Lentiviral vector gene therapy and cftr modulators show comparable effectiveness in cystic fibrosis rat airway models

HIGHLIGHTS

• What: The aim of this study was to characterise CFTR function in airliquid interface (ALI) cultures derived from the trachea of Phe508del and KO rats, and to determine if Phe508del rat airway cells are responsive to ETI. Animals This study was approved by the University of Adelaide animal ethics committee under application M-2019-038 and was conducted in accordance with ARRIVE guidelines. The study demonstrated that CF rat trachea-derived ALI cultures could be successfully established, with immunohistochemical staining confirming the presence of cell types including ciliated, basal, and goblet cells. The authors went on to . . .
  

   

  

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