Featured Publications
In vivo correction of cystic fibrosis mediated by PNA nanoparticles
Piotrowski-Daspit AS, Barone C, Lin CY, Deng Y, Wu D, Binns TC, Xu E, Ricciardi AS, Putman R, Garrison A, Nguyen R, Gupta A, Fan R, Glazer PM, Saltzman WM, Egan ME. In vivo correction of cystic fibrosis mediated by PNA nanoparticles. Science Advances 2022, 8: eabo0522. PMID: 36197984, PMCID: PMC9534507, DOI: 10.1126/sciadv.abo0522.Peer-Reviewed Original ResearchCystic fibrosisF508del miceIntravenous deliveryPrimary nasal epithelial cellsMultiple organ dysfunctionNasal epithelial cellsUssing chamber assaysOrgan dysfunctionF508del cystic fibrosisVivo treatmentGI tissuesCF transmembrane conductance regulator (CFTR) geneChamber assaySystemic deliveryEpithelial cellsCF-causing mutationsFibrosisCFTR functionMiceTransmembrane conductance regulator geneTarget effectsAir-liquid interfaceDeliveryPartial gainViable option
2024
219 CFTR dysfunction shapes airway immune cell compositions contributing to lung pathogenesis in children with cystic fibrosis
Kizilirmak T, Yin H, Garrison A, Browne J, Bruscia E, Egan M, Britto C. 219 CFTR dysfunction shapes airway immune cell compositions contributing to lung pathogenesis in children with cystic fibrosis. Journal Of Cystic Fibrosis 2024, 23: s119. DOI: 10.1016/s1569-1993(24)01059-2.Peer-Reviewed Original ResearchLocalization and function of humanized F508del-CFTR in mouse intestine following activation of serum glucocorticoid kinase 1 and Trikafta
Dastoor P, Muiler C, Garrison A, Egan M, Carlos Dos Reis D, Santos A, Ameen N. Localization and function of humanized F508del-CFTR in mouse intestine following activation of serum glucocorticoid kinase 1 and Trikafta. European Journal Of Pharmacology 2024, 978: 176771. PMID: 38925289, DOI: 10.1016/j.ejphar.2024.176771.Peer-Reviewed Original ResearchSerum glucocorticoid kinase 1Glucocorticoid kinase 1F508del-CFTRCystic fibrosisMouse modelF508del-CFTR miceCFTR-expressing epithelial cellsCF mouse modelsHumanized mouse modelTreatment of ratsIntestinal diseaseIntestinal segmentsKinase 1CFTR modulatorsCFTR mutationsCF patientsTrikaftaDex treatmentLung diseaseEfficacy of compoundsDelta F508CFTRCombined treatmentEpithelial cellsLoss of function
2023
Sa1217 ROLE OF TRIKAFTA AND SERUM GLUCOCORTICOID KINASE (SGK1) IN RESCUE OF DF508 CFTR IN A HUMANIZED MOUSE MODEL OF CYSTIC FIBROSIS
Dastoor P, Barone C, dos Reis D, Garrison A, Egan M, Santos A, Ameen N. Sa1217 ROLE OF TRIKAFTA AND SERUM GLUCOCORTICOID KINASE (SGK1) IN RESCUE OF DF508 CFTR IN A HUMANIZED MOUSE MODEL OF CYSTIC FIBROSIS. Gastroenterology 2023, 164: s-329. DOI: 10.1016/s0016-5085(23)01750-x.Peer-Reviewed Original Research