Journal article
Future Directions in Early Cystic Fibrosis Lung Disease Research: An NHLBI Workshop Report
American journal of respiratory and critical care medicine, Vol.185(8), pp.887-892
04/15/2012
DOI: 10.1164/rccm.201111-2068WS
PMCID: PMC3360572
PMID: 22312017
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 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 reflects CF pathophysiology, clinical outcome, and response to treatment; (
4
) exploring the role of genetics/genomics (e.g., modifier genes, gene–environmental interactions, and epigenetics) in early CF pathogenesis; (
5
) defining early microbiological events in CF lung disease; and (
6
) elucidating the initial airway inflammatory, remodeling, and repair mechanisms in CF lung disease.
Details
- Title: Subtitle
- Future Directions in Early Cystic Fibrosis Lung Disease Research: An NHLBI Workshop Report
- Creators
- Bonnie W Ramsey - Seattle Children's Research Institute andSusan Banks-Schlegel - National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MarylandFrank J Accurso - University of Colorado School of Medicine, Denver, ColoradoRichard C Boucher - University of North Carolina School of Medicine, Chapel Hill, North CarolinaGarry R Cutting - Johns Hopkins School of Medicine, Baltimore, MarylandJohn F Engelhardt - University of Iowa School of Medicine, Iowa City, IowaWilliam B Guggino - Johns Hopkins School of Medicine, Baltimore, MarylandChristopher L Karp - Division of Molecular Immunology, Cincinnati Children's Hospital Research Foundation, and the University of Cincinnati College of Medicine, Cincinnati, OhioMichael R Knowles - University of North Carolina School of Medicine, Chapel Hill, North CarolinaJay K Kolls - Louisiana State University School of Medicine at New Orleans, New Orleans, LouisianaJohn J LiPuma - University of Michigan School of Public Health, Ann Arbor, MichiganSusan Lynch - University of California at San Francisco School of Medicine, San Francisco, CaliforniaPaul B McCray - University of Iowa Children's Hospital, Iowa City, IowaRonald C Rubenstein - Children's Hospital of Philadelphia and Perelman School of Medicine, University of Pennsylvania, Philadelphia, PennsylvaniaPradeep K Singh - University of Washington School of Medicine, Seattle, WashingtonEric Sorscher - University of Alabama at Birmingham School of Medicine, Birmingham, Alabama; andMichael Welsh - Howard Hughes Medical Institute, Roy J. and Lucille A. Carver College of Medicine, University of Iowa, Iowa City, Iowa
- Resource Type
- Journal article
- Publication Details
- American journal of respiratory and critical care medicine, Vol.185(8), pp.887-892
- DOI
- 10.1164/rccm.201111-2068WS
- PMID
- 22312017
- PMCID
- PMC3360572
- NLM abbreviation
- Am J Respir Crit Care Med
- ISSN
- 1073-449X
- eISSN
- 1535-4970
- Publisher
- American Thoracic Society
- Language
- English
- Date published
- 04/15/2012
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Neurology; Molecular Physiology and Biophysics; Pulmonary, Critical Care, and Occupational Medicine; Microbiology and Immunology; Pulmonary Medicine; Anatomy and Cell Biology; Stead Family Department of Pediatrics; Radiation Oncology; Fraternal Order of Eagles Diabetes Research Center; Neurosurgery; Internal Medicine
- Record Identifier
- 9984025343002771
Metrics
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