Figure 1.

AAV5-Mediated Expression of CR2-fH in the Mouse RPE

(A–D) RPE/choroid sections were collected one month following subretinal injection and stained with CR2 antibody before they were flattened and mounted to glass coverslips for fluorescence microscopy. Mice injected with AAV5-VMD2-mCherry demonstrated red autofluorescence in RPE flat mounts (A), whereas mice injected with AAV5-VMD2-CR2-fH produced green CR2 immunofluorescence (D). As negative controls, mice injected with AAV-mCherry did not fluoresce when stained with CR2 (B), and mice injected with AAV5-VMD2-CR2-fH did not demonstrate red autofluorescence (C). (E) Using dot blot analysis staining for anti-CR2, CR2-fH was detected in mice injected with AAV5-VMD2-CR2-fH both in the RPE/choroid and, to a lesser extent, in the retina. (F) Bioavailability of CR2-fH in RPE/choroid was compared between intravenous injection of CR2-fH and subretinal injection of AAV5-VMD2-CR2-fH.