PRIMATE RETINAL CELLS TREATED WITH GENE THERAPY TECHNIQUES

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. Experimental evidence in other animal models has demonstrated that retinal function can be restored using gene therapy in adults. However, the effects on normal retinal physiology following gene therapy has not been investigated at the cellular level. This is an important question that is central to determining whether ocular gene therapy can ultimately be used to treat human vision anomalies and disease. The model system we will test is non-human primate retinal physiology following incorporation of new genetic material via viral vector. Following expression of a gene for green fluorescence in retinal cells, physiology and morphology will be assessed in vitro. Modifications to the promoter genes will be made so as to potentially target specific retinal cell subpopulations.