Generation of an induced pluripotent stem cell line from a patient with non-syndromic CLN3-associated retinal degeneration and a coisogenic control line
Summary
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. Copyright © 2018 The Authors. Published by Elsevier B.V. All rights reserved.
| Authors | Zhang X, Zhang D, Chen SC, Lamey T, Thompson JA, McLaren T, De Roach JN, Chen FK, McLenachan S |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2018 May;29:245-249 |
| PubMed | 29753273 |
| DOI | 10.1016/j.scr.2018.04.014 |