Production of CFTR-ΔF508 rabbits

D Yang, X Liang, B Pallas, M Hoenerhoff, Z Ren… - Frontiers in …, 2021 - frontiersin.org
Cystic Fibrosis (CF) is a lethal autosomal recessive disease caused by mutations in the
gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR). The most …

[HTML][HTML] Sheep models of F508del and G542X cystic fibrosis mutations show cellular responses to human therapeutics

IV Perisse, Z Fan, A Van Wettere, Y Liu… - FASEB …, 2021 - ncbi.nlm.nih.gov
Cystic Fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane
conductance regulator (CFTR) gene. The F508del and G542X are the most common …

Characterization of two rat models of cystic fibrosis—KO and F508del CFTR—Generated by Crispr‐Cas9

E Dreano, M Bacchetta, J Simonin… - Animal models and …, 2019 - Wiley Online Library
Background Genetically engineered animals are essential for gaining a proper
understanding of the disease mechanisms of cystic fibrosis (CF). The rat is a relevant …

Animal and model systems for studying cystic fibrosis

BH Rosen, M Chanson, LR Gawenis, J Liu… - Journal of Cystic …, 2018 - Elsevier
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 …

[HTML][HTML] Phenotypic characterization and comparison of cystic fibrosis rat models generated using CRISPR/Cas9 gene editing

A McCarron, P Cmielewski, N Reyne, C McIntyre… - The American journal of …, 2020 - Elsevier
Animal models of cystic fibrosis (CF) are essential for investigating disease mechanisms and
trialing potential therapeutics. This study generated two CF rat models using clustered …

A novel G542X CFTR rat model of cystic fibrosis is sensitive to nonsense mediated decay

J Sharma, J Abbott, L Klaskala, G Zhao… - Frontiers in …, 2020 - frontiersin.org
Nonsense mutations that lead to the insertion of a premature termination codon (PTC) in the
cystic fibrosis transmembrane conductance regulator (CFTR) transcript affect 11% of …

[HTML][HTML] A sheep model of cystic fibrosis generated by CRISPR/Cas9 disruption of the CFTR gene

Z Fan, IV Perisse, CU Cotton, M Regouski, Q Meng… - JCI insight, 2018 - ncbi.nlm.nih.gov
Cystic fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane
conductance regulator (CFTR) gene. The major cause of limited life span in CF patients is …

Genome editing for cystic fibrosis

G Wang - Cells, 2023 - mdpi.com
Cystic fibrosis (CF) is a monogenic recessive genetic disorder caused by mutations in the
CF Transmembrane-conductance Regulator gene (CFTR). Remarkable progress in basic …

Powerful tools for genetic modification: Advances in gene editing

EA Roesch, ML Drumm - Pediatric Pulmonology, 2017 - Wiley Online Library
Recent discoveries and technical advances in genetic engineering, methods called gene or
genome editing, provide hope for repairing genes that cause diseases like cystic fibrosis …

On the corner of models and cure: gene editing in cystic fibrosis

M Ensinck, A Mottais, C Detry, T Leal… - Frontiers in …, 2021 - frontiersin.org
Cystic fibrosis (CF) is a severe genetic disease for which curative treatment is still lacking.
Next generation biotechnologies and more efficient cell-based and in vivo disease models …