Stem Cell Therapy for Retinal Disease Treatment: An Update

Abstract

At this time, the main targets of stem cell therapy for retinal degenerative disease are age-related macular degeneration, Stargardt disease, and retinitis pigmentosa. The goal of stem cell therapy is to either to “rescue” the surviving retinal cells (by providing the necessary support or generating neurotrophic agents) and/or to “replace” the cells that have degenerated. Stem cells being used in ongoing early human trials to treat degenerative retinal disease include induced pluripotent stem cell (iPSC)-derived retinal pigment epithelium (RPE), embryonic stem cell-derived RPE, iPSC-neural progenitor cells, bone marrow-derived stem cells, and human central nervous system derived stem cells among others. It is too early to judge the outcome of these sources of tissue, but early results are positive. Continuing research in various aspects of transplantation- establishing cell lines without danger of tumor formation or immune rejection, refining surgical techniques and instruments, and identifying factors that promote cell survival, differentiation, and integration of the transplanted cells, should allow for rapid and continued progress in the field.