Faculty of Medicine, Hacettepe University, Ankara 06100, Turkey.
Faculty of Medicine, Hacettepe University, Ankara 06100, Turkey.
Life Sci. 2023 Jan 1;312:121204. doi: 10.1016/j.lfs.2022.121204. Epub 2022 Nov 17.
Treatment of genetic disorders by genomic manipulation has been the unreachable goal of researchers for many decades. Although our understanding of the genetic basis of genetic diseases has advanced tremendously in the last few decades, the tools developed for genomic editing were not efficient and practical for their use in the clinical setting until now. The recent advancements in the research of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated protein (Cas) systems offered an easy and efficient way to edit the genome and accelerated the research on their potential use in the treatment of genetic disorders. In this review, we summarize the clinical trials that evaluate the CRISPR/Cas systems for treating different genetic diseases and highlight promising preclinical research on CRISPR/Cas mediated treatment of a great diversity of genetic disorders. Ultimately, we discuss the future of CRISPR/Cas mediated genome editing in genetic diseases.
几十年来,通过基因组操作来治疗遗传疾病一直是研究人员遥不可及的目标。尽管我们对遗传疾病的遗传基础的理解在过去几十年中取得了巨大进展,但直到现在,为基因组编辑开发的工具在临床环境中使用还不够高效和实用。最近在对规律成簇间隔短回文重复序列 (CRISPR) 和 CRISPR 相关蛋白 (Cas) 系统的研究进展提供了一种简单有效的编辑基因组的方法,并加速了对其在治疗遗传疾病中的潜在用途的研究。在这篇综述中,我们总结了评估 CRISPR/Cas 系统治疗不同遗传疾病的临床试验,并重点介绍了 CRISPR/Cas 介导治疗多种遗传疾病的有前途的临床前研究。最终,我们讨论了 CRISPR/Cas 介导的基因组编辑在遗传疾病中的未来。