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Review
. 2018 Mar;17(2S):S28-S34.
doi: 10.1016/j.jcf.2017.09.001. Epub 2017 Sep 19.

Animal and Model Systems for Studying Cystic Fibrosis

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Free PMC article
Review

Animal and Model Systems for Studying Cystic Fibrosis

Bradley H Rosen et al. J Cyst Fibros. .
Free PMC article

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.

Keywords: Animal models; CF lung disease; Cellular systems; Gene editing; Pancreatic disease.

Conflict of interest statement

Conflict of Interest : None of the authors have a conflict of interest relevant to this publication.

Figures

Figure 1
Figure 1
Organ disease presence or absence in various CFTR-knockout (CF) animal models. Organs affected in humans with CF are listed on the left. Check marks below each CF model species indicates disease presence, while question marks denote disease has yet to be evaluated. Organs marked by an X indicate lack of overt disease. NA, not applicable as rats do not have a gallbladder. Superscript denotes references for the relevant studies in the literature.
Figure 2
Figure 2
Uses of CRISPR-Cas9 in CF research and therapies. Delivery of CRISPR-Cas9 System by lentivirus or other innovative vehicles will ease gene editing of polarized epithelia in vitro and in patients.

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