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