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12. Mai 2014

ERS Monograph on Cystic Fibrosis

News 13-2014 DE

DZL Director Marcus Mall and DZL Scientific Advisory Board member Stuart Elborn co-edited the European Respiratory Monograph on Cystic Fibrosis (CF)

DZL Director Marcus Mall of the Translational Lung Research Center Heidelberg and DZL Scientific Advisory Board member Stuart Elborn co-edited the European Respiratory Monograph on Cystic Fibrosis (CF) on the occasion of the 25th anniversary of the discovery of the CFTR gene. The discovery of the CFTR gene is a significant milestone in the search for a cure for CF.  25 years on we know that there are in excess of 1500 known mutations in the gene, highlighting the complex pathogenesis of CF lung disease. The introduction of high-throughput screening greatly advanced the development of the first CFTR mutation-specific therapies for CF. This breakthrough in CF research may also serve as a model for other rare lung diseases.  Another important milestone has been the generation of animal models for mechanistic studies and pre-clinical testing of novel mucolytic, anti-inflammatory and antibacterial therapies. While the hunt for a curative therapy is still ongoing, significant progress has been achieved in the way CF is treated in the clinic. Of great benefit has been the introduction of newborn screening for CF, which allows for early and even preventive treatment with disease modifying therapies. The ERS monograph highlights important recent developments and discusses the next steps that will be required for further improvement of life expectancy and quality of life of patients with CF.  The DZL has an active program  in CF research involving close to 20 investigators across four DZL centers

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