Table 1.

Summary of preclinical gene therapy studies for hemophilia A.

fVIII transgene	Transgene species	Vector	Delivery	Model	Inhibitor status
BDD-fVIII	Human	AAV	I.V.	Mouse	Sometimesa
BDD-fVIII	Human	AAV	I.V.	NHP	Yesb
hfVIII-N6 or -V3	Human	AAV	I.V.	NHP	Yesc
BDD-cfVIII	Canine	AAV	I.V.	Dog	Rarelyd
ET3	Human/Porcine	AAV	I.V.	Mouse	Sometimese
ET3	Human/Porcine	LV	HSCT	Mouse	Nof
An53	Ancestral (95% Human)	AAV	I.V.	Mouse	Nog
BDD-cfVIII	Canine	LV	I.V.	Mouse	Yesh
BDD-pfVIII	Porcine	LV	HSCT	Mouse	Noi
BDD-fVIII	Human	LV	I.V.	Mouse	Sometimesj
BDD-fVIII	Human	AAV	I.V.	Dog	Yesk

^aGreig et al. (88); Sack et al. (89); Qadura et al. (90).

^bBunting et al. (25).

^cMcintosh et al. (87).

^dSabatino et al. (85); Callan et al. (91); Finn et al. (92).

^eLytle et al. (22); Brown et al. (21).

^fDoering et al. (17, 19, 24).

^gBrown et al. (23).

^hStaber et al. (93).

ⁱGangadharan et al. (16); Ide et al. (18, 94).

^jMerlin et al. (95); Wang et al. (96).

^kSun et al. (97).