Human stem cell-based retina-on-chip as new translational model for validation of AAV retinal gene therapy vectors
K. Achberger, M. Cipriano, M. Duechs, C. Schoen, S. Michelfelder, B. Stierstorfer, T. Lamla, S. Kauschke, J. Chuchuy, J. Roosz, L. Mesch, V. Cora, S. Pars, N. Pashkovskaia, S. Corti, , P. Loskill
The paper evaluates seven AAV vectors using a human stem cell-derived Retina-on-chip model to assess their efficacy, kinetics, and cell tropism.
bioRxiv · Mar 2, 2021Bioengineering