Gene Therapy for Retinal Disease

© 2015 Elsevier Inc. All rights reserved.. Treatment of inherited retinal diseases (IRDs) through the use of gene therapy strategies has made considerable advances in recent years. The majority of research has focused on the use of adeno-associated viral (AAV) vectors to treat retinal degenerations through preventing or delaying the onset of blindness. Data from human clinical trials of gene therapy for retinal disease show encouraging signs of safety and efficacy from AAV vectors. There has been much progress in enhancing cell-specific targeting and transduction efficiency of the various retinal layers plus the use of AAV-delivered growth factors to augment the therapeutic effect and limit cell death within the retina. This chapter will provide an overview of the encouraging developments in gene therapy targeting the retina with a focus on serotype use and promoter selection in preclinical studies in mouse models of IRDs and an overview of recent and current human clinical trials.