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American journal of respiratory and critical care medicine, ISSN 1073-449X, 03/2018, Volume 197, Issue 5, pp. 632 - 643
Abstract Rationale: MicroRNAs (miRNAs) destabilize mRNA transcripts and inhibit protein translation. miR-145 is of particular interest in cystic fibrosis (CF)... 
Cystic fibrosis | TGF-b | MicroRNA | Lumacaftor | CFTR modulation | Hypertension | Proteins | MicroRNAs | Lung diseases | Genomics | Genes | Respiratory diseases | Stem cells | Growth factors | Binding sites
Journal Article
The New England journal of medicine, ISSN 1533-4406, 2017, Volume 377, Issue 21, pp. 2013 - 2023
Journal Article
The New England journal of medicine, ISSN 1533-4406, 2017, Volume 377, Issue 21, pp. 2024 - 2035
Journal Article
The New England journal of medicine, ISSN 1533-4406, 2017, Volume 377, Issue 21, pp. 2085 - 2088
Cystic fibrosis is a disease of abnormal ion transport through epithelium that results in progressive lung disease as well as the involvement of other organs... 
MEDICINE, GENERAL & INTERNAL | LUMACAFTOR | IVACAFTOR | POTENTIATOR | PHE508DEL CFTR | MUTATION | Proteins | Clinical trials | Cystic fibrosis | Protein expression | Mutation | Drug therapy
Journal Article
Scientific reports, ISSN 2045-2322, 2019, Volume 9, Issue 1, pp. 7234 - 9
Several placebo-controlled trials have been recently published evaluating novel therapies targeting the defective CFTR protein. This systematic review examines... 
IN-VITRO | THERAPY | TEZACAFTOR-IVACAFTOR | MULTIDISCIPLINARY SCIENCES | PHE508DEL CFTR | MUTATION | DOUBLE-BLIND | F508DEL-CFTR | PHASE-3 | LUMACAFTOR/IVACAFTOR COMBINATION | POTENTIATOR | Clinical trials | Cystic fibrosis | Systematic review | Mutation | Cystic fibrosis transmembrane conductance regulator | Nonsense mutation
Journal Article
American journal of respiratory and critical care medicine, ISSN 1073-449X, 12/2016, Volume 194, Issue 11, pp. 1375 - 1382
Journal Article
Journal of cystic fibrosis, ISSN 1569-1993, 2020, Volume 19, Issue 1, pp. 99 - 107
Eluforsen is an antisense oligonucleotide designed to bind to the mRNA region around the F508-encoding deletion and restore the cystic fibrosis transmembrane... 
Clinical trial | Delta F508 | CFQ-R RSS | Antisense oligonucleotide | Pulmonary medicine | LUMACAFTOR | RESPIRATORY SYSTEM | TEZACAFTOR-IVACAFTOR | PHE508DEL CFTR | PHASE-2 | LUNG CLEARANCE INDEX | CFTR POTENTIATOR
Journal Article
American journal of respiratory and critical care medicine, ISSN 1535-4970, 2018, Volume 197, Issue 2, pp. 214 - 224
Rationale: Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane... 
Sweat chloride | Cystic fibrosis transmembrane conductance regulator corrector | Forced expiratory volume | CFTR modulator | EFFICACY | SAFETY | LUMACAFTOR-IVACAFTOR | IDENTIFICATION | INVESTIGATIONAL CFTR CORRECTOR | TRANSMEMBRANE CONDUCTANCE REGULATOR | G551D MUTATION | F508DEL-CFTR MUTATION | GENE | RESPIRATORY SYSTEM | cystic fibrosis transmembrane conductance regulator corrector | PHE508DEL CFTR | forced expiratory volume | sweat chloride | CRITICAL CARE MEDICINE | Prognosis | Humans | Male | Benzodioxoles - administration & dosage | Aminophenols - adverse effects | Indoles - administration & dosage | Dose-Response Relationship, Drug | Young Adult | Quinolones - adverse effects | Adult | Benzodioxoles - adverse effects | Female | Drug Therapy, Combination | Molecular Targeted Therapy - methods | Severity of Illness Index | Double-Blind Method | Drug Administration Schedule | Risk Assessment | Administration, Oral | Treatment Outcome | Maximum Tolerated Dose | Quinolones - administration & dosage | Indoles - adverse effects | Cystic Fibrosis - genetics | Adolescent | Cystic Fibrosis - diagnosis | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Respiratory Function Tests | Aminophenols - administration & dosage | Cystic Fibrosis - drug therapy | Proteins | Pediatrics | Chloride | Cysts | Cystic fibrosis | Mutation | Kinases | Patients | Drug dosages | Original
Journal Article
Journal of cystic fibrosis, ISSN 1569-1993, 2019, Volume 18, Issue 1, pp. 22 - 34
Journal Article