Gene therapy for genetic and acquired retinal diseases

Abstract

We present an overview of the current status of basic science and translational research being applied to gene therapy for eye disease, focusing on diseases of the retina. We discuss the viral and nonviral methods being used to transfer genes to the retina and retinal pigment epithelium, and the advantages and disadvantages of each approach. We review the various genetic and somatic treatment strategies that are being used for genetically determined and acquired diseases of the retina, including gene replacement, gene silencing by ribozymes and antisense oligonucleotides, suicide gene therapy, antiapoptosis, and growth factor therapies. The rationales for the specific therapeutic approaches to each disease are discussed. Schematics of gene transfer methods and therapeutic approaches are presented together with a glossary of gene transfer terminology.