Camille Bouchard, Kelly Godbout, Jacques P Tremblay
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引用次数: 0
摘要
基因编辑是一个不断发展的领域,Prime 编辑技术是其中最新的一种。它能利用切割 DNA 单链的 Cas9 切断酶修改基因。这种 Cas9 切断酶与反转录酶融合在一起,反转录酶复制研究人员合成的单个指导 RNA。这种技术一方面用于制造致病突变,以获得具有特定突变的细胞或动物模型。另一方面,Prime 编辑也可用于研究,通过纠正与致病作用相关的突变来治疗遗传性疾病。向生物体内受影响细胞提供治疗的方式是一大挑战。人们研究了不同的方法,以达到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 Prime 编辑的可能用途而需要解决的挑战。
[Correcting pathogenic mutations using prime editing: an overview].
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.
期刊介绍:
m/s offers high-quality review articles in French, covering all areas of biomedical and health research, in a monthly magazine format (10 issues / year). m/s is read by the whole French-speaking community, in France but also in Belgium, Switzerland, Canada, Morocco, Algeria, Tunisia etc. m/s is not a primary publication, and thus will not consider unpublished data. Most articles are invited by the Editors, but spontaneous proposals are welcomed. Each issue combines news and views on the most recent scientific publications, as well as broadly accessible and updated review articles on a specific topic, and essays on science and society, history of science, public health, or reactions to published articles. Each year, m/s also publishes one or two thematic issues focused on a research topic of high interest. All review articles and essays are peer-reviewed.