Generation of a gene-corrected isogenic control iPSC line from cystic fibrosis patient-specific iPSCs homozygous for p.Phe508del mutation mediated by TALENs and ssODN.
Stem Cell Res
; 23: 95-97, 2017 08.
Article
en En
| MEDLINE
| ID: mdl-28925369
ABSTRACT
Cystic fibrosis (CF) is a monogenetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which affects multiple organs. Human induced pluripotent stem cells (iPSCs) derived from CF patients and the generation of isogeneic gene-corrected control cell lines enable disease modelling, drug discovery or toxicological studies and therefore the development of CF patient-specific therapies. We have previously generated a hiPSC line from a CF patient homozygous for the p.Phe508del mutation. Here we used TALENs and single-stranded oligonucleotides to correct the mutated triplet in our CF-iPSC line.
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Oligodesoxirribonucleótidos
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Fibrosis Quística
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Células Madre Pluripotentes Inducidas
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Nucleasas de los Efectores Tipo Activadores de la Transcripción
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Mutación
Límite:
Humans
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Male
Idioma:
En
Revista:
Stem Cell Res
Año:
2017
Tipo del documento:
Article