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Tapping Stem Cells to Target AMD: Challenges and Prospects

1
Institute of Human Genetics, University of Regensburg, Franz-Josef-Strauss-Allee 11, 93053 Regensburg, Germany
2
Department of Ophthalmology, University Hospital Regensburg, Franz-Josef-Strauss-Allee 11, 93042 Regensburg, Germany
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Author to whom correspondence should be addressed.
Academic Editors: Margaret DeAngelis and Lindsay Farrer
J. Clin. Med. 2015, 4(2), 282-303; https://doi.org/10.3390/jcm4020282
Received: 1 September 2014 / Accepted: 13 January 2015 / Published: 29 January 2015
(This article belongs to the Special Issue Age-Related Macular Disease)
Human pluripotent stem cells (hPSCs) are increasingly gaining attention in biomedicine as valuable resources to establish patient-derived cell culture models of the cell type known to express the primary pathology. The idea of “a patient in a dish” aims at basic, but also clinical, applications with the promise to mimic individual genetic and metabolic complexities barely reflected in current invertebrate or vertebrate animal model systems. This may particularly be true for the inherited and complex diseases of the retina, as this tissue has anatomical and physiological aspects unique to the human eye. For example, the complex age-related macular degeneration (AMD), the leading cause of blindness in Western societies, can be attributed to a large number of genetic and individual factors with so far unclear modes of mutual interaction. Here, we review the current status and future prospects of utilizing hPSCs, specifically induced pluripotent stem cells (iPSCs), in basic and clinical AMD research, but also in assessing potential treatment options. We provide an outline of concepts for disease modelling and summarize ongoing and projected clinical trials for stem cell-based therapy in late-stage AMD. View Full-Text
Keywords: stem cells; induced pluripotent stem cells (iPSCs); retinal pigment epithelium (RPE); age-related macular degeneration (AMD); disease modelling; drug screening; cell-based transplantation therapy; RNA-sequencing stem cells; induced pluripotent stem cells (iPSCs); retinal pigment epithelium (RPE); age-related macular degeneration (AMD); disease modelling; drug screening; cell-based transplantation therapy; RNA-sequencing
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Brandl, C.; Grassmann, F.; Riolfi, J.; Weber, B.H.F. Tapping Stem Cells to Target AMD: Challenges and Prospects. J. Clin. Med. 2015, 4, 282-303.

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