I Edvard Smith C, Zain Rula, Blomberg Pontus
professor, överläkare, Karolins-ka ATMP-cent-rum; institutionen för laboratorie-medicin, Karolinska institutet.
docent, Centrum för sällsynta diagnoser (CSD), Klinisk genetik och genomik, Karolins-ka universitetssjukhuset; Karolins-ka ATMP-centrum; institutionen för laboratorie-medicin, Karolinska institutet.
Lakartidningen. 2025 Jan 29;122:24028.
Gene editing is a novel technology within gene therapy, which changes sequences in chromosomal DNA with precision. Even if there are alternative strategies, the Nobel Prize-winning CRISPR/Cas technology has become the dominating principle. During recent years base editing and prime editing, permitting editing without DNA double-strand breaks, have been developed. The first clinical gene editing results were reported in 2021; since then many patients have been treated, and recently the first treatment was approved as a novel therapy in the UK and later in USA and in EU. This update describes various aspects including methodological developments and safety.
基因编辑是基因治疗中的一项新技术，它能精确改变染色体DNA中的序列。尽管存在其他替代策略，但获得诺贝尔奖的CRISPR/Cas技术已成为主导原则。近年来，已开发出碱基编辑和引导编辑技术，可在不产生DNA双链断裂的情况下进行编辑。2021年报道了首个临床基因编辑结果；从那时起，许多患者接受了治疗，最近首个治疗方案在英国获批成为一种新型疗法，随后在美国和欧盟也获得批准。本综述介绍了包括方法学进展和安全性在内的各个方面。
