Future directions in early cystic fibrosis lung disease research: an NHLBI workshop report.

ABSTRACT: Since the 1989 discovery that mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause cystic fibrosis (CF), there has been substantial progress toward understanding the molecular basis for CF lung disease, leading to the discovery and development of new therapeutic approaches. However, the earliest impact of the loss of CFTR function on airway physiology and structure and its relationship to initial infection and inflammation are poorly understood. Universal newborn screening for CF in the United States represents an unprecedented opportunity for investigating CF clinical manifestations very early in life. Recently developed animal models with pulmonary phenotypic manifestations also provide a window into the early consequences of this genetic disorder. For t

SUBMITTER: Ramsey BW 

PROVIDER: S-EPMC3360572 | biostudies-literature | 2012 Apr

REPOSITORIES: biostudies-literature