Animal and Model Systems for Studying Cystic Fibrosis

Overview

Journal J Cyst Fibros

Publisher Elsevier

Specialty Pulmonary Medicine

Date 2017 Sep 24

PMID 28939349

Citations 49

Authors

Bradley H Rosen

Marc Chanson

Lara R Gawenis

Jinghua Liu

Aderonke Sofoluwe

Alice Zoso

John F Engelhardt

Affiliations

Soon will be listed here.

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.

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