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PEDF Gene Transfer:
A Promising Potential Treatment for Ocular Neovascularization

Peter A. Campochiaro

Local delivery of antiangiogenic agents is an appealing approach for the treatment of ocular neovascularization, since it is likely to avoid systemic side effects. Local delivery of an endogenous antiangiogenic protein may have the added advantage of minimizing local adverse effects. But delivery of proteins is problematic. Gene transfer provides a means to attain sustained local delivery of proteins. We investigated the effect of AV- and AAV-mediated gene transfer of PEDF in three models of ocular neovascularization. Increased intraocular expression of PEDF resulted in significant inhibition of retinal and choroidal neovascularization, and treatment of already established neovascularization resulted in regression due to apoptosis of vascular cells within neovascularization. These exciting preclinical data provide a strong rationale for clinical trials to investigate PEDF gene transfer in patients with ocular neovascularization.