Thonhofer Martin, Weber Patrick, Gonzalez Santana Andres, Tysoe Christina, Fischer Roland, Pabst Bettina M, Paschke Eduard, Schalli Michael, Stütz Arnold E, Tschernutter Marion, Windischhofer Werner, Withers Stephen G
Glycogroup, Institute of Organic Chemistry, Graz University of Technology, Stremayrgasse 9, A-8010 Graz, Austria.
Chemistry Department, University of British Columbia, 2036 Main Mall, Vancouver, BC, Canada V6T 1Z1.
Carbohydr Res. 2016 Jun 24;429:71-80. doi: 10.1016/j.carres.2016.03.020. Epub 2016 Mar 31.
From an easily available partially protected analog of 1-deoxy-L-gulo-nojirimycin, by chain-branching at C-4 and suitable modification, lipophilic analogs of the powerful β-D-galactosidase inhibitor 4-epi-isofagomine have been prepared. New compounds exhibit considerably improved inhibitory activities when compared with the unsubstituted parent compound and may serve as leads toward new pharmacological chaperones for GM1-gangliosidosis and Morquio B disease.
从一种易于获得的1-脱氧-L-古洛糖-野尻霉素部分保护类似物出发，通过在C-4位进行链支化和适当修饰，制备了强效β-D-半乳糖苷酶抑制剂4-表异法戈明的亲脂性类似物。与未取代的母体化合物相比，新化合物表现出显著提高的抑制活性，并且可作为开发用于GM1-神经节苷脂病和莫尔基奥B病的新药理学伴侣分子的先导化合物。
