PRJNA517666
Editing aberrant splice sites efficiently restores Beta-globin expression in Beta-thalassemia
Aberrant HBB splice sites were disrupted to restore gene function in CD34+ hematopoietic stem cells from Beta-thalassemia patient donors. The IVS1-110G>A mutation was targeted with Cas9 RNP and the IVS2-654C>T mutation with Cas12a/Cpf1 RNP.