Abstract
Age-related macular degeneration (AMD) is the leading cause of blindness in people over age 55 in the U.S. and the developed world. This condition leads to the progressive impairment of central visual acuity. There are significant limitations in the understanding of disease progression in AMD as well as a lack of effective methods of treatment. Lately, there has been considerable enthusiasm for application of stem cell biology for both disease modeling and therapeutic application. Human embryonic stem cells and induced pluripotent stem cells (iPSCs) have been used in cell culture assays and in vivo animal models. Recently a clinical trial was approved by FDA to investigate the safety and efficacy of the human embryonic stem cell-derived retinal pigment epithelium (RPE) transplantation in sub-retinal space of patients with dry AMD These studies suggest that stem cell research may provide both insight regarding disease development and progression, as well as direction for therapeutic innovation for the millions of patients afflicted with AMD.
Highlights
Age-related macular degeneration (AMD) is a devastating neurodegenerative disease and leading cause of blindness in people over 55 years of age that affects a central nervous system tissue, the retinal pigmented epithelium (RPE) [1]
Transplanted RPE that were differentiated from Embryonic Stem Cell (ESC) have led to improvements in visual acuity in preclinical models of the disease [16,70] In addition, human induced pluripotent stem cells (iPSCs) have been differentiated towards functional RPE cells, and we have demonstrated that human iPSC-derived RPE are functionally and phenotypically similar to native RPE [18]
Concerns remain regarding the process of pluripotency induction and residual epigenetics in induced pluripotent stem (iPS) cells, since there are unique characteristics that may significantly affect the propensity of differentiation and may influence ongoing attempts to use iPSCs for disease modeling
Summary
Age-related macular degeneration (AMD) is a devastating neurodegenerative disease and leading cause of blindness in people over 55 years of age that affects a central nervous system tissue, the retinal pigmented epithelium (RPE) [1]. In vivo subretinal transplant of purified hESC-derived RPEs into the Royal College of Science (RCS) rat and the Elov mouse, an animal model for Stargardt disease, have shown marked improvements in visual function [118] and survival of the graft without teratoma formation or cellular hyperproliferation [14,16]. These data are promising, the use of hESCs is challenging due to the ethical issues, the immunological reaction and the long-term risks of teratoma formation [10]. Photoreceptor transplant in macular degeneration may confer a great therapeutic avenue in the future for rescue visual acuity
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