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The Cystic Fibrosis Airway Milieu Enhances Rescue of F508del in a Pre-Clinical Model


Extract

Cystic Fibrosis (CF) is a life-shortening genetic disease with autosomal recessive inheritance. Most CF morbidity and mortality is associated with pulmonary disease. The cystic fibrosis transmembrane conductance regulator (CFTR) gene, which codes for a chloride channel required for proper hydration of airway epithelial surfaces, is mutated in CF, resulting in airway dehydration. As a result, CF patients suffer from chronic airway infection, inflammation, and overproduction of mucus, which leads to airway obstruction. During the last decade, basic and clinical research has led to strategies for targeted therapies to successfully restore CFTR function in CF patients.

Footnotes

This manuscript has recently been accepted for publication in the European Respiratory Journal. It is published here in its accepted form prior to copyediting and typesetting by our production team. After these production processes are complete and the authors have approved the resulting proofs, the article will move to the latest issue of the ERJ online. Please open or download the PDF to view this article.

Conflict of interest: Dr. Gentzsch has nothing to disclose.

Conflict of interest: Dr. Cholon has nothing to disclose.

Conflict of interest: Dr. Quinney has nothing to disclose.

Conflict of interest: Dr. Boyles has nothing to disclose.

Conflict of interest: Dr. Martino has nothing to disclose.

Conflict of interest: Dr. Ribeiro has nothing to disclose.

sumber : https://erj.ersjournals.com/content/early/2018/09/27/13993003.01133-2018.abstract

 
 

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