Using inducible lentiviral vectors to generate induced pluripotent stem cell line ZOCi001-A from peripheral blood cells of a patient with CRB1-/- retinitis pigmentosa.
Stem Cell Res
; 45: 101817, 2020 05.
Article
em En
| MEDLINE
| ID: mdl-32387899
ABSTRACT
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.
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Base de dados:
MEDLINE
Assunto principal:
Retinose Pigmentar
/
Células-Tronco Pluripotentes Induzidas
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article