Gene therapy in <i>rd1</i> mice.

<p>(A) Schematic of the rAAV expression cassette with internal tandem repeats (ITR), the mGluR6 enhancer element (<i>GRM6</i>), the sv40 eukaryotic promoter sequence (psv40), an internal ribosomal entry site (IRES), woodchuck posttranscriptional regulatory element (WPRE), and bovine growth hormone polyadenylation sequence (BGHpA). (B) Retinal whole mount transduced by intravitreal injection and stained with an antibody against TurboFP635. The green dots represent transfected ON-bipolar cells. Scale bar, 500 μm. (C) Confocal image of a traverse section through a C57BL/6 retina stained against the rod bipolar cell marker PKCα (green), anti-TurboFP635 (red), and DAPI (blue), showing expressing rod bipolar cells (arrows). Also see <a href="http://www.plosbiology.org/article/info:doi/10.1371/journal.pbio.1002143#pbio.1002143.s003" target="_blank">S1 Fig</a>. Scale bar, 20 μm. (D) Raster plots of seven RGCs from rAAV_Opto-mGluR6–treated <i>rd1</i> retinas showing light responses to 1-s-long blue light pulses (bright band), four traces per cell. Cells are numbered and separated by stippled lines. (E) Fluorescent micrograph of the ganglion cell (green) giving rise to the transient ON response from D (star), with the underlying TurboFP635-labeled bipolar cells shown in red. Scale bar, 40 μm.