Strategies in early clinical development for the treatment of basic defects of cystic fibrosis

Expert Opinion on Investigational Drugs

Volumn 25, Issue 4, 2016, Pages 423-436

  Dhooghe, Barbara ^a   Haaf, Jérémy Boris ^a   Noel, Sabrina ^a   Leal, Teresinha ^a  

^a UNIVERSITÉ CATHOLIQUE DE LOUVAIN   (Belgium)

Author keywords

CFTR; clinical trials; correctors; Cystic Fibrosis; gene therapy; mRNA repair; non CFTR ion channels; pharmacotherapy; phosphodiesterase type 5 inhibitors; potentiators

Indexed keywords

ATALUREN; CHAPERONE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; IVACAFTOR; LUMACAFTOR; MESSENGER RNA; OLIGONUCLEOTIDE; PHOSPHODIESTERASE V INHIBITOR; QR 010; SILDENAFIL; TEZACAFTOR; UNCLASSIFIED DRUG; VARDENAFIL;

CFTR GENE; CLINICAL TRIAL (TOPIC); CONTROLLED CLINICAL TRIAL (TOPIC); CYSTIC FIBROSIS; GENE; GENE TRANSFER; GENETIC TRANSCRIPTION; HUMAN; MULTICENTER STUDY (TOPIC); NONHUMAN; NONSENSE MUTATION; NONVIRAL GENE THERAPY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PROTEIN HOMEOSTASIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; VIRAL GENE THERAPY; ANIMAL; GENETICS; METABOLISM; MUTATION;

ANIMALS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; HUMANS; MUTATION; TRANSCRIPTION, GENETIC;

EID: 84961828393     PISSN: 13543784     EISSN: 17447658     Source Type: Journal    
DOI: 10.1517/13543784.2016.1154041     Document Type: Review

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