Fig. 6
From: Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse
AAV-CRISPR therapy cures hemophilia A mice. a Schematic of the AAV vectors used in the study and experimental overview for in vivo studies. SA85 on the donor functions as a splice acceptor. b Stable F8 activity after injection of AAV-Cas9, three different AAV-sgRNAs targeting Alb introns 11 or 13, and AAV-BDDF8 donor. (n = 3 for each group). A group of mice treated with AAV-BDDF8 donor only (n = 3) serves as a negative control