Predicting clinical efficacy of lung gene delivery in people with cystic fibrosis: which in vitro and in vivo models best predict outcomes in human lungs?

INTRODUCTION: and preclinical work, and lung function improvements in people with CF. AREAS COVERED: animal models for CF research from databases including Web of Science and PubMed. Data were used to evaluate the performance of inhaled nucleic acid delivery systems for CF and examine why positive outcomes in these models fail to predict clinical responses. EXPERT OPINION: air-liquid interface models that accurately recapitulate the CF airways are important starting points, followed by delivery and functional evaluation in CF animal models that closely resemble human lung physiology and disease phenotype, including pigs and ferrets.