Animal and model systems for studying cystic fibrosis

Bradley H Rosen; Marc Chanson; Lara R Gawenis; Jinghua Liu; Aderonke Sofoluwe; Alice Zoso; John F Engelhardt

doi:10.1016/j.jcf.2017.09.001

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Animal and model systems for studying cystic fibrosis

Journal article

Open access

Peer reviewed

Animal and model systems for studying cystic fibrosis

Bradley H Rosen, Marc Chanson, Lara R Gawenis, Jinghua Liu, Aderonke Sofoluwe, Alice Zoso and John F Engelhardt

Journal of cystic fibrosis, Vol.17(2), pp.S28-S34

03/2018

DOI: 10.1016/j.jcf.2017.09.001

PMCID: PMC5828943

PMID: 28939349

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Abstract

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.

Cellular systems

Animal models

CF lung disease

Gene editing

Pancreatic disease

Details

Title: Subtitle: Animal and model systems for studying cystic fibrosis
Creators: Bradley H Rosen - Department of Anatomy & Cell Biology, Carver College of Medicine at the University of Iowa, Iowa City, IA 52242, USA
Marc Chanson - Department of Pediatrics, Faculty of Medicine at the University of Geneva, Switzerland
Lara R Gawenis - Dalton Cardiovascular Research Center at the University of Missouri, Columbia, MO 65211, USA
Jinghua Liu - Dalton Cardiovascular Research Center at the University of Missouri, Columbia, MO 65211, USA
Aderonke Sofoluwe - Department of Pediatrics, Faculty of Medicine at the University of Geneva, Switzerland
Alice Zoso - Department of Pediatrics, Faculty of Medicine at the University of Geneva, Switzerland
John F Engelhardt - Department of Anatomy & Cell Biology, Carver College of Medicine at the University of Iowa, Iowa City, IA 52242, USA
Resource Type: Journal article
Publication Details: Journal of cystic fibrosis, Vol.17(2), pp.S28-S34
Publisher: Elsevier B.V
DOI: 10.1016/j.jcf.2017.09.001
PMID: 28939349
PMCID: PMC5828943
ISSN: 1569-1993
eISSN: 1873-5010
Grant note: DOI: 10.13039/100000002, name: National Institutes of Health, award: P30 DK54759, R24HL123482, DK096518; DOI: 10.13039/100000897, name: Cystic Fibrosis Foundation, award: ROSEN17I0, ROSEN17XX0; name: Swiss CF Foundation; DOI: 10.13039/100001935, name: Foundation ABCF; DOI: 10.13039/501100006342, name: Association Vaincre la Mucoviscidose; DOI: 10.13039/501100001711, name: Swiss National Science Foundation, award: 310030_172909/1
Language: English
Date published: 03/2018
Academic Unit: Roy J. Carver Department of Biomedical Engineering; Pulmonary, Critical Care, and Occupational Medicine; Anatomy and Cell Biology; Radiation Oncology; Internal Medicine
Record Identifier: 9984025413602771

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