Therapeutical Approach of TargetAMD

The innovative therapeutic approach, realised by the interdisciplinary and international TargetAMD consortium, funded within the 7^th 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.

TargetAMD Approach sketch — Fig. 1: The TargetAMD approach. (A) Illustration of a retina harmed by AMD with imbalanced intraocular protein composition. (B) Secretion of recombinant PEDF by subretinal transplanted autologous gene modified pigment epithelial cells. The overexpression of PEDF results in a re-balance of the intraocular protein concentrations of VEGF and PEDF.. (PR: photoreceptors; RPE: retinal pigment epithelium cells; BM: Bruch’s membrane; BV= blood vessels; GMC= genetically modified cells).