Targeting a genetic defect: Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis

European Respiratory Review

Volumn 22, Issue 127, 2013, Pages 58-65

  Derichs, Nico ^a  

^a CHARITÉ UNIVERSITÄTSMEDIZIN BERLIN   (Germany)

Author keywords

CFTR modulators; Cystic fibrosis; G551D mutation; Ivacaftor; Personalised medicine; VX 770

Indexed keywords

AMINOGLYCOSIDE ANTIBIOTIC AGENT; ATALUREN; BIOLOGICAL MARKER; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; GENTAMICIN; IVACAFTOR; LUMACAFTOR; NAKED DNA; SODIUM CHLORIDE; VIRUS VECTOR;

AZOOSPERMIA; CHANNEL GATING; CHLORIDE TRANSPORT; CHROMOSOME 7; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR GENE; DEHYDRATION; FAILURE TO THRIVE; FORCED EXPIRATORY VOLUME; FRAMESHIFT MUTATION; GENE MUTATION; GENE THERAPY; HETEROZYGOTE; HIGH THROUGHPUT SCREENING; HUMAN; MUCOCILIARY TRANSPORT; NEPHROTOXICITY; NONHUMAN; NONSENSE MUTATION; PANCREAS INSUFFICIENCY; PERSONALIZED MEDICINE; PROTEIN STRUCTURE; PROTEIN SYNTHESIS; REVIEW; SPLICING DEFECT; STEATORRHEA; SWEAT TEST; VAS DEFERENS;

AMINOPHENOLS; ANIMALS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DRUG DISCOVERY; GENETIC PREDISPOSITION TO DISEASE; GENETIC THERAPY; HIGH-THROUGHPUT SCREENING ASSAYS; HUMANS; INDIVIDUALIZED MEDICINE; MOLECULAR TARGETED THERAPY; MUTATION; PHENOTYPE; PROGNOSIS; PROTEIN CONFORMATION; QUINOLONES; RESPIRATORY SYSTEM AGENTS; STRUCTURE-ACTIVITY RELATIONSHIP;

EID: 84874726028     PISSN: 09059180     EISSN: 16000617     Source Type: Journal    
DOI: 10.1183/09059180.00008412     Document Type: Review

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