The influence of CFTR complex alleles on precision therapy of cystic fibrosis
Author:
Funder
French cystic fibrosis association Vaincre la mucoviscidose
Publisher
Elsevier BV
Subject
Pulmonary and Respiratory Medicine,Pediatrics, Perinatology, and Child Health
Reference27 articles.
1. Cystic fibrosis genetics: from molecular understanding to clinical application;Cutting;Nat Rev Genet,2015
2. From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations;Veit;Mol Biol Cell,2016
3. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation;Ramsey;N Engl J Med,2011
4. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809;Van Goor;Proc Natl Acad Sci U S A,2011
5. Lumacaftor–Ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR;Wainwright;N Engl J Med,2015
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1. The novel CFTR haplotype E583G/F508del in CFTR-related disorder;Molecular Biology Reports;2024-07-25
2. Advances in the Study of Common and Rare CFTR Complex Alleles Using Intestinal Organoids;Journal of Personalized Medicine;2024-01-23
3. The Effect of Complex Alleles of the CFTR Gene on the Clinical Manifestations of Cystic Fibrosis and the Effectiveness of Targeted Therapy;International Journal of Molecular Sciences;2023-12-21
4. Charting the path to expanded access for CFTR modulator drugs: the nose knows;European Respiratory Journal;2023-10
5. Tissue-Specific Regulation of CFTR Gene Expression;International Journal of Molecular Sciences;2023-06-26
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