BASE EDITING SYSTEM AND METHOD FOR SPECIFICALLY REPAIRING HBB GENE MUTATIONS OF HUMANS, REAGENT KIT, AND APPLICATIONS THEREOF

摘要

Provided are a method for specifically repairing, by using a base editing method, AG pathogenic mutations causing β-thalassemia of humanms, a gRNA specifically targeting the mutations, and a base editing protein. By means of the method, HBB:c-79AG and HBB:c-78AG pathogenic mutations of humans can be precisely repaired. By importing the gene-editing system into human cells or individuals, the AG pathogenic mutations can be precisely repaired, so as to cure the β-thalassemia disease.