Therapeutical Approach of TargetAMD
The innovative therapeutic approach, realised by the interdisciplinary and international TargetAMD consortium, funded within the 7th Framework Program of the European Commission, comprises the subretinal transplantation of genetically modified, autologous iris pigment epithelial (IPE) cells.
Gene transfection with the recombinant PEDF will cause an overexpression of the protein providing a long-lasting cure for exudative AMD (Fig. 1). Stable PEDF gene delivery will be based on the well-established non-viral hyperactive Sleeping Beauty (SB100X) transposon system, which combines the efficacy of viral delivery with the safety of naked DNA plasmids. This personalised approach, combining cell with gene therapy, will replace degenerated RPE cells, regenerate the neuroretinal environment, and offer a long-life therapeutic solution.