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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. Among authors: sikora t. 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.
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: sikora tu. Sci Transl Med. 2015 Feb 25;7(276):276ra26. doi: 10.1126/scitranslmed.3010101. Sci Transl Med. 2015. PMID: 25717099 Free PMC article.
A Novel APOC2 Missense Mutation Causing Apolipoprotein C-II Deficiency With Severe Triglyceridemia and Pancreatitis.
Ueda M, Dunbar RL, Wolska A, Sikora TU, Escobar MDR, Seliktar N, deGoma E, DerOhannessian S, Morrell L, McIntyre AD, Burke F, Sviridov D, Amar M, Shamburek RD, Freeman L, Hegele RA, Remaley AT, Rader DJ. Ueda M, et al. Among authors: sikora tu. J Clin Endocrinol Metab. 2017 May 1;102(5):1454-1457. doi: 10.1210/jc.2016-3903. J Clin Endocrinol Metab. 2017. PMID: 28201738 Free PMC article.
47 results