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: | , , , , , , , , , , , , , , , , , , , , , , , , , |
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Elsevier,
2016-01-01T00:00:00Z.
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| 700 | 1 | 0 | |a Horst Fischer |e author |
| 700 | 1 | 0 | |a Alaleh Esmaeili-Shandiz |e author |
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| 700 | 1 | 0 | |a Judy Chang |e author |
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| 700 | 1 | 0 | |a Dieter C Gruenert |e author |
| 245 | 0 | 0 | |a TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs |
| 260 | |b Elsevier, |c 2016-01-01T00:00:00Z. | ||
| 500 | |a 2162-2531 | ||
| 500 | |a 10.1038/mtna.2015.43 | ||
| 520 | |a 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. | ||
| 546 | |a EN | ||
| 690 | |a cystic fibrosis iPS cells | ||
| 690 | |a iPSC directed differentiation | ||
| 690 | |a polynucleotide gene targeting/genome editing | ||
| 690 | |a sequence-specific chimeric endonucleases | ||
| 690 | |a SFHR | ||
| 690 | |a Therapeutics. Pharmacology | ||
| 690 | |a RM1-950 | ||
| 655 | 7 | |a article |2 local | |
| 786 | 0 | |n Molecular Therapy: Nucleic Acids, Vol 5, Iss C (2016) | |
| 787 | 0 | |n http://www.sciencedirect.com/science/article/pii/S2162253116300099 | |
| 787 | 0 | |n https://doaj.org/toc/2162-2531 | |
| 856 | 4 | 1 | |u https://doaj.org/article/aa7f6c58c90142c2a88dd3a6fd6f2d5f |z Connect to this object online. |