Bouchard Camille, Godbout Kelly, Tremblay Jacques P
Département de médecine moléculaire, Université Laval, Québec, Canada - Centre de recherche du CHU de Québec, Université Laval, Québec, Canada.
Med Sci (Paris). 2024 Oct;40(10):748-756. doi: 10.1051/medsci/2024109. Epub 2024 Oct 25.
Gene editing is an ever-evolving field and Prime editing technology is among the latest ones. It makes it possible to modify a gene using a Cas9 nickase that cuts a single strand of DNA. This Cas9 nickase is fused with a reverse transcriptase that copies a single guide RNA synthetized by the researcher. This technique is used on one hand to create pathogenic mutations to obtain cell or animal models with a specific mutation. On the other hand, Prime editing is also used in research to treat hereditary diseases by correcting mutations associated with a pathogenic effect. The mode of delivery of the treatment to the affected cells in living organisms constitutes a main challenge. Different methods are studied to reach the organs specific to each disease. This review article presents the latest results in the field as well as the challenges to solve to optimize the possible uses of Prime editing.
基因编辑是一个不断发展的领域,而碱基编辑技术是最新的技术之一。它使得使用切割单链DNA的Cas9切口酶来修饰基因成为可能。这种Cas9切口酶与逆转录酶融合,该逆转录酶可复制研究人员合成的单导向RNA。一方面,这项技术用于产生致病突变,以获得具有特定突变的细胞或动物模型。另一方面,碱基编辑也用于研究中,通过纠正与致病效应相关的突变来治疗遗传性疾病。将治疗方法递送至生物体中受影响细胞的方式构成了一项主要挑战。人们正在研究不同的方法来靶向每种疾病特有的器官。这篇综述文章介绍了该领域的最新成果以及为优化碱基编辑的可能用途而需要解决的挑战。