Cystic Fibrosis: Translating Molecular Mechanisms into Effective Therapies.

ABSTRACT: Cystic fibrosis is a genetic disease that affects approximately 75,000 individuals around the world. Long regarded as a lethal and life-limiting disease, with the most severe manifestations expressed in the progressive decline of lung function, treatment advances focusing on airway clearance and management of chronic lung infection have resulted in improved outcomes for individuals with cystic fibrosis. These advances have been realized in conjunction with an improved understanding of the genetic basis of this disease, dating back to the discovery of the cystic fibrosis gene in 1989. The identification of the cystic fibrosis gene and the advancement of our understanding of the resultant cystic fibrosis transmembrane conductance regulator protein have led to the development of a new class o

SUBMITTER: Jennings MT 

PROVIDER: S-EPMC6322038 | biostudies-literature | 2018 Aug

REPOSITORIES: biostudies-literature