Figure 6.
Transduction efficiency of AAV vectors in the iPSC-derived RPE. (a) Comparison of the transduction efficiency over time of a panel of AAV vectors (2/2, 2/4, 2/5, 2/8, and 2/9) expressing EGFP under the control of the CMV promoter. This transduction efficiency was also compared with that of an AAV2/5 vector expressing EGFP under control of the CAG promoter (2/5 CAG) and with nontransduced cells (–). Transduction was performed with 25,000 vector genomes (vg) per cell, and the number of EGFP-positive cells was analyzed by flow cytometry. (b) Comparison of the transduction efficiency in wild-type and CHM1 RPE 4 weeks postincubation with 100,000 vg per cell of AAV2/5-CAG-EGFP. AAV, adeno-associated virus; CAG, chicken β-actin with a CMV enhancer; iPSC, induced pluripotent stem cell; RPE, retinal pigment epithelium.