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Journal of Translational Medicine

, 11:53

Cell, tissue and gene therapy


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.

KeywordsAge-related macular degeneration RPE Stem cells AbbreviationsAMDAge-related Macular Degeneration

RPERetinal-Pigmented Epithelium

IPSInduced Pluripotent Stem cell

VEGFVascular Endothelial Growth Factor

ESCEmbryonic Stem Cell

hECShuman Embryonic Stem cell

CFHComplement Factor H

C2Complement 2

CFBComplement Factor B

C3Complement 3

CFHR1Complement Factor H-Related gene

CFIComplement Factor I

ARMS2Age-related Maculopathy Susceptibility 2

APOEApolipoprotein E

CETPCholesteryl Ester Transfer Protein

GSTM1GSTM5, Glutathione S-transferase isoforms mu1, mu5

PLLAPoly L-Lactic Acid

PLGAPoly D, L-Lactic Co-Glycolic Acid

RPCRetinal Progenitor Cells

PGSPoly Glycerol Sebacate

PCLPoly Ε-Caprolactone.

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Autor: Heather Melville - Matthew Carpiniello - Kia Hollis - Andrew Staffaroni - Nady Golestaneh


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