[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 …

[HTML][HTML] Phenotypes of CF rabbits generated by CRISPR/Cas9-mediated disruption of the CFTR gene

J Xu, A Livraghi-Butrico, X Hou, C Rajagopalan… - JCI insight, 2021 - ncbi.nlm.nih.gov
Existing animal models of cystic fibrosis (CF) have provided key insights into CF
pathogenesis but have been limited by short lifespans, absence of key phenotypes, and/or …

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 …

Innovative therapeutic strategies for cystic fibrosis: moving forward to CRISPR technique

M Marangi, G Pistritto - Frontiers in pharmacology, 2018 - frontiersin.org
One of the most revolutionary technologies in recent years in the field of molecular biology is
CRISPR-Cas9. CRISPR technology is a promising tool for gene editing that provides …

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 …

Sequential targeting of CFTR by BAC vectors generates a novel pig model of cystic fibrosis

N Klymiuk, L Mundhenk, K Kraehe, A Wuensch… - Journal of molecular …, 2012 - Springer
Cystic fibrosis (CF) is the most common lethal inherited disease in Caucasians and is
caused by mutations in the CFTR gene. The disease is incurable and medical treatment is …

A G542X cystic fibrosis mouse model for examining nonsense mutation directed therapies

DR McHugh, MS Steele, DM Valerio, A Miron… - PLoS …, 2018 - journals.plos.org
Nonsense mutations are present in 10% of patients with CF, produce a premature
termination codon in CFTR mRNA causing early termination of translation, and lead to lack …

[HTML][HTML] Lentiviral-mediated phenotypic correction of cystic fibrosis pigs

AL Cooney, MH Abou Alaiwa, VS Shah, DC Bouzek… - JCI insight, 2016 - ncbi.nlm.nih.gov
Cystic Fibrosis (CF) is an autosomal recessive disease caused by mutations in CF
transmembrane conductance regulator (CFTR), resulting in defective anion transport …

Production of CFTR-null and CFTR-ΔF508 heterozygous pigs by adeno-associated virus–mediated gene targeting and somatic cell nuclear transfer

CS Rogers, Y Hao, T Rokhlina… - The Journal of …, 2008 - Am Soc Clin Investig
Progress toward understanding the pathogenesis of cystic fibrosis (CF) and developing
effective therapies has been hampered by lack of a relevant animal model. CF mice fail to …