Table 1.

Summary of long-term transgene expression and host immunity at 5 years post perfusion

NHP	Mode of vector delivery	Transgene expression	Anti-AAV1 Ab	Anti-AAV1 NF	Anti-rtTA Ab	rtTA directed IFN-γ cellular response	Muscle infiltrates
Mac 19	LR	Persisting	+	+	–	–	–
Mac 20	LR	Persisting	+	+	–	–	–
Mac 21	LR	Persisting	+	+	–	–	–

NHP, nonhuman primate; AAV, adeno-associated virus; Ab, antibodies, IFN-γ, interferon gamma.