|Title||Synthesis of C-5a-chain extended derivatives of 4-epi-isofagomine: Powerful beta-galactosidase inhibitors and low concentration activators of G(M1)-gangliosidosis-related human lysosomal beta-galactosidase|
|Publication Type||Journal Article|
|Year of Publication||2016|
|Authors||Thonhofer, M, Weber, P, Santana, AGonzalez, Fischer, R, Pabst, BM, Paschke, E, Schalli, M, Stuetz, AE, Tschernutter, M, Windischhofer, W, Withers, SG|
|Journal||BIOORGANIC & MEDICINAL CHEMISTRY LETTERS|
|Date Published||MAR 1|
From an easily available partially protected formal derivative of 1-deoxymannojirimycin, by hydroxymethyl chain-branching and further elaboration, lipophilic analogs of the powerful beta-D-galactosidase inhibitor 4-epi-isofagomine have become available. New compounds exhibit improved inhibitory activities comparable to benchmark compound NOEV (N-octyl-epi-valienamine) and may serve as leads towards improved and more selective pharmacological chaperones for G(M1)-gangliosidosis. (C) 2016 Elsevier Ltd. All rights reserved.