| http://purl.uniprot.org/citations/27103391 | http://www.w3.org/1999/02/22-rdf-syntax-ns#type | http://purl.uniprot.org/core/Journal_Citation |
| http://purl.uniprot.org/citations/27103391 | http://www.w3.org/2000/01/rdf-schema#comment | "Small-molecule therapies that restore defects in cystic fibrosis transmembrane conductance regulator (CFTR) gating (potentiators) or trafficking (correctors) are being developed for cystic fibrosis (CF) in a mutation-specific fashion. Options for pharmacological correction of CFTR-p.Phe508del (F508del) are being extensively studied but correction of other trafficking mutants that may also benefit from corrector treatment remains largely unknown.We studied correction of the folding mutants CFTR-p.Phe508del, -p.Ala455Glu (A455E) and -p.Asn1303Lys (N1303K) by VX-809 and 18 other correctors (C1-C18) using a functional CFTR assay in human intestinal CF organoids.Function of both CFTR-p.Phe508del and -p.Ala455Glu was enhanced by a variety of correctors but no residual or corrector-induced activity was associated with CFTR-p.Asn1303Lys. Importantly, VX-809-induced correction was most dominant for CFTR-p.Phe508del, while correction of CFTR-p.Ala455Glu was highest by a subgroup of compounds called bithiazoles (C4, C13, C14 and C17) and C5.These data support the development of mutation-specific correctors for optimal treatment of different CFTR trafficking mutants, and identify C5 and bithiazoles as the most promising compounds for correction of CFTR-p.Ala455Glu."xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.org/dc/terms/identifier | "doi:10.1183/13993003.01192-2015"xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/author | "Vonk A.M."xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/author | "Beekman J.M."xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/author | "van der Ent C.K."xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/author | "Kruisselbrink E."xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/author | "Dekkers J.F."xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/author | "Janssens H.M."xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/author | "de Winter-de Groot K.M."xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/author | "Gogorza Gondra R.A."xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/date | "2016"xsd:gYear |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/name | "Eur Respir J"xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/pages | "451-458"xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/title | "Optimal correction of distinct CFTR folding mutants in rectal cystic fibrosis organoids."xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://purl.uniprot.org/core/volume | "48"xsd:string |
| http://purl.uniprot.org/citations/27103391 | http://www.w3.org/2004/02/skos/core#exactMatch | http://purl.uniprot.org/pubmed/27103391 |
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