Figure 1.

Schematic of rAAV tropism testing for in vitro and ex vivo-isolated cells. Human in vitro cellular models (including induced pluripotent stem cells (iPSC), iPSC-derived retinal pigment epithelium (iPSC-RPE), and iPSC-derived human cortical neurons) and ex vivo-isolated rat cortical neurons were generated and cultured. Before transduction testing, cells were collected and seeded at equal numbers in each well of two Matrigel-coated 96-well plates. Eleven AAV serotypes at different multiplicity of infection (MOI) were introduced to the cells in triplicate. GFP expression was measured by scanning the plate in Typhoon scanner followed by an analysis with protein array analyzer on ImageJ. The AAV-eGFP transgene expression was normalized with background and used as data to compare the critical effect of cell types and AAV serotypes on transgene expression.