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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: Shingo Suzuki (Author), R Geoffrey Sargent (Author), Beate Illek (Author), Horst Fischer (Author), Alaleh Esmaeili-Shandiz (Author), Michael J Yezzi (Author), Albert Lee (Author), Yanu Yang (Author), Soya Kim (Author), Peter Renz (Author), Zhongxia Qi (Author), Jingwei Yu (Author), Marcus O Muench (Author), Ashley I Beyer (Author), Alessander O Guimarães (Author), Lin Ye (Author), Judy Chang (Author), Eli J Fine (Author), Thomas J Cradick (Author), Gang Bao (Author), Meghdad Rahdar (Author), Matthew H Porteus (Author), Tsuyoshi Shuto (Author), Hirofumi Kai (Author), Yuet W Kan (Author), Dieter C Gruenert (Author)
Format: Book
Published: Elsevier, 2016-01-01T00:00:00Z.
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Summary: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.
Item Description:2162-2531
10.1038/mtna.2015.43

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