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dc.contributor.author
Suzuki, Shingo
dc.contributor.author
Sargent, R. Geoffrey
dc.contributor.author
Illek, Beate
dc.contributor.author
Fischer, Horst
dc.contributor.author
Esmaeili-Shandiz, Alaleh
dc.contributor.author
Yezzi, Michael J.
dc.contributor.author
Lee, Albert
dc.contributor.author
Yang, Yanu
dc.contributor.author
Kim, Soya
dc.contributor.author
Renz, Peter
dc.contributor.author
Qi, Zhongxia
dc.contributor.author
Yu, Jingwei
dc.contributor.author
Muench, Marcus O.
dc.contributor.author
Beyer, Ashley I.
dc.contributor.author
Guimarães, Alessander O.
dc.contributor.author
Ye, Lin
dc.contributor.author
Chang, J.
dc.contributor.author
Fine, Eli J.
dc.contributor.author
Cradick, Thomas J.
dc.contributor.author
Bao, Gang
dc.contributor.author
Rahdar, Meghdad
dc.contributor.author
Porteus, Matthew H.
dc.contributor.author
Shuto, Tsuyoshi
dc.contributor.author
Kai, Hirofumi
dc.contributor.author
Kan, Yuet W.
dc.contributor.author
Gruenert, Dieter C.
dc.date.accessioned
2019-10-04T15:45:34Z
dc.date.available
2017-06-11T23:49:57Z
dc.date.available
2019-10-04T15:45:34Z
dc.date.issued
2016
dc.identifier.issn
2162-2531
dc.identifier.other
10.1038/mtna.2015.43
en_US
dc.identifier.uri
http://hdl.handle.net/20.500.11850/111974
dc.identifier.doi
10.3929/ethz-b-000111974
dc.description.abstract
Cystic fibrosis (CF) is a recessive inherited disease associated with multiorgan damage that compromises epithelial and inflammatory cell function. Induced pluripotent stem cells (iPSCs) have significantly advanced the potential of developing a personalized cell-based therapy for diseases like CF by generating patient-specific stem cells that can be differentiated into cells that repair tissues damaged by disease pathology. The F508del mutation in airway epithelial cell-derived CF-iPSCs was corrected with small/short DNA fragments (SDFs) and sequence-specific TALENs. An allele-specific PCR, cyclic enrichment strategy gave ≃100-fold enrichment of the corrected CF-iPSCs after six enrichment cycles that facilitated isolation of corrected clones. The seamless SDF-based gene modification strategy used to correct the CF-iPSCs resulted in pluripotent cells that, when differentiated into endoderm/airway-like epithelial cells showed wild-type (wt) airway epithelial cell cAMP-dependent Cl ion transport or showed the appropriate cell-type characteristics when differentiated along mesoderm/hematopoietic inflammatory cell lineage pathways.
en_US
dc.format
application/pdf
en_US
dc.language.iso
en
en_US
dc.publisher
Cell Press
en_US
dc.rights.uri
http://creativecommons.org/licenses/by-nc-nd/4.0/
dc.subject
Cystic fibrosis iPS cells
en_US
dc.subject
IPSC directed differentiation
en_US
dc.subject
Polynucleotide gene targeting/genome editing
en_US
dc.subject
Sequence-specific chimeric endonucleases
en_US
dc.subject
SFHR
en_US
dc.title
TALENs facilitate single-step seamless SDF correction of F508del CFTR in airway epithelial submucosal gland cell-derived CF-iPSCs
en_US
dc.type
Journal Article
dc.rights.license
Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International
dc.date.published
2016-12-14
ethz.journal.title
Molecular Therapy - Nucleic Acids
ethz.journal.volume
5
en_US
ethz.pages.start
e273
en_US
ethz.size
10 p.
en_US
ethz.version.deposit
publishedVersion
en_US
ethz.identifier.wos
ethz.identifier.scopus
ethz.identifier.nebis
010196119
ethz.publication.place
Cambridge
en_US
ethz.publication.status
published
en_US
ethz.date.deposited
2017-06-11T23:50:41Z
ethz.source
ECIT
ethz.identifier.importid
imp5936540d8290d75266
ethz.ecitpid
pub:173465
ethz.eth
yes
en_US
ethz.availability
Open access
en_US
ethz.rosetta.installDate
2017-07-14T12:21:37Z
ethz.rosetta.lastUpdated
2019-10-04T15:45:46Z
ethz.rosetta.versionExported
true
ethz.COinS
ctx_ver=Z39.88-2004&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.atitle=TALENs%20facilitate%20single-step%20seamless%20SDF%20correction%20of%20F508del%20CFTR%20in%20airway%20epithelial%20submucosal%20gland%20cell-derived%20CF-iPSCs&rft.jtitle=Molecular%20Therapy%20-%20Nucleic%20Acids&rft.date=2016&rft.volume=5&rft.spage=e273&rft.issn=2162-2531&rft.au=Suzuki,%20Shingo&Sargent,%20R.%20Geoffrey&Illek,%20Beate&Fischer,%20Horst&Esmaeili-Shandiz,%20Alaleh&rft.genre=article&
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