Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo.

Traditional gene therapy vectors have demonstrated limited utility for treatment of chronic lung diseases such as cystic fibrosis (CF). Herein we describe a vector based on a Filovirus envelope protein-pseudotyped HIV vector, which we chose after systematically evaluating multiple strategies. The vector efficiently transduces intact airway epithelium from the apical surface, as demonstrated in bothin vitro and in vivo model systems. This shows the potential of pseudo typing in expanding the utility of lentiviral vectors. Pseudotyped lentiviral vectors may hold promise for the treatment of CF. [ABSTRACT FROM AUTHOR]