To further evaluate the i53 variants in a clinically relevant gene editing strategy, they were used for HSPC editing using an AAV6 donor designed to correct the sickle cell disease causing polymorphism in the HBB gene (HBB-SNP AAV6)20,21,26,27 followed by direct amplicon sequencing as a readout (Supplementary Fig. S3.1C). The gene discussed is HBB; the disease is sickle cell disease.