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Exp Eye Res. 2014 Jun;123:161-72. doi: 10.1016/j.exer.2013.12.001. Epub 2014 Feb 16.

Induced pluripotent stem cells as custom therapeutics for retinal repair: progress and rationale.

Author information

1
Waisman Center, University of Wisconsin, Madison, WI, USA; McPherson Eye Research Institute, University of Wisconsin, Madison, WI, USA.
2
Department of Ophthalmology, Lozano Blesa Hospital and Aragones Health Sciences Institute, Zaragoza, Spain.
3
Waisman Center, University of Wisconsin, Madison, WI, USA.
4
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.

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.

KEYWORDS:

clinical good manufacturing practice; human induced pluripotent stem cells; induced pluripotent stem cells; photoreceptor; retina; retinal pigmented epithelium; transplantation

PMID:
24534198
PMCID:
PMC4047146
DOI:
10.1016/j.exer.2013.12.001
[Indexed for MEDLINE]
Free PMC Article

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