Long-Term Correction of Hemophilia A via Integration of a Functionally Enhanced FVIII Gene into the AAVS1 Locus by Nickase in Patient-Derived iPSCs

Researchers employed CRISPR/Cas9 nickase-mediated knock-in to insert either the wild-type B domain-deleted (BDD)-FVIII gene or a functionally enhanced BDD-FVIII gene driven by a human elongation factor-1 alpha promoter into the adeno-associated virus site 1 locus of iPSCs derived from a patient with Hemophilia A.