Table 4.

First AAV Micro-dystrophin Clinical Trial (Local Injection)

Patient	Dose (vg/kg)	Cortico Steroid	Pre-Nab to AAV	Positive Myofiber	AAV Genome	CTL to m-Dystrophin	CTL to Revertant Fiber	CTL to AAV Capsid
1	2 × 1010	yes			+			+
2	2 × 1010	yes	1:800				+	±
3	2 × 1010	no		3∼4 (day 42)	+			±
4	1 × 1011	no						±
5	1 × 1011	yes	1:100		++	+
6	1 × 1011	yes		1 (day 42)	++

AAV genome: +, 0.5∼1 copy/nucleus; ++, 1.5∼3 copies/nucleus. Abbreviations: CTL, cytotoxic T cell response; Pre-Nab, preexisting neutralizing antibody.