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Generation of an induced pluripotent stem cell line from a patient with non-syndromic CLN3-associated retinal degeneration and a coisogenic control line.
- Source :
-
Stem cell research [Stem Cell Res] 2018 May; Vol. 29, pp. 245-249. Date of Electronic Publication: 2018 May 01. - Publication Year :
- 2018
-
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.<br /> (Copyright © 2018 The Authors. Published by Elsevier B.V. All rights reserved.)
- Subjects :
- CRISPR-Cas Systems
Cell Line
Female
Gene Editing
Humans
Kruppel-Like Factor 4
MicroRNAs biosynthesis
MicroRNAs genetics
Middle Aged
Transcription Factors biosynthesis
Transcription Factors genetics
Dermis metabolism
Dermis pathology
Fibroblasts metabolism
Fibroblasts pathology
Induced Pluripotent Stem Cells metabolism
Induced Pluripotent Stem Cells pathology
Membrane Glycoproteins genetics
Molecular Chaperones genetics
Retinal Degeneration genetics
Retinal Degeneration metabolism
Retinal Degeneration pathology
Subjects
Details
- Language :
- English
- ISSN :
- 1876-7753
- Volume :
- 29
- Database :
- MEDLINE
- Journal :
- Stem cell research
- Publication Type :
- Academic Journal
- Accession number :
- 29753273
- Full Text :
- https://doi.org/10.1016/j.scr.2018.04.014