Gene and cell therapy of retinal diseases

Abstract

Gene and cell therapy are tightly associated and quicklydeveloping areas of biomedicine, which purpose is developmentof methods to cure diseases caused by genetic defects and/ordeath of certain cell types. Current state of methods of retinaldiseases gene and cell therapy is analyzed in this review. Wereviewed development of gene therapeutic approaches totreatment of the 2-nd form of Lebers congenital amaurosisand other retinal diseases. We also compared therapeuticpotential of pluripotent stem cells and human adult retinalstem cells. Therapeutic potential of pluripotent stem cellsseems to be better due to their ability for unlimited expansionand organogenesis.

Keywords

gene therapy / cell therapy / Leber's congenital amaurosis / retinal disease / pluripotent stem cells

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