Based on the pathogenic mechanisms of age-related macular degeneration (AMD), tremendous preclinical and clinical trials have demonstrated that cell transplantation which aim to replace impaired retinal pigment epithelium (RPE) with healthy RPE cells is a promising approach to treat AMD. So far, choices of cell sources mainly are autologous RPE, iris pigment epithelium, fetal RPE, human embryonic stem cell-derived RPE and human induced pluripotent stem cell-derived RPE, and some of them are undergoing clinical researches. Grafting manners in cell-based therapies are various including RPE sheet or RPE-choroid complex transplantation, RPE cell suspension injection, and RPE sheet transplantation with scaffolds. This review is limited to cell-based therapies for RPE that damaged first in the progress of AMD and focus on recent advances in cell sources, transplantation methods, preclinical and clinical trials, and the obstacles that must be overcome.
Clustered regularly interspersed short palindromic repeats/Cas system is a powerful genome-editing tool for efficient and precise genome engineering both in vitro and in vivo, with the advantages of easy, convenient and low cost. This technology makes it possible to simultaneously mutate multiple genes in a single fertilized egg, thus to study the gene expression, genetic interaction and gene function. Even though this method is still in its immature stage and its stability is inconclusive, making precision models of ocular diseases through genome editing may provide a positive effect to explore gene targeted therapy in genetic eye disease.