Figure 4.

In vivo morphological rescue potential of miniCEP290^580–1180. Cep290^rd16 retinas injected with indicated miniCEP290, GFP, or uninjected Cep290^rd16 were stained with DAPI (blue) (A). The outer nuclear layer (ONL) is marked with vertical lines. WT retinal section is shown for comparison. INL, inner nuclear layer. (B) Quantification of the ONL thickness by counting the number of layers of the vector transduced region of the injected Cep290^rd16 mice compared to uninjected retinas is represented. There is initial preservation of the number of layers, but progressive decline is detected with age. ***p < 0.0001. (C) The injected Cep290^rd16 mouse retinas were also examined by ultrathin sectioning to assess morphological preservation at 3 weeks post injection. The ONL is marked with vertical lines. The outer segment (OS) layer is clearly visible in the miniCEP290^580–1180 injected retinas. (D) Improved expression of RDS (red) is detected in the miniCEP290^580–1180 injected Cep290^rd16 mice. Staining with GFP (green) marked the injected regions. Color images available online at www.liebertpub.com/hum