Personalized medicine with drugs targeting the underlying protein defect in cystic fibrosis: is monitoring of treatment response necessary?
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DOI: 10.1177/20406223221108627
Abstract: Cystic fibrosis (CF) is caused by two mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. In the last years, drugs targeting the underlying protein defect like lumacaftor/ivacaftor (LUM/IVA) or tezacaftor/ivacaftor (TEZ/IVA) and more… read more here.
Keywords: cftr function; cftr; iva; medicine ... See more keywords
A helper-dependent adenoviral vector rescues CFTR to wild-type functional levels in cystic fibrosis epithelial cells harbouring class I mutations
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DOI: 10.1183/13993003.00205-2020
Abstract: Cystic fibrosis (CF) is a genetic disorder affecting multiple organs, including the pancreas, hepatobiliary system and reproductive organs; however, lung disease is responsible for the majority of morbidity and mortality. Management of CF involves CF… read more here.
Keywords: vector; cystic fibrosis; cftr function; cftr ... See more keywords
Evaluation of secondhand smoke effects on CFTR function in vivo
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DOI: 10.1186/s12931-020-1324-3
Abstract: Background Cystic fibrosis transmembrane conductance regulator (CFTR) is an anion channel expressed on the mucosal surface of epithelial cells lining several tissues including airways. Inherited defects in the CFTR gene cause cystic fibrosis (CF), an… read more here.
Keywords: cftr function; secondhand smoke; exposure; physiology ... See more keywords
Anti-Infectives Restore ORKAMBI® Rescue of F508del-CFTR Function in Human Bronchial Epithelial Cells Infected with Clinical Strains of P. aeruginosa
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DOI: 10.3390/biom10020334
Abstract: Chronic infection and inflammation are the primary causes of declining lung function in Cystic Fibrosis (CF) patients. ORKAMBI® (Lumacaftor-Ivacaftor) is an approved combination therapy for Cystic Fibrosis (CF) patients bearing the most common mutation, F508del,… read more here.
Keywords: cftr function; f508del cftr; rescue; human bronchial ... See more keywords
Assays of CFTR Function In Vitro, Ex Vivo and In Vivo
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DOI: 10.3390/ijms23031437
Abstract: Cystic fibrosis, a multi-organ genetic disease, is characterized by abnormal function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, a chloride channel at the apical membrane of several epithelia. In recent years, therapeutic strategies… read more here.
Keywords: function vitro; vivo; function; vivo vivo ... See more keywords