FIG. 1.

A single AAV vector expresses both RHO301 and siRNA301. (A) Map of AAV5-RHO301-siRNA301 (AAV-RS301). The expression of RHO301 was controlled by the proximal promoter region of the mouse opsin gene (MOPS500), and the small hairpin RNA containing the siRNA was controlled by the human H1 promoter. The entire coding region was contained between AAV2 terminal repeats (TR) and packaged in AAV5 capsids. (B) Various target sequences of human rhodopsin, mouse rhodopsin, and a resistant version of mouse rhodopsin (RHO301). (C) Subretinal injection of AAV2/5-MOPS-GFP leads to widespread transduction of the retina. One month after injection of our control virus, AAV-MOPS-GFP expression of GFP could be detected in a large area of the retina in a living mouse, using a Micron III fundus microscope equipped with a fluorescein filter. Color images available online at www.liebertonline.com/hum