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Testing gene therapy vectors in human primary nasal epithelial cultures.
Cao H, Ouyang H, Ip W, Du K, Duan W, Avolio J, Wu J, Duan C, Yeger H, Bear CE, Gonska T, Hu J, Moraes TJ. Cao H, et al. Among authors: moraes tj. Mol Ther Methods Clin Dev. 2015 Sep 9;2:15034. doi: 10.1038/mtm.2015.34. eCollection 2015. Mol Ther Methods Clin Dev. 2015. PMID: 26730394 Free PMC article.
Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia.
Ahmadi S, Bozoky Z, Di Paola M, Xia S, Li C, Wong AP, Wellhauser L, Molinski SV, Ip W, Ouyang H, Avolio J, Forman-Kay JD, Ratjen F, Hirota JA, Rommens J, Rossant J, Gonska T, Moraes TJ, Bear CE. Ahmadi S, et al. Among authors: moraes tj. NPJ Genom Med. 2017 Apr 14;2:12. doi: 10.1038/s41525-017-0015-6. NPJ Genom Med. 2017. PMID: 28649446 Free PMC article.
Orkambi® and amplifier co-therapy improves function from a rare CFTR mutation in gene-edited cells and patient tissue.
Molinski SV, Ahmadi S, Ip W, Ouyang H, Villella A, Miller JP, Lee PS, Kulleperuma K, Du K, Di Paola M, Eckford PD, Laselva O, Huan LJ, Wellhauser L, Li E, Ray PN, Pomès R, Moraes TJ, Gonska T, Ratjen F, Bear CE. Molinski SV, et al. Among authors: moraes tj. EMBO Mol Med. 2017 Sep;9(9):1224-1243. doi: 10.15252/emmm.201607137. EMBO Mol Med. 2017. PMID: 28667089 Free PMC article.
The CF Canada-Sick Kids Program in individual CF therapy: A resource for the advancement of personalized medicine in CF.
Eckford PDW, McCormack J, Munsie L, He G, Stanojevic S, Pereira SL, Ho K, Avolio J, Bartlett C, Yang JY, Wong AP, Wellhauser L, Huan LJ, Jiang JX, Ouyang H, Du K, Klingel M, Kyriakopoulou L, Gonska T, Moraes TJ, Strug LJ, Rossant J, Ratjen F, Bear CE. Eckford PDW, et al. Among authors: moraes tj. J Cyst Fibros. 2019 Jan;18(1):35-43. doi: 10.1016/j.jcf.2018.03.013. Epub 2018 Oct 10. J Cyst Fibros. 2019. PMID: 29685812 Free article. Review.
ORKAMBI-Mediated Rescue of Mucociliary Clearance in Cystic Fibrosis Primary Respiratory Cultures Is Enhanced by Arginine Uptake, Arginase Inhibition, and Promotion of Nitric Oxide Signaling to the Cystic Fibrosis Transmembrane Conductance Regulator Channel.
Wu YS, Jiang J, Ahmadi S, Lew A, Laselva O, Xia S, Bartlett C, Ip W, Wellhauser L, Ouyang H, Gonska T, Moraes TJ, Bear CE. Wu YS, et al. Among authors: moraes tj. Mol Pharmacol. 2019 Oct;96(4):515-525. doi: 10.1124/mol.119.117143. Epub 2019 Aug 19. Mol Pharmacol. 2019. PMID: 31427400
Functional rescue of c.3846G>A (W1282X) in patient-derived nasal cultures achieved by inhibition of nonsense mediated decay and protein modulators with complementary mechanisms of action.
Laselva O, Eckford PD, Bartlett C, Ouyang H, Gunawardena TN, Gonska T, Moraes TJ, Bear CE. Laselva O, et al. Among authors: moraes tj. J Cyst Fibros. 2020 Sep;19(5):717-727. doi: 10.1016/j.jcf.2019.12.001. Epub 2019 Dec 9. J Cyst Fibros. 2020. PMID: 31831337 Free article.
203 results