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Gene replacement therapy in the CNS: A view from the retina

Published online by Cambridge University Press:  04 February 2010

Gail M. Seigel
Affiliation:
Department of Neurobiology and Anatomy, University of Rochester School of Medicine, Rochester, NY 14642

Abstract

Gene replacement therapy holds great promise in the treatment of many genetic CNS disorders. This commentary discusses the feasibility of gene replacement therapy in the unique context of the retina, with regard to: (1) the genetics of retinal neoplasia and degeneration, (2) available gene transfer technology, and (3) potential gene delivery vehicles.

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