Gene Therapy for Retinal Degeneration using Home-grown Gene Transfer Vectors-Aim for Clinical Trial-

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1. The assessment of the efficacy of gene therapy for the animal models of retinal degeneration1) Development of new gene therapy technologyWe developed the new SIV (simian immunodeficiency virus) vectors carrying hFGF-2 or both hPEDF and hFGF-2 (SIV-FGF-2 or SIV-dual). Moreover, we developed the system for large-scale production of SIV-hPEDF with the aim of the clinical trail.2) We showed the synergistic therapeutic effect using SIV-hPEDF and SIV-hFGF-2 for two animal models of retinal degeneration (RCS rats and rds mice)2. The establishment of new gene therapy strategy by use of neural progenitor cells1) Development of the culture method to isolate neural progenitor cells from ciliary body of the eyeWe identified a new and divergent mechanism (a reprogramming system) underlying the neural differentiation of ciliary body-derived cells via sphere formation. The nestin-negative epithelial-like cells from ciliary body came to express nestin.2) We demonstrated the efficient gene transfer to neural progenitor cells from ciliary body of the eye via SIV vector3. Remains1) We evaluated the ability of our 3rd generation SIV vectors to transfer genes into nonhuman primate retinas.2) We suggested the possibility that VEGF-C and VEGF-D expression in RPE modify the ocular angiogenesis, such as age-related macular degeneration (AMD) as angiogenic stimulators.