University of Algiers 1, Faculty of Medicine, Algiers, Algeria.
Biochem Med (Zagreb). 2023 Oct 15;33(3):030601. doi: 10.11613/BM.2023.030601. Epub 2023 Aug 5.
It's been 10 years now from the debut of clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR/Cas9) era in which gene engineering has never been so accessible, precise and efficient. This technology, like a refined surgical procedure, has offered the ability of removing different types of disease causing mutations and restoring key proteins activity with ease of outperforming the previous resembling methods: zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs). Additionally, CRISPR-Cas9 systems can systematically introduce genetic sequences to the specific sites in the human genome allowing to stimulate desired functions such as anti-tumoral and anti-infectious faculties. The present brief review provides an updated resume of CRISPR-Cas9's top achievements from its first appearance to the current date focusing on the breakthrough research including and human studies. This enables the evaluation of the previous phase 'the proof-of-concept phase' and marks the beginning of the next phase which will probably bring a spate of clinical trials.
从 clustered regularly interspaced short palindromic repeats-associated protein 9(CRISPR/Cas9)时代问世至今已经有 10 年了,基因工程从未如此普及、精确和高效。这项技术就像一种精细化的外科手术,具有轻松去除不同类型致病突变和恢复关键蛋白活性的能力,优于以前类似的方法:锌指核酸酶(ZFNs)和转录激活因子样效应核酸酶(TALENs)。此外,CRISPR-Cas9 系统可以系统地将遗传序列引入人类基因组的特定位点,从而刺激抗肿瘤和抗感染等所需功能。本综述简要介绍了从首次出现到目前为止 CRISPR-Cas9 的最高成就,重点介绍了包括临床前和临床研究在内的突破性研究。这使得我们能够评估之前的“概念验证阶段”,并标志着下一阶段的开始,下一阶段可能会带来大量临床试验。