Figure 7.

Systemic administration of 17-AAG coupled with barrier modulation using claudin-5 targeting siRNA protects the ONL in the RP10 mouse model. WT mice were first sub-retinally injected with AAV-mutant224. Two weeks post-injection, claudin-5 siRNA was administered using in vivo-JetPEI by tail vein injection. Forty-eight hours post-barrier modulation, 17-AAG was administered systemically by an i.p. injection, and claudin-5 siRNA and 17-AAG were administered once more a week later. (A) Retinal sections (12 μm) were immunostained with hrGFP primary antibody and counterstained with goat polyclonal to rabbit IgG Cy2 secondary antibody. Green, hrGFP; blue, DAPI; ONL, outer nuclear layer; INL, inner nuclear layer. All images show merge hrGFP and DAPI staining. Scale bar denotes 20 μm. (B) Data represent average positively transduced ONL area per unit length (μm) ± SEM (***P ≤ 0.0001, n = 5).