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The evidence for long-term benefits of restoration of CFTR function continues to grow

Michael P Boyle1,2

  • 11 Division of Pulmonary and Critical Care Medicine Johns Hopkins University School of Medicine Baltimore, Maryland and.

American Journal of Respiratory and Critical Care Medicine
|October 2, 2015
PubMed
Abstract

No abstract available in PubMed .

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Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
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Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
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