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
2006
Assessment of cystic fibrosis transmembrane conductance regulator (CFTR) activity in CFTR-null mice after bone marrow transplantation
Bruscia EM, Price JE, Cheng EC, Weiner S, Caputo C, Ferreira EC, Egan ME, Krause DS. Assessment of cystic fibrosis transmembrane conductance regulator (CFTR) activity in CFTR-null mice after bone marrow transplantation. Proceedings Of The National Academy Of Sciences Of The United States Of America 2006, 103: 2965-2970. PMID: 16481627, PMCID: PMC1413802, DOI: 10.1073/pnas.0510758103.Peer-Reviewed Original ResearchConceptsCftr-/- miceEpithelial cellsNasal epitheliumBM-derived cellsBone marrow transplantationWild-type BMAirway epithelial cellsCystic fibrosis transmembrane conductance regulator (CFTR) activityCystic fibrosis miceRare epithelial cellsCftr-null miceMarrow transplantationBM transplantationFibrosis miceRespiratory tractCFTR activityGI tractBone marrowGastrointestinalChloride secretionCFTR-dependent chloride secretionIndividual miceTransplantationDifferent dosesMice