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

Abstract

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