Figure 3. Retinal microdystrophin expression in gene transferred 3-month-old mdx^3cv mouse. Subretinal delivery of an AAV-9 human microdystrophin vector resulted in efficient OPL transduction in mdx^3cv mouse retina. A-F are from mdx^3cv mouse eyes, and G is from a C57BL/6J mouse eye. A-C are representative serial sections from an eye infected with AAV-9 microdystrophin vector. D-F are representative serial sections from an eye mock-infected with HEPES buffer. A, D show retinal structure of mdx^3cv mouse (H&E staining). B, E show immunostaining with the Dys-3 antibody, which recognizes microdystrophin. At 5 weeks after subretinal injection, microdystrophin expression was evident in the injected retina (B), but not in the mock-infected eye (E). C and F display immunostaining with the Dys-2 antibody, which recognizes endogenous dystrophin. Neither the AAV-9-infected nor the mock-infected eye showed endogenous dystrophin expression. G shows immunostaining with the Dys-2 antibody on the C57BL/6J retina. Dystrophin expression is seen in the outer plexiform layer (OPL). Arrows point to the OPL. Abbreviations: inner nuclear layer (INL), outer nuclear layer (ONL).