Phenotypic Characterization and Comparison of Phe508del and Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Knockout Rat Models of Cystic Fibrosis Generated by CRISPR/Cas9 Gene Editing

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Animal models of cystic fibrosis (CF) are essential for investigating disease mechanisms and trialling potential therapeutics. This study generated two CF rat models using clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR associated protein 9 (Cas9) gene editing. One rat model carries the common human Phe508del (ΔF508) CF transmembrane conductance regulator (CFTR) mutation, whereas the second is a CFTR knockout model. Phenotype was characterized using a range of functional and histological assessments including nasal potential difference to measure electrophysiological function in the upper airways, RNAscope in situ hybridization and quantitative PCR to assess CFTR mRNA expression in the lungs, immunohistochemistry to localize CFTR protein in the airways, and histopathological assessments in a range of tissues.... More of this in  The American Journal of Pathology Blog.

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