Figure 5.
Comparable hepatic gene transfer efficiency by AAV2-cFVIII, AAV6-cFVIII, and AAV8-cFVIII in hemophilia A dogs. Southern blotting analysis for canine B-domain-deleted (BDD) FVIII transgene DNA in liver biopsies taken from multiple lobes in Morag and Floopers at 20 weeks, Vector at 102 weeks, Elisa at 177 weeks, Alexis at 77 weeks, and Maurizio at 49 weeks after vector injection. AAV-treated canine liver genomic DNAs yield 2 positive bands from BamHI digestion, representing the endogenous FVIII and transgene BDD-FVIII. The standards are generated with BamHI-digested plasmid DNAs spiked in liver genomic DNAs from naive hemophilia A dogs. RL indicates right lateral lobe; LL, left lateral lobe; RM, right medial lobe; LM, left medial lobe; Q, quadrate lobe; and C, caudate lobe.