Adenovirus-Mediated Transfer of the CFTR Gene to Lung of Nonhuman Primates: Biological Efficacy Study

Abstract

Overview summary Recombinant adenoviruses hold tremendous promise for gene therapies of lung disease in cystic fibrosis (CF). An evaluation of the feasibility and safety of this technology in nonhuman primates is critical in the design of clinical protocols. In a series of two papers, Wilson and colleagues describe an extensive study in baboons designed to evaluate the feasibility and safety of direct instillation of CF transmembrane conductance regulator (CFTR)-expressing adenoviruses into the airway. This paper by Engelhardt et al., addresses the biological efficacy of E1-deleted adenoviruses for gene therapy of CF lung disease.