Table 1. T cell responses to AAV in intramuscular gene transfer trials.

Serotype	Transgene	Route	Dose (vg)	Capsid T cell reactivity	Transgene expression in muscle biopsies	Reference (ClinicalTrials.gov ID#)
AAV1	Gamma sarcoglycan	IM	3×109	0/3	0/3	Herson et al., Brain 2012 (NCT01344798)
			1.5×1010	0/3	0/3
			4.5×1010	1/3	3/3
AAV1	Alpha sarcoglycan	IM	3.25×1011	1/3	3/3	Mendell et al., Ann Neurol 2009 (NCT00494195)
			3.25×1011	2/3	2/3	Mendell et al., Ann Neurol 2010 (NCT00494195)
AAV1	Acid-alpha glucosi-dase	IM (diaphragm)	1×1012	0/3 ([H3] prolif.)	N/A	Smith et al., Hum Gene Ther 2013 (NCT00976352)
			5×1012	0/2 ([H3] prolif.)	N/A
AAV2.5	Minidystrophin	IM	6×1011	1/2 (IS)	0/2	Bowles et al., Mol Ther 2012 Mendell et al., NEJM 2009 (NCT00428935)
			3×1012	0/2	1/2
			3×1012 6.6×1012 capsid particles	0/2 (immunosup-pression, IS)	1/2
AAV1	Follistatin	IM	2.1×1012	1/3	2/2	Mendell et al., Mol Ther 2015 (NCT01519349)
			4.2×1012	3/3	2/2
AAV1	Lipoprotein lipase	IM	7.0×1012	2/4	2/3	Mingozzi et al., Blood 2009
			2.1×1013	3/6	3/6
			2.1×1013	1/4 (IS)	1/2	Ferreira et al. , Front immunol 2014 (NCT01109498; NCT00891306)
			7.0×1013	4/13 (IS)	6/8
AAV1	Alpha-1 Antitrypsin	IM	6.9×1012	N/A	0/3*	Brantly et al., PNAS 2009 (NCT00430768)
			2.2×1013	2/2	Transient*
			6.0×1013	3/3	3/3*
			4.2×1013	3/3	1/3*	Flotte et al., Hum Gene Ther 2012 Mueller et al. , J Clin Invest 2013 (NCT01054339)
			1.3×1014	3/3	2/3*
			4.2×1014	3/3	3/3*