From U Washington/Seattle Children's: Future directions in Cystic Fibrosis research

http://www.ncbi.nlm.nih.gov/pubmed/22312017

Am J Respir Crit Care Med. 2012 Feb 3. [Epub ahead of print]
Future Directions in Early Cystic Fibrosis Lung Disease Research.
Ramsey BW, Banks-Schlegel S, Accurso FJ, Boucher RC, Cutting GR, Engelhardt JF, Guggino WB, Karp CL, Knowles MR, Kolls JK, Lipuma JJ, Lynch S, McCray Jr PB Jr, Rubenstein RC, Singh PK, Sorscher E, Welsh M.
Source
Seattle Children's Research Institute and University of Washington School of Medicine, Seattle, Washington, United States.

Abstract
Since the 1989 discovery that mutations in the CFTR gene cause cystic fibrosis (CF), there has been substantial progress towards 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 is 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 these reasons, the National Heart, Lung, and Blood Institute (NHLBI) convened a working group of extramural experts, entitled "Future Research Directions in Early CF Lung Disease" on September 21-22, 2010, to identify future research directions of great promise in CF. The priority areas identified included: 1) exploring pathogenic mechanisms of early CF lung disease; 2) leveraging newborn screening to elucidate the natural history of early lung disease; 3) developing a spectrum of biomarkers of early lung disease that reflect CF pathophysiology, clinical outcome and response to treatment; 4) exploring the role of genetics/genomics (e.g., modifier genes, gene-environmental interactions, epigenetics, etc.) in early CF pathogenesis; 5) defining early microbiologic events in CF lung disease; and 6) elucidating the initial airway inflammatory, remodeling, and repair mechanisms in CF lung disease.