J Biomed Nanotechnol. 2021 Mar 1;17(3):331-356. doi: 10.1166/jbn.2021.3045.
CRISPR is a powerful gene editing tool for correcting disease-causing mutations. It is becoming more and more evident that CRISPR is a promising approach to treating human genetic diseases. The technologies for adding or removing genes have made significant advances over the past few years and have shown promising potential outcomes. In the current study, we mainly introduce the CRISPR/Cas system and there are several applications in the treatment of genetic diseases, particularly during embryo development.
CRISPR 是一种强大的基因编辑工具,可用于纠正致病突变。越来越明显的是,CRISPR 是治疗人类遗传疾病的一种很有前途的方法。在过去的几年中,添加或去除基因的技术取得了重大进展,并显示出了有希望的潜在结果。在当前的研究中,我们主要介绍了 CRISPR/Cas 系统及其在治疗遗传疾病中的几种应用,特别是在胚胎发育期间。