当前位置： SCI文献检索 > Molecular Therapy-Nucleic Acids期刊下所有文献 > TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs.

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

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.

journal_name

Mol Ther Nucleic Acids

journal_title

Molecular therapy. Nucleic acids

authors

Suzuki S,Sargent RG,Illek B,Fischer H,Esmaeili-Shandiz A,Yezzi MJ,Lee A,Yang Y,Kim S,Renz P,Qi Z,Yu J,Muench MO,Beyer AI,Guimarães AO,Ye L,Chang J,Fine EJ,Cradick TJ,Bao G,Rahdar M,Porteus MH,Shuto T,Kai H,

doi

10.1038/mtna.2015.43

subject

Has Abstract

pub_date

2016-01-05 00:00:00

pages

e273

issn

2162-2531

pii

mtna201543

journal_volume

5

pub_type

杂志文章

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