| abstract |
Provided are a method for specifically repairing, by using a base editing method, A>G 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-79A>G and HBB:c-78A>G pathogenic mutations of humans can be precisely repaired. By importing the gene-editing system into human cells or individuals, the A>G pathogenic mutations can be precisely repaired, so as to cure the β-thalassemia disease. |