TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs

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...

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Main Authors:	Suzuki, Shingo, Sargent, R Geoffrey, Illek, Beate, Fischer, Horst, Esmaeili-Shandiz, Alaleh, Yezzi, Michael J, Lee, Albert, Yang, Yanu, Kim, Soya, Renz, Peter, Qi, Zhongxia, Yu, Jingwei, Muench, Marcus O, Beyer, Ashley I, Guimarães, Alessander O, Ye, Lin, Chang, Judy, Fine, Eli J, Cradick, Thomas J, Bao, Gang, Rahdar, Meghdad, Porteus, Matthew H, Shuto, Tsuyoshi, Kai, Hirofumi, Kan, Yuet W, Gruenert, Dieter C
Format:	Online
Language:	English
Published:	Nature Publishing Group 2016
Online Access:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5012545/

id	pubmed-5012545
recordtype	oai_dc
spelling	pubmed-50125452016-09-16 TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs Suzuki, Shingo Sargent, R Geoffrey Illek, Beate Fischer, Horst Esmaeili-Shandiz, Alaleh Yezzi, Michael J Lee, Albert Yang, Yanu Kim, Soya Renz, Peter Qi, Zhongxia Yu, Jingwei Muench, Marcus O Beyer, Ashley I Guimarães, Alessander O Ye, Lin Chang, Judy Fine, Eli J Cradick, Thomas J Bao, Gang Rahdar, Meghdad Porteus, Matthew H Shuto, Tsuyoshi Kai, Hirofumi Kan, Yuet W Gruenert, Dieter C Original Article 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. Nature Publishing Group 2016-01 2016-01-05 /pmc/articles/PMC5012545/ /pubmed/26730810 http://dx.doi.org/10.1038/mtna.2015.43 Text en Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy http://creativecommons.org/licenses/by-nc-nd/4.0/ This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International License. The images or other third party material in this article are included in the article's Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/
repository_type	Open Access Journal
institution_category	Foreign Institution
institution	US National Center for Biotechnology Information
building	NCBI PubMed
collection	Online Access
language	English
format	Online
author	Suzuki, Shingo Sargent, R Geoffrey Illek, Beate Fischer, Horst Esmaeili-Shandiz, Alaleh Yezzi, Michael J Lee, Albert Yang, Yanu Kim, Soya Renz, Peter Qi, Zhongxia Yu, Jingwei Muench, Marcus O Beyer, Ashley I Guimarães, Alessander O Ye, Lin Chang, Judy Fine, Eli J Cradick, Thomas J Bao, Gang Rahdar, Meghdad Porteus, Matthew H Shuto, Tsuyoshi Kai, Hirofumi Kan, Yuet W Gruenert, Dieter C
spellingShingle	Suzuki, Shingo Sargent, R Geoffrey Illek, Beate Fischer, Horst Esmaeili-Shandiz, Alaleh Yezzi, Michael J Lee, Albert Yang, Yanu Kim, Soya Renz, Peter Qi, Zhongxia Yu, Jingwei Muench, Marcus O Beyer, Ashley I Guimarães, Alessander O Ye, Lin Chang, Judy Fine, Eli J Cradick, Thomas J Bao, Gang Rahdar, Meghdad Porteus, Matthew H Shuto, Tsuyoshi Kai, Hirofumi Kan, Yuet W Gruenert, Dieter C TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs
author_facet	Suzuki, Shingo Sargent, R Geoffrey Illek, Beate Fischer, Horst Esmaeili-Shandiz, Alaleh Yezzi, Michael J Lee, Albert Yang, Yanu Kim, Soya Renz, Peter Qi, Zhongxia Yu, Jingwei Muench, Marcus O Beyer, Ashley I Guimarães, Alessander O Ye, Lin Chang, Judy Fine, Eli J Cradick, Thomas J Bao, Gang Rahdar, Meghdad Porteus, Matthew H Shuto, Tsuyoshi Kai, Hirofumi Kan, Yuet W Gruenert, Dieter C
author_sort	Suzuki, Shingo
title	TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs
title_short	TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs
title_full	TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs
title_fullStr	TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs
title_full_unstemmed	TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs
title_sort	talens facilitate single-step seamless sdf correction of f508del cftr in airway epithelial submucosal gland cell-derived cf-ipscs
description	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.
publisher	Nature Publishing Group
publishDate	2016
url	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5012545/
_version_	1613644205796622336

TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs

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