2024
264 Poly(amine-co-ester) nanoparticle delivery of CFTR mRNA shows restoration of CFTR activity in cystic fibrosis airway models
Garrison A, Lee J, Browne J, Akhtar L, Peterec K, Suberi A, Eaton D, Ene M, Zhang X, Whang C, Oez H, Kizilirmak T, Bruscia E, Piotrowski-Daspit A, Saltzman W, Egan M. 264 Poly(amine-co-ester) nanoparticle delivery of CFTR mRNA shows restoration of CFTR activity in cystic fibrosis airway models. Journal Of Cystic Fibrosis 2024, 23: s140-s141. DOI: 10.1016/s1569-1993(24)01104-4.Peer-Reviewed Original Research
2002
Isolation of CF cell lines corrected at ΔF508-CFTR locus by SFHR-mediated targeting
Bruscia E, Sangiuolo F, Sinibaldi P, Goncz KK, Novelli G, Gruenert DC. Isolation of CF cell lines corrected at ΔF508-CFTR locus by SFHR-mediated targeting. Gene Therapy 2002, 9: 683-685. PMID: 12032687, DOI: 10.1038/sj.gt.3301741.Peer-Reviewed Original ResearchConceptsSmall fragment homologous replacementAllele-specific PCRPrimer extension productsCell linesCFTR mRNAHuman airway epithelial cellsAllele-specific PCR amplificationΔF508 mutationCF transmembrane conductance regulator (CFTR) proteinExon 10CF cell lineMutant cell linesPCR amplificationAirway epithelial cellsEpithelial cellsGenomic DNA fingerprintingEndogenous CFTRHomologous replacementRegulator proteinDNA fingerprint analysisGenomic DNAWT mRNADNA fragmentsΔF508 alleleSequence analysis
1994
Transmembrane Mutations Alter the Channel Characteristics of the Cystic Fibrosis Transmembrane Conductance Regulator Expressed in Xenopus Oocytes
Carroll T, Mclntosh I, Egan M, Zeitlin P, Cutting G, Guggino W. Transmembrane Mutations Alter the Channel Characteristics of the Cystic Fibrosis Transmembrane Conductance Regulator Expressed in Xenopus Oocytes. Cellular Physiology And Biochemistry 1994, 4: 10-18. DOI: 10.1159/000154705.Peer-Reviewed Original ResearchCystic fibrosis transmembrane conductance regulatorFibrosis transmembrane conductance regulatorWild-type CFTRXenopus oocytesAnti-CFTR antibodiesTransmembrane conductance regulatorCFTR Cl- channelTransmembrane regionMutant CFTRMutant formsMutation altersConductance regulatorCFTR mRNACl- currentReduced cAMPCF disease severityCl- channelsOpen channel probabilityWestern blottingCFTROocytesMutationsCl- conductanceChannel propertiesCAMP
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