1997
In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR.
Rubenstein R, Egan M, Zeitlin P. In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR. Journal Of Clinical Investigation 1997, 100: 2457-2465. PMID: 9366560, PMCID: PMC508446, DOI: 10.1172/jci119788.Peer-Reviewed Original ResearchConceptsDelta F508-CFTRIB3-1 cellsCystic fibrosis epithelial cellsFunctional chloride channelChloride channelsHigh molecular mass formsModification of oligosaccharidesCF epithelial cellsEpithelial cellsMolecular mass formsTranscriptional regulatorsPlasma membraneGolgi apparatusEndoplasmic reticulumCystic fibrosis transmembrane conductance regulator (CFTR) mutationsCF phenotypeIB3-1Cell surfaceDelta F508 mutationWhole cellsMass formNasal polyp epitheliumRegulator mutationsCFTRF508 mutation
1996
Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line.
Kearns W, Afione S, Fulmer S, Pang M, Erikson D, Egan M, Landrum M, Flotte T, Cutting G. Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line. Gene Therapy 1996, 3: 748-55. PMID: 8875221.Peer-Reviewed Original ResearchConceptsIB3-1 cellsWild-type AAVEpithelial cell lineMetaphase spreadsSite-specific fashionCFTR cDNACell linesLow molecular weight DNA fractionCF bronchial epithelial cell lineHuman chromosome 19Immortalized epithelial cell lineWeight DNA fractionMetaphase chromosome preparationsMetaphase chromosome spreadsBronchial epithelial cell lineDifferent chromosomesAAV-CFTR vectorCDNA integrationChromosome spreadsChromosome 19Genomic DNASite-specific mannerDNA fractionChromosome preparationsCFTR expression