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Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.

par Sermet-Gaudelus, Isabelle;Boeck, Kris De;Casimir, Georges ;Vermeulen, François ;Leal, Teresinha;Mogenet, Agnès;Roussel, Delphine;Fritsch, Janine;Hanssens, Laurence ;Hirawat, Samit;Miller, Nilsen L;Constantine, Scott;Reha, Allen;Ajayi, Temitayo;Elfring, Gary L;Miller, Langdon L
Référence American journal of respiratory and critical care medicine, 182, 10, page (1262-1272)
Publication Publié, 2010-11

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Titre:	Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
Auteur:	Sermet-Gaudelus, Isabelle; Boeck, Kris De; Casimir, Georges; Vermeulen, François; Leal, Teresinha; Mogenet, Agnès; Roussel, Delphine; Fritsch, Janine; Hanssens, Laurence; Hirawat, Samit; Miller, Nilsen L; Constantine, Scott; Reha, Allen; Ajayi, Temitayo; Elfring, Gary L; Miller, Langdon L
Informations sur la publication:	American journal of respiratory and critical care medicine, 182, 10, page (1262-1272)
Statut de publication:	Publié, 2010-11
Sujet CREF:	Sciences bio-médicales et agricoles
Mots-clés:	Codon, nonsense
	Cystic fibrosis transmembrane conductance regulator
	Drugs, investigational
	Membrane potentials
	Pediatrics
MeSH keywords:	Adolescent
	Child
	Codon, Nonsense -- drug effects -- genetics -- physiology
	Cross-Over Studies
	Cystic Fibrosis -- drug therapy -- genetics -- metabolism -- physiopathology
	Cystic Fibrosis Transmembrane Conductance Regulator -- biosynthesis -- physiology
	Dose-Response Relationship, Drug
	Female
	Gene Expression Regulation -- drug effects -- genetics -- physiology
	Humans
	Male
	Nasal Mucosa -- metabolism -- physiopathology
	Oxadiazoles -- administration & dosage -- pharmacology -- therapeutic use
Note générale:	Journal Article
	Randomized Controlled Trial
	Research Support, Non-U.S. Gov't
	SCOPUS: ar.j
Langue:	Anglais
Identificateurs:	urn:issn:1073-449X
	info:doi/10.1164/rccm.201001-0137OC
	info:pii/201001-0137OC
	info:scp/78349290383
	info:pmid/20622033