Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse
Abstract Background Hemophilia A, a bleeding disorder resulting from F8 mutations, can only be cured by gene therapy.A promising strategy is CRISPR-Cas9-mediated precise insertion of F8 in hepatocytes at highly expressed gene loci, such as albumin (Alb).Unfortunately, the precise in vivo integration efficiency of a long insert is very low (~ 0.1%