Stem Cells, Mechanism-Based Therapies and Regenerative Medicine Approaches

The degeneration of photoreceptors and retinal pigment epithelium is the cause of many inherited and acquired ocular disorders that can lead to blindness. Some of these disorders are caused by single gene mutations while others are caused by polygenic mutations or environmental factors. In the past, animal models and gene therapy have aimed at correcting single gene mutations in diseased retina. But more recently, advances in stem cell research, together with advances in retinal tissue transplantation, have moved forward the possibility of treating patients with polygenic or environmental causes of retinal degeneration. Stem cells can now be derived from many sources, including peripheral blood, and successfully differentiated into retinal precursors. In addition, retinal tissue transplantation has been successfully demonstrated in both animals and humans. This article will review recent advances in the study and treatment of retinal disease.