Building global development strategies for cf therapeutics during a transitional cftr modulator era
نویسندگان
چکیده
منابع مشابه
Optimizing Nasal Potential Difference Analysis for CFTR Modulator Development: Assessment of Ivacaftor in CF Subjects with the G551D-CFTR Mutation
Nasal potential difference (NPD) is used as a biomarker of the cystic fibrosis transmembrane conductance regulator (CFTR) and epithelial sodium channel (ENaC) activity. We evaluated methods to detect changes in chloride and sodium transport by NPD based on a secondary analysis of a Phase II CFTR-modulator study. Thirty-nine subjects with CF who also had the G551D-CFTR mutation were randomized t...
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The cystic fibrosis transmembrane conductance regulator (CFTR) protein is a small conductance chloride ion channel that may interact directly with other channels including the epithelial sodium channel (ENaC). CFTR is known to be more abundant in the airway epithelium during the second trimester of human development than after birth. This could be a consequence of the change in function of the ...
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Cystic fibrosis (CF), the most common life-shortening genetic disorder in Caucasians, affects approximately 70 000 individuals worldwide. In 1998, the Cystic Fibrosis Foundation (CFF) launched the CF Therapeutics Development Network (CF-TDN) as a central element of its Therapeutics Development Programme. Designed to accelerate the clinical evaluation of new therapies needed to fulfil the CFF mi...
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University of Pittsburgh, Swanson School of Engineering Submission Date 11.01.2016 1 Disclaimer—This paper partially fulfills a writing requirement for first year (freshman) engineering students at the University of Pittsburgh Swanson School of Engineering. This paper is a student, not a professional, paper. This paper is based on publicly available information and may not provide complete anal...
متن کاملCFTR is a modulator of airway inflammation.
ALTHOUGH IT HAS BEEN 17 YEARS since the identification of the cystic fibrosis transmembrane ion receptor (CFTR) gene and protein, it remains enigmatic how abnormalities in CFTR can cause chronic and persistent pulmonary infection and inflammation that lead to bronchiectasis and end-stage lung disease (9). Because CFTR regulates transepithelial chloride and sodium conductance, the most prominent...
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ژورنال
عنوان ژورنال: Journal of Cystic Fibrosis
سال: 2020
ISSN: 1569-1993
DOI: 10.1016/j.jcf.2020.05.011