Generation of an induced pluripotent stem cell line from a patient with non-syndromic CLN3-associated retinal degeneration and a coisogenic control line

Stem Cell Research
Xiao ZhangSamuel McLenachan

Abstract

We report the generation of the human iPSC line LEIi004-A from a patient with late-onset non-syndromic retinitis pigmentosa caused by compound heterozygous mutations in the CLN3 gene. Reprogramming of primary dermal fibroblasts was performed using episomal plasmids containing OCT4, SOX2, KLF4, L-MYC, LIN28, shRNA for p53 and mir302/367 microRNA. To create a coisogenic control line, one CLN3 variant was corrected in the patient-iPSC using CRISPR/Cas9 gene editing to generate the iPSC line LEIi004-A-1.

Citations

Nov 15, 2019·Cell Transplantation·Aline Yen Ling Wang, Charles Yuen Yung Loh
Jul 19, 2021·Neuroscience Letters·Susan L Cotman, Stéphane Lefrancois

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