title: Induced pluripotent stem cells as custom therapeutics for retinal repair: progress and rationale.
abstract: Human pluripotent stem cells have made a remarkable impact on science, technology and medicine by providing a potentially unlimited source of human cells for basic research and clinical applications. In recent years, knowledge gained from the study of human embryonic stem cells and mammalian somatic cell reprogramming has led to the routine production of human induced pluripotent stem cells (hiPSCs) in laboratories worldwide. hiPSCs show promise for use in transplantation, high throughput drug screening, "disease-in-a-dish" modeling, disease gene discovery, and gene therapy testing. This review will focus on the first application, beginning with a discussion of methods for producing retinal lineage cells that are lost in inherited and acquired forms of retinal degenerative disease. The selection of appropriate hiPSC-derived donor cell type(s) for transplantation will be discussed, as will the caveats and prerequisite steps to formulating a clinical Good Manufacturing Practice (cGMP) product for clinical trials.
authors.affiliation: Waisman Center, University of Wisconsin, Madison, WI, USA; McPherson Eye Research Institute, University of Wisconsin, Madison, WI, USA.
authors.forename: Lynda S
authors.initials: LS
authors.lastname: Wright
authors.affiliation: Waisman Center, University of Wisconsin, Madison, WI, USA; McPherson Eye Research Institute, University of Wisconsin, Madison, WI, USA.
authors.forename: M Joseph
authors.initials: MJ
authors.lastname: Phillips
authors.affiliation: Department of Ophthalmology, Lozano Blesa Hospital and Aragones Health Sciences Institute, Zaragoza, Spain.
authors.forename: Isabel
authors.initials: I
authors.lastname: Pinilla
authors.affiliation: Waisman Center, University of Wisconsin, Madison, WI, USA.
authors.forename: Derek
authors.initials: D
authors.lastname: Hei
authors.affiliation: Waisman Center, University of Wisconsin, Madison, WI, USA; McPherson Eye Research Institute, University of Wisconsin, Madison, WI, USA; Department of Ophthalmology and Visual Sciences, University of Wisconsin, Madison, WI, USA. Electronic address: dgamm@wisc.edu.
authors.forename: David M
authors.initials: DM
authors.lastname: Gamm
mesh_terms: D019070:Cell Lineage; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology*; D012162:Retinal Degeneration / Q000628:therapy*; D033581:Stem Cell Transplantation
keywords: clinical good manufacturing practice; human induced pluripotent stem cells; induced pluripotent stem cells; photoreceptor; retina; retinal pigmented epithelium; transplantation