期刊
EXPERIMENTAL EYE RESEARCH
卷 123, 期 -, 页码 161-172出版社
ACADEMIC PRESS LTD- ELSEVIER SCIENCE LTD
DOI: 10.1016/j.exer.2013.12.001
关键词
human induced pluripotent stem cells; induced pluripotent stem cells; photoreceptor; retinal pigmented epithelium; retina; clinical good manufacturing practice; transplantation
资金
- Foundation Fighting Blindness Wynn-Gund Translational Research Award
- Reeves Foundation
- Choroideremia Research Foundation
- Muskingum County Community Foundation
- NIH [R01 EY021218, HD03352]
- UW-Madison McPherson Eye Research Institute
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. (C) 2013 The Authors. Published by Elsevier Ltd. All rights reserved.
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