Figure 1.

Cell type-specific expression of NMNAT1 using various promoters

Diagrams show the design of AAV constructs with various promoters. Immunofluorescence images demonstrate the cell type-specific expression of hNMNAT1 (red) after delivery of scAAV9 at a titer of 1E9 vg/μL, 2 and 4 weeks post subretinal injection. Expression of NMNAT1 using (A) the CASI promoter, which led to expression in most cell types; (B) hGRK1, which led to expression in the photoreceptor cells as well as some expression in RPE and the INL (arrows); (C) ProC1, which led to expression specifically in the photoreceptor cells; (D) VMD2, which led to expression in the RPE; (E) ProC3, which led to expression in the retinal ganglion cells; and (F) ProD5, which led to expression in the rods. GCL, ganglion cell layer; INL, inner nuclear layer; ONL, outer nuclear layer; RPE, retinal pigment epithelium. Top panels show staining with an antibody specific to human NMNAT1 (hNMNAT1). Bottom panels are counterstained with DAPI (blue). Scale bar indicates 20 μm.