Work

1. Species Choice for Animal Modeling Cystic Fibrosis and Gene Therapy to the Airway

Year: 2006

Type: article

Abstract: The development of gene therapies for cystic fibrosis (CF) lung disease requires animal models that can appropriately reproduce the human disease phenotype and human vector biology of gene transfer to the airway. However, the efficacy of certain viral vector systems for gene delivery to the airways in CF mice has been notably different than that in humanized systems. We systematically analyzed gene transfer properties of in vitro polarized airway epithelia from a number of species including human, mouse, pig, ferret, and non-human primates using air-liquid interface (ALI) models.

Source: Molecular Therapy

DOI: 10.1016/j.ymthe.2006.08.011