Behavioral and Brain Sciences

Open Peer Commentary

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

Gail M. Seigela1

a1 Department of Neurobiology and Anatomy, University of Rochester School of Medicine, Rochester, NY 14642


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.