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Pharmacological Chaperones for the Treatment of α-Mannosidosis.

J. Med. Chem.. 2019; 
Rísquez-CuadroRocío,MatsumotoReimi,Ortega-CaballeroFernando,NanbaEiji,HigakiKatsumi,García FernándezJosé Manuel,Ortiz MelletCa
Products/Services Used Details Operation
Custom Vector Construction Human mutant MAN2B1 cDNA expression vectors were obtained from GenScript Japan (Tokyo, Japan). Get A Quote

摘要

α-Mannosidosis (AM) results from deficient lysosomal α-mannosidase (LAMAN) activity and subsequent substrate accumulation in the lysosome, leading to severe pathology. Many of the AM-causative mutations compromise enzyme folding and could be rescued with purpose-designed pharmacological chaperones (PCs). We found that PCs combining a LAMAN glycone-binding motif based on the 5 N,6 O-oxomethylidenemannojirimycin (OMJ) glycomimetic core and different aglycones, in either mono- or multivalent displays, elicit binding modes involving glycone and nonglycone enzyme regions that reinforce the protein folding and stabilization potential. Multivalent derivatives exhibited potent enzyme inhibition that generally... More

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