A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis

Cystic fibrosis (CF) is the most common serious autosomal recessive disease, affecting between 1 and 2,000 and 1 in 4,500 children of Caucasian origin. The main characteristics of CF are malabsorption due to exocrine pancreatic insufficiency, recurrent bacterial infections of the lower exocrine pancreatic insufficiency, recurrent bacterial infections of the lower respiratory tract, abnormal regulation of salt transport across the gastrointestinal and respiratory epithelium, and male infertility due to absence or stenosis of the vas deferens. Early in the disease, the airways become filled with thick purulent mucus. There is chronic bacterial colonization of the airway epithelium, particularly with Pseudomonas aeruginosa, and intense neutrophil inflammation. Over time, the inflammation injures the airways and results in bronchiectasis, parenchymal scarring and destruction, and consequent progressive respiratory failure. In the US, the average lifespan of an individual with CF is 27.6 years.