Synthesis of lipophilic 1-deoxygalactonojirimycin derivatives as D-galactosidase inhibitors

Scholarly Works

• Ashmus, R. A.; Wang, Y.; González-Cuesta, M.; King, D. T.; Tiet, B.; Gilormini, P.-A.; Fernández, J. M. G.; Mellet, C. O.; Britton, R.; Vocadlo, D. J. Rational design of cell active C2-modified DGJ analogues for the inhibition of human α-galactosidase A (GALA). Organic & biomolecular chemistry 2021, 19, 8057–8062. doi:10.1039/d1ob01526e
• Mohamed, F. E.; M, A. S.; Ghattas, M. A.; Al-Gazali, L.; Al-Dirbashi, O. Y.; Al-Jasmi, F.; Ali, B. R. The pharmacological chaperone N-n-butyl-deoxygalactonojirimycin enhances β-galactosidase processing and activity in fibroblasts of a patient with infantile GM1-gangliosidosis. Human genetics 2020, 139, 657–673. doi:10.1007/s00439-020-02153-3
• Flick, A. C.; Ding, H. X.; Leverett, C. A.; Fink, S. J.; O’Donnell, C. J. Synthetic Approaches to New Drugs Approved During 2016. Journal of medicinal chemistry 2018, 61, 7004–7031. doi:10.1021/acs.jmedchem.8b00260
• Meanwell, M.; Sutherland, M.; Britton, R. Application of sequential proline-catalyzed α-chlorination and aldol reactions in the total synthesis of 1-deoxygalactonojirimycin. Canadian Journal of Chemistry 2018, 96, 144–147. doi:10.1139/cjc-2017-0318
• Convertino, M.; Das, J.; Dokholyan, N. V. Pharmacological Chaperones: Design and Development of New Therapeutic Strategies for the Treatment of Conformational Diseases. ACS chemical biology 2016, 11, 1471–1489. doi:10.1021/acschembio.6b00195
• Sánchez-Fernández, E. M.; Fernández, J. M. G.; Mellet, C. O. Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases. Chemical communications (Cambridge, England) 2016, 52, 5497–5515. doi:10.1039/c6cc01564f
• Alcaide, A.; Trapero, A.; Pérez, Y.; Llebaria, A. Galacto configured N-aminoaziridines: a new type of irreversible inhibitor of β-galactosidases. Organic & biomolecular chemistry 2015, 13, 5690–5697. doi:10.1039/c5ob00532a
• Yu, Y.; Mena-Barragán, T.; Higaki, K.; Johnson, J. L.; Drury, J. E.; Lieberman, R. L.; Nakasone, N.; Ninomiya, H.; Tsukimura, T.; Sakuraba, H.; Suzuki, Y.; Nanba, E.; Mellet, C. O.; Fernández, J. M. G.; Ohno, K. Molecular basis of 1-deoxygalactonojirimycin arylthiourea binding to human α-galactosidase a: pharmacological chaperoning efficacy on Fabry disease mutants. ACS chemical biology 2014, 9, 1460–1469. doi:10.1021/cb500143h
• Biela-Banaś, A.; Gallienne, E.; Front, S.; Martin, O. R. Stereoselective synthesis of 1-C-alkyl iminogalactitol derivatives, potential chaperones for galactosidase-linked LSDs: a real challenge. Tetrahedron Letters 2014, 55, 838–841. doi:10.1016/j.tetlet.2013.12.024
• Galvis, J.; González, J.; Torrente, D.; Velasco, H.; Barreto, G. E. In silico Analysis of Iduronate 2 Sulfatase Mutations in Colombian Patients with Hunter Syndrome (MPSII). Advances in Intelligent Systems and Computing; Springer International Publishing, 2014; pp 205–212. doi:10.1007/978-3-319-01568-2_30
• Boyd, R.; Lee, G.; Rybczynski, P. J.; Benjamin, E. R.; Khanna, R.; Wustman, B.; Valenzano, K. J. Pharmacological Chaperones as Therapeutics for Lysosomal Storage Diseases. Journal of medicinal chemistry 2013, 56, 2705–2725. doi:10.1021/jm301557k
• Du, Y.; Ye, H.; Gill, T.; Wang, L.; Guo, F.; Cuconati, A.; Guo, J.-T.; Block, T. M.; Chang, J.; Xu, X. N-Alkyldeoxynojirimycin derivatives with novel terminal tertiary amide substitution for treatment of bovine viral diarrhea virus (BVDV), Dengue, and Tacaribe virus infections. Bioorganic & medicinal chemistry letters 2013, 23, 2172–2176. doi:10.1016/j.bmcl.2013.01.108
• Aymami, J.; Barril, X.; Rodríguez-Pascau, L.; Martinell, M. Pharmacological chaperones for enzyme enhancement therapy in genetic diseases. Pharmaceutical patent analyst 2013, 2, 109–124. doi:10.4155/ppa.12.74
• Lee, J. C.; Francis, S.; Dutta, D.; Gupta, V.; Yang, Y.; Zhu, J.-Y.; Tash, J. S.; Schönbrunn, E.; Georg, G. I. Synthesis and evaluation of eight- and four-membered iminosugar analogues as inhibitors of testicular ceramide-specific glucosyltransferase, testicular β-glucosidase 2, and other glycosidases. The Journal of organic chemistry 2012, 77, 3082–3098. doi:10.1021/jo202054g
• Lindhorst, T. K. Synthesis in the glycosciences II. Beilstein journal of organic chemistry 2012, 8, 411–412. doi:10.3762/bjoc.8.45
• Valenzano, K. J.; Khanna, R.; Powe, A. C.; Boyd, R.; Lee, G.; Flanagan, J. J.; Benjamin, E. R. Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders. Assay and drug development technologies 2011, 9, 213–235. doi:10.1089/adt.2011.0370
• Fröhlich, R. F.; Furneaux, R. H.; Mahuran, D. J.; Saf, R.; Stütz, A. E.; Tropak, M. B.; Wicki, J.; Withers, S. G.; Wrodnigg, T. M. 1-Deoxy-d-galactonojirimycins with dansyl capped N-substituents as β-galactosidase inhibitors and potential probes for GM1 gangliosidosis affected cell lines. Carbohydrate research 2011, 346, 1592–1598. doi:10.1016/j.carres.2011.05.010
• Stütz, A. E.; Wrodnigg, T. M. Imino sugars and glycosyl hydrolases: historical context, current aspects, emerging trends. Advances in carbohydrate chemistry and biochemistry 2011, 66, 187–298. doi:10.1016/b978-0-12-385518-3.00004-3

Explore citations to this article at