Using inducible lentiviral vectors to generate induced pluripotent stem cell line ZOCi001-A from peripheral blood cells of a patient with CRB1-/- retinitis pigmentosa


We isolated peripheral blood mononuclear cells from a patient diagnosed with early-onset non-syndromic retinitis pigmentosa caused by compound heterozygous mutations in CRB1 (NM_001257965): c.1369C>T (p.R457X) and c.2027C>T (p.T676M). These cells were transfected with four inducible plasmids encoding human OCT4, SOX2, KLF4 or C-MYC together. Transfected cells were induced to form pluripotent stem cells (iPSCs) expressing the pluripotent stem cell markers SOX2, OCT4, KLF4, SSEA4 and NANOG and presenting a normal karyotype. These cells could be differentiated into three germ layers as well as retinal organoids, and thus provide a valuable cellular model for the study of development of major retina diseases. Copyright © 2020 The Authors. Published by Elsevier B.V. All rights reserved.

Authors Tang X, Liu X, Chen Z, Luo L, Liu X, Deng J, Li DW, Liu Y
Journal Stem cell research
Publication Date 2020 May;45:101817
PubMed 32387899
DOI 10.1016/j.scr.2020.101817

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