A Preliminary Study of Aerosolized Recombinant Human Deoxyribonuclease I in the Treatment of Cystic Fibrosis

CYSTIC fibrosis is a lethal recessive genetic disorder caused by mutations of the cystic fibrosis transmembrane conductance regulator, a gene comprising 27 exons and 250 kilobases that resides on chromosome 7.1 2 3 4 5 The most important clinical manifestation of cystic fibrosis is chronic progressive lung disease, which despite aggressive antibiotic therapy and chest physiotherapy, remains the principal cause of disability and death.1 Airway secretions play a major part in the respiratory dysfunction in cystic fibrosis. The secretions are thick, viscous, and difficult to expectorate, and they obstruct airways and contribute to reduced lung volumes and expiratory flow rates.1 , 2 , 6 The presence of high . . .