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A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis.
Takai T, Higaki K, Aguilar-Moncayo M, Mena-Barragán T, Hirano Y, Yura K, Yu L, Ninomiya H, García-Moreno MI, Sakakibara Y, Ohno K, Nanba E, Ortiz Mellet C, García Fernández JM, Suzuki Y. Takai T, et al. Among authors: sakakibara y. Mol Ther. 2013 Mar;21(3):526-32. doi: 10.1038/mt.2012.263. Epub 2013 Jan 22. Mol Ther. 2013. PMID: 23337983 Free PMC article.
Chemical chaperone therapy: chaperone effect on mutant enzyme and cellular pathophysiology in β-galactosidase deficiency.
Higaki K, Li L, Bahrudin U, Okuzawa S, Takamuram A, Yamamoto K, Adachi K, Paraguison RC, Takai T, Ikehata H, Tominaga L, Hisatome I, Iida M, Ogawa S, Matsuda J, Ninomiya H, Sakakibara Y, Ohno K, Suzuki Y, Nanba E. Higaki K, et al. Among authors: sakakibara y. Hum Mutat. 2011 Jul;32(7):843-52. doi: 10.1002/humu.21516. Hum Mutat. 2011. PMID: 21520340
Tuning glycosidase inhibition through aglycone interactions: pharmacological chaperones for Fabry disease and GM1 gangliosidosis.
Aguilar-Moncayo M, Takai T, Higaki K, Mena-Barragán T, Hirano Y, Yura K, Li L, Yu Y, Ninomiya H, García-Moreno MI, Ishii S, Sakakibara Y, Ohno K, Nanba E, Ortiz Mellet C, García Fernández JM, Suzuki Y. Aguilar-Moncayo M, et al. Among authors: sakakibara y. Chem Commun (Camb). 2012 Jul 4;48(52):6514-6. doi: 10.1039/c2cc32065g. Epub 2012 May 23. Chem Commun (Camb). 2012. PMID: 22618082
926 results