Figure 2 | Gene Therapy

Figure 2

From: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65

Figure 2

Transduction of human iPS-derived RPE cells with AAV2/5 vectors. RPE cells derived from human induced pluripotent stem (iPS) cells were transduced using AAV2/5-NA65p.GFP. Phalloidin (a) and ZO-1 (b) staining was used to visualize individual cells in the cultured RPE monolayer. 4′,6-diamidino-2-phenylindole was used to visualize nuclei. Light field, merged and merged without nuclear stain images are shown for both panels. Scale bar, 25 μm. (c) Comparison of human iPS-derived RPE transduction efficiencies of AAV2/2-CMV.GFP and AAV2/5-CMV.GFP suggests AAV2/5 more efficiently transduces human RPE cells, although the difference just failed to reach significance (P=0.058; mean±s.e.m.; n=3).

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