New horizons for cystic fibrosis treatment

Pharmacology and Therapeutics

Volumn 170, Issue , 2017, Pages 205-211

  Fajac, Isabelle ^a,b   De Boeck, Kris ^c  

^a UNIVERSITÉ PARIS DESCARTES   (France)

^b Institut Cochin   (France)

^c UNIVERSITY HOSPITALS LEUVEN   (Belgium)

Author keywords

CFTR; CFTR modulator; Corrector; Cystic fibrosis; Gene therapy; Potentiator; Read through agent

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; ION CHANNEL; IVACAFTOR; MESSENGER RNA; OLIGONUCLEOTIDE;

CELL MEMBRANE; CLINICAL FEATURE; CYSTIC FIBROSIS; DRUG EFFICACY; DRUG POTENCY; DRUG SAFETY; EX VIVO STUDY; GENE EDITING; GENE EXPRESSION; GENE MUTATION; GENE THERAPY; HUMAN; MULTICENTER STUDY (TOPIC); PALLIATIVE THERAPY; PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROTEIN SYNTHESIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; RNA REPAIR; STOP CODON; DRUG DESIGN; GENETICS; METABOLISM; MUTATION; PATHOPHYSIOLOGY; PROCEDURES;

CELL MEMBRANE; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DRUG DESIGN; GENE EDITING; GENETIC THERAPY; HUMANS; MUTATION; OLIGONUCLEOTIDES, ANTISENSE; RNA, MESSENGER;

EID: 85007565989     PISSN: 01637258     EISSN: 1879016X     Source Type: Journal    
DOI: 10.1016/j.pharmthera.2016.11.009     Document Type: Review

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