Gene therapy in the treatment of retinal dystrophies

Abstract

Gene therapy is defined as procedures to transfer genetic material to specific organs, with the purpose of producing therapeutic effects that seek to correct defects or genetic diseases, either directly (<em>in vivo</em>) or indirectly (<em>ex vivo</em>), by using cells as a delivery vehicle. Eye diseases, mainly macular diseases, have a high genetic component. This has led to several studies suggesting alternative treatments such as gene therapy for their treatment. Studies have concluded that gene therapy is a novel and promising therapeutic strategy that could provide a more effective way to treat these diseases. The objective of this article is to present a review of the concepts of gene therapy, types of vectors, and gene therapy in retinal dystrophies.