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Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling.
Fan M, Sidhu R, Fujiwara H, Tortelli B, Zhang J, Davidson C, Walkley SU, Bagel JH, Vite C, Yanjanin NM, Porter FD, Schaffer JE, Ory DS. Fan M, et al. Among authors: bagel jh. J Lipid Res. 2013 Oct;54(10):2800-14. doi: 10.1194/jlr.M040618. Epub 2013 Jul 23. J Lipid Res. 2013. PMID: 23881911 Free PMC article.
Cholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann-Pick C1 (NPC1).
Tortelli B, Fujiwara H, Bagel JH, Zhang J, Sidhu R, Jiang X, Yanjanin NM, Shankar RK, Carillo-Carasco N, Heiss J, Ottinger E, Porter FD, Schaffer JE, Vite CH, Ory DS. Tortelli B, et al. Among authors: bagel jh. Hum Mol Genet. 2014 Nov 15;23(22):6022-33. doi: 10.1093/hmg/ddu331. Epub 2014 Jun 25. Hum Mol Genet. 2014. PMID: 24964810 Free PMC article. Clinical Trial.
Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease.
Vite CH, Bagel JH, Swain GP, Prociuk M, Sikora TU, Stein VM, O'Donnell P, Ruane T, Ward S, Crooks A, Li S, Mauldin E, Stellar S, De Meulder M, Kao ML, Ory DS, Davidson C, Vanier MT, Walkley SU. Vite CH, et al. Among authors: bagel jh. Sci Transl Med. 2015 Feb 25;7(276):276ra26. doi: 10.1126/scitranslmed.3010101. Sci Transl Med. 2015. PMID: 25717099 Free PMC article.
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.
Gurda BL, De Guilhem De Lataillade A, Bell P, Zhu Y, Yu H, Wang P, Bagel J, Vite CH, Sikora T, Hinderer C, Calcedo R, Yox AD, Steet RA, Ruane T, O'Donnell P, Gao G, Wilson JM, Casal M, Ponder KP, Haskins ME. Gurda BL, et al. Mol Ther. 2016 Feb;24(2):206-216. doi: 10.1038/mt.2015.189. Epub 2015 Oct 8. Mol Ther. 2016. PMID: 26447927 Free PMC article.
27 results