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

PubMed ID: 24534198

Author(s): Wright LS, Phillips MJ, Pinilla I, Hei D, Gamm DM. Induced pluripotent stem cells as custom therapeutics for retinal repair: progress and rationale. Exp Eye Res. 2014 Jun;123:161-72. doi: 10.1016/j.exer.2013.12.001. Epub 2014 Feb 16. Review. PMID 24534198

Journal: Experimental Eye Research, Volume 123, Jun 2014

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.