title: Tapping Stem Cells to Target AMD: Challenges and Prospects.
abstract: 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.
authors.affiliation: Institute of Human Genetics, University of Regensburg, Franz-Josef-Strauss-Allee 11, 93053 Regensburg, Germany. Caroline.Brandl@klinik.uni-regensburg.de.|Department of Ophthalmology, University Hospital Regensburg, Franz-Josef-Strauss-Allee 11, 93042 Regensburg, Germany. Caroline.Brandl@klinik.uni-regensburg.de.
authors.forename: Caroline
authors.initials: C
authors.lastname: Brandl
authors.affiliation: Institute of Human Genetics, University of Regensburg, Franz-Josef-Strauss-Allee 11, 93053 Regensburg, Germany. Felix.Grassmann@klinik.uni-regensburg.de.
authors.forename: Felix
authors.initials: F
authors.lastname: Grassmann
authors.affiliation: Institute of Human Genetics, University of Regensburg, Franz-Josef-Strauss-Allee 11, 93053 Regensburg, Germany. Julia.Riolfi@stud.uni-regensburg.de.
authors.forename: Julia
authors.initials: J
authors.lastname: Riolfi
authors.affiliation: Institute of Human Genetics, University of Regensburg, Franz-Josef-Strauss-Allee 11, 93053 Regensburg, Germany. bweb@klinik.uni-regensburg.de.
authors.forename: Bernhard H F
authors.initials: BH
authors.lastname: Weber
keywords: RNA-sequencing; age-related macular degeneration (AMD); cell-based transplantation therapy; disease modelling; drug screening; induced pluripotent stem cells (iPSCs); retinal pigment epithelium (RPE); stem cells