Generation of an induced pluripotent stem cell line (FRIMOi002-A) from a retinitis pigmentosa patient carrying compound heterozygous mutations in USH2A gene.


Journal

Stem cell research
ISSN: 1876-7753
Titre abrégé: Stem Cell Res
Pays: England
ID NLM: 101316957

Informations de publication

Date de publication:
03 2019
Historique:
received: 19 12 2018
revised: 10 01 2019
accepted: 16 01 2019
pubmed: 28 1 2019
medline: 5 9 2019
entrez: 28 1 2019
Statut: ppublish

Résumé

A human induced pluripotent stem cell (iPSC) line was generated from a female patient affected by autosomal recessive retinitis pigmentosa with two mutations in the USH2A gene: c.2209C > T (p.Arg737Ter) and c.8693A > C (p.Tyr2898Ser). Skin fibroblasts were infected with Sendai virus containing the Yamanaka factors and the resulting cells were fully characterized to confirm successful reprogramming. The iPSC line expressed several pluripotency markers, could generate the three germ layers, had a normal karyotype, carried the two USH2A mutations and was free of Sendai virus. This cell line will serve as a model to unravel the pathogenic mechanisms underlying USH2A-associated retinal degeneration.

Identifiants

pubmed: 30685615
pii: S1873-5061(19)30014-5
doi: 10.1016/j.scr.2019.101386
pii:
doi:

Substances chimiques

Extracellular Matrix Proteins 0
USH2A protein, human 0

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

101386

Informations de copyright

Copyright © 2019 The Authors. Published by Elsevier B.V. All rights reserved.

Auteurs

Marina Riera (M)

Fundació de Recerca de l'Institut de Microcirurgia Ocular, Barcelona, Spain; Departament de Genètica, Institut de Microcirurgia Ocular (IMO), Barcelona, Spain; Stem Cell Core Facility, Columbia University, New York (NY), USA; Department of Ophthalmology, Columbia University, New York (NY), USA. Electronic address: genetica.riera@imo.es.

Achchhe Patel (A)

Stem Cell Core Facility, Columbia University, New York (NY), USA.

Borja Corcostegui (B)

Fundació de Recerca de l'Institut de Microcirurgia Ocular, Barcelona, Spain; Departament de Retina, Institut de Microcirurgia Ocular (IMO), Barcelona, Spain.

Stanley Chang (S)

Department of Ophthalmology, Columbia University, New York (NY), USA.

Barbara Corneo (B)

Stem Cell Core Facility, Columbia University, New York (NY), USA.

Janet R Sparrow (JR)

Department of Ophthalmology, Columbia University, New York (NY), USA.

Esther Pomares (E)

Fundació de Recerca de l'Institut de Microcirurgia Ocular, Barcelona, Spain; Departament de Genètica, Institut de Microcirurgia Ocular (IMO), Barcelona, Spain. Electronic address: pomares@imo.es.

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Classifications MeSH