - Columbia, Columbia, Missouri.
PhD candidate in the Department of Animal Sciences, University of Missouri - Columbia, Columbia, Missouri.
Mo Med. 2024 Mar-Apr;121(2):170-176.
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) has emerged as a powerful gene editing technology that is revolutionizing biomedical research and clinical medicine. The CRISPR system allows scientists to rewrite the genetic code in virtually any organism. This review provides a comprehensive overview of CRISPR and its clinical applications. We first introduce the CRISPR system and explain how it works as a gene editing tool. We then highlight current and potential clinical uses of CRISPR in areas such as genetic disorders, infectious diseases, cancer, and regenerative medicine. Challenges that need to be addressed for the successful translation of CRISPR to the clinic are also discussed. Overall, CRISPR holds great promise to advance precision medicine, but ongoing research is still required to optimize delivery, efficacy, and safety.
CRISPR（Clustered Regularly Interspaced Short Palindromic Repeats）已经成为一种强大的基因编辑技术，正在彻底改变生物医学研究和临床医学。CRISPR 系统允许科学家在几乎任何生物体中重写基因密码。本综述全面介绍了 CRISPR 及其临床应用。我们首先介绍了 CRISPR 系统，并解释了它如何作为一种基因编辑工具发挥作用。然后，我们重点介绍了 CRISPR 在遗传疾病、传染病、癌症和再生医学等领域的当前和潜在临床用途。我们还讨论了将 CRISPR 成功转化为临床应用所需解决的挑战。总的来说，CRISPR 有望推进精准医学，但仍需要开展进一步的研究来优化其传递、疗效和安全性。
