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.

Animal and model systems for studying cystic fibrosis / Rosen, B. H.; Chanson, M.; Gawenis, L. R.; Liu, J.; Sofoluwe, A.; Zoso, A.; Engelhardt, J. F.. - In: JOURNAL OF CYSTIC FIBROSIS. - ISSN 1569-1993. - ELETTRONICO. - 17:2(2018), pp. S28-S34. [10.1016/j.jcf.2017.09.001]

Animal and model systems for studying cystic fibrosis

Zoso A.;
2018

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.
File in questo prodotto:
File Dimensione Formato  
JCF_review - Proof.pdf

accesso aperto

Descrizione: Articolo principale
Tipologia: 2. Post-print / Author's Accepted Manuscript
Licenza: Creative commons
Dimensione 384.02 kB
Formato Adobe PDF
384.02 kB Adobe PDF Visualizza/Apri
Animal and model systems for studying cystic fibrosis _ Elsevier Enhanced Reader.pdf

non disponibili

Tipologia: 2a Post-print versione editoriale / Version of Record
Licenza: Non Pubblico - Accesso privato/ristretto
Dimensione 4.71 MB
Formato Adobe PDF
4.71 MB Adobe PDF   Visualizza/Apri   Richiedi una copia
Pubblicazioni consigliate

Caricamento pubblicazioni consigliate

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11583/2784960