Journal
JOURNAL OF CYSTIC FIBROSIS
Volume 17, Issue 2, Pages S28-S34Publisher
ELSEVIER SCIENCE BV
DOI: 10.1016/j.jcf.2017.09.001
Keywords
Animal models; Cellular systems; CF lung disease; Pancreatic disease; Gene editing
Categories
Funding
- National Institutes of Health [P30 DK54759, R24HL123482, DK096518]
- Cystic Fibrosis Foundation [ROSEN17I0, ROSEN17XX0]
- Swiss CF Foundation
- Foundation ABCF
- Association Vaincre la Mucoviscidose
- Swiss National Science Foundation [310030_172909/1]
- NATIONAL HEART, LUNG, AND BLOOD INSTITUTE [P01HL051670, R24HL123482] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES [R24DK096518, R01DK047967, P30DK054759] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF ENVIRONMENTAL HEALTH SCIENCES [P30ES005605] Funding Source: NIH RePORTER
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The cystic fibrosis (CF) field is the beneficiary of five species of animal models that lack functional cystic fibrosis transmembrane conductance regulator (CFTR) channel. These models are rapidly informing mechanisms of disease pathogenesis and CFTR function regardless of how faithfully a given organ reproduces the human CF phenotype. New approaches of genetic engineering with RNA-guided nucleases are rapidly expanding both the potential types of models available and the approaches to correct the CFTR defect. The application of new CRISPR/Cas9 genome editing techniques are similarly increasing capabilities for in vitro modeling of CFTR functions in cell lines and primary cells using air liquid interface cultures and organoids. Gene editing of CFTR mutations in somatic stem cells and induced pluripotent stem cells is also transforming gene therapy approaches for CF. This short review evaluates several areas that are key to building animal and cell systems capable of modeling CF disease and testing potential treatments. (C) 2017 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
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