Retinal Pigment Epithelium Replacement Therapy for Age-Related Macular Degeneration: Are We There Yet?
- PMID: 31914900
- PMCID: PMC8783375
- DOI: 10.1146/annurev-pharmtox-010919-023245
Retinal Pigment Epithelium Replacement Therapy for Age-Related Macular Degeneration: Are We There Yet?
Abstract
Pluripotent stem cells (PSCs) are a potential replacement tissue source for degenerative diseases. Age-related macular degeneration (AMD) is a blinding disease triggered by degeneration of the retinal pigment epithelium (RPE), a monolayer tissue that functionally supports retinal photoreceptors. Recently published clinical and preclinical studies have tested PSC-derived RPE as a potential treatment for AMD. Multiple approaches have been used to manufacture RPE cells, to validate them functionally, to confirm their safety profile, and to deliver them to patients either as suspension or as a monolayer patch. Since most of these studies are at an early regulatory approval stage, the primary outcome has been to determine the safety of RPE transplants in patients. However, preliminary signs of efficacy were observed in a few patients. Here, we review the current progress in the PSC-derived RPE transplantation field and provide a comparative assessment of various approaches under development as potential therapeutics for AMD.
Keywords: cell therapy; clinical grade; induced pluripotent stem cells; patch; pluripotent stem cells; retinal degeneration; retinal pigment epithelium.
Figures

References
-
- Thomson JA, Itskovitz-Eldor J, Shapiro SS, Waknitz MA, Swiergiel JJ, et al. 1998. Embryonic stem cell lines derived from human blastocysts. Science (New York, N.Y.) 282:1145–7 - PubMed
-
- Takahashi K, Yamanaka S. 2006. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 126:663–76 - PubMed
-
- Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, et al. 2007. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131:861–72 - PubMed
-
- Yu J, Vodyanik MA, Smuga-Otto K, Antosiewicz-Bourget J, Frane JL, et al. 2007. Induced pluripotent stem cell lines derived from human somatic cells. Science 318:1917–20 - PubMed
-
- Schwartz SD, Hubschman JP, Heilwell G, Franco-Cardenas V, Pan CK, et al. 2012. Embryonic stem cell trials for macular degeneration: a preliminary report. Lancet 379:713–20 - PubMed
Publication types
MeSH terms
Grants and funding
LinkOut - more resources
Full Text Sources
Other Literature Sources
Medical