Restoration of R117H CFTR folding and function in human airway cells through combination treatment with VX-809 and VX-770

American Journal of Physiology - Lung Cellular and Molecular Physiology

Volumn 311, Issue 3, 2016, Pages L550-L559

  Gentzsch, Martina ^a   Ren, Hong Y ^a   Houck, Scott A ^a   Quinney, Nancy L ^a   Cholon, Deborah M ^a   Sopha, Pattarawut ^a   Chaudhry, Imron G ^a   Das, Jhuma ^a   Dokholyan, Nikolay V ^a   Randell, Scott H ^a   Cyr, Douglas M ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

Cystic fibrosis; Heterozygote; Orkambi; R117H; Rare mutation; VX 770; VX 809; W1282X

Indexed keywords

CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; IVACAFTOR; LUMACAFTOR; 1,3 BENZODIOXOLE DERIVATIVE; AMINOPHENOL DERIVATIVE; AMINOPYRIDINE DERIVATIVE; CFTR PROTEIN, HUMAN; QUINOLONE DERIVATIVE;

AIRWAY CELL; ARTICLE; CONTROLLED STUDY; CYSTIC FIBROSIS; EMBRYO; HEK293 CELL LINE; HUMAN; HUMAN CELL; HUMAN TISSUE; LUNG FIBROSIS; MOLECULAR MODEL; MUTATION; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN FOLDING; PROTEIN MISFOLDING; CELL LINE; DRUG EFFECTS; GENE DELETION; GENETICS; METABOLISM; MISSENSE MUTATION; PRECLINICAL STUDY;

AMINOPHENOLS; AMINOPYRIDINES; BENZODIOXOLES; CELL LINE; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DRUG EVALUATION, PRECLINICAL; HUMANS; MUTATION, MISSENSE; PROTEIN FOLDING; QUINOLONES; SEQUENCE DELETION;

EID: 84986564652     PISSN: 10400605     EISSN: 15221504     Source Type: Journal    
DOI: 10.1152/ajplung.00186.2016     Document Type: Article

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