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

Mutation-agnostic treatments such as airway gene therapy have the potential to treat any individual with cystic fibrosis (CF), irrespective of their CF transmembrane conductance regulator (CFTR) gene variants. The aim of this study was to employ two CF rat models, Phe508del and CFTR knockout to assess the comparative effectiveness of CFTR modulators and lentiviral vector-mediated gene therapy.

Citation:
McCarron A, Ling KM, Montgomery ST, Martinovich KM, Cmielewski P, Rout-Pitt N, Kicic A, Parsons D, Donnelley M. Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models. Gene Ther. 2024.

Keywords:
Cystic fibrosis; gene therapy; CFTR modulators

Abstract:
Mutation-agnostic treatments such as airway gene therapy have the potential to treat any individual with cystic fibrosis (CF), irrespective of their CF transmembrane conductance regulator (CFTR) gene variants. The aim of this study was to employ two CF rat models, Phe508del and CFTR knockout to assess the comparative effectiveness of CFTR modulators and lentiviral vector-mediated gene therapy.