上海科技大学知识管理系统

一种碱基编辑系统及碱基编辑方法，所述碱基编辑系统包括：1)C2C9核酸酶和//或编码所述C2C9核酸酶的核酸；2)引导RNA和//或编码所述引导RNA的核酸；3)脱氨酶和//或编码所述脱氨酶的核酸。所述碱基编辑方法包括将靶基因与所述的碱基编辑系统接触，以实现靶基因上单碱基的编辑。成功实现了靶位点上两种不同类型的单碱基突变，且极大的缩小了碱基编辑系统的核酸编码尺寸，最优的碱基编辑系统其蛋白部分基因尺寸仅为2793bp，远远小于单个AAV能够装载的最大外源基因大小。

A base editing system and a base editing method. The base editing system comprises : 1) a C2C9 nuclease and//or a nucleic acid encoding the C2C9 nuclease; 2) a guide RNA and//or a nucleic acid encoding the guide RNA; and 3) a deaminase and//or a nucleic acid encoding the deaminase. The base editing method comprises bringing a target gene into contact with the base editing system to achieve the editing of a single base on the target gene. The method and the system successfully achieve two different types of single base mutations at a target site, and greatly reduce the nucleic acid encoding size of the base editing system. The gene size of the protein moiety of the optimal base editing system is only 2793 bp, which is far smaller than the size of the maximum foreign gene that can be loaded by a single AAV.