کد مقاله | کد نشریه | سال انتشار | مقاله انگلیسی | نسخه تمام متن |
---|---|---|---|---|
1396515 | 1501189 | 2010 | 18 صفحه PDF | دانلود رایگان |
![عکس صفحه اول مقاله: Evaluation of 2-thioxo-2,3,5,6,7,8-hexahydropyrimido[4,5-d]pyrimidin-4(1H)-one analogues as GAA activators Evaluation of 2-thioxo-2,3,5,6,7,8-hexahydropyrimido[4,5-d]pyrimidin-4(1H)-one analogues as GAA activators](/preview/png/1396515.png)
Pompe disease is a lysosomal storage disease (LSD) caused by a deficiency in the lysosomal enzyme acid alpha-glucosidase. In several LSDs, enzyme inhibitors have been used as small molecule chaperones to facilitate and increase the translocation of mutant protein from the endoplasmic reticulum to the lysosome. Enzyme activators with chaperone activity would be even more desirable as they would not inhibit the enzyme after translocation and might potentiate the activity of the enzyme that is successfully translocated. Herein we report our initial findings of a new series of acid alpha-glucosidase activators.
Compound 1 and analogues are able to activate acid alpha-glucosidase's hydrolysis of resorufin α-d-glucopyranoside and 4-methylumbelliferyl-α-d-glucopyranoside in a selective and dose-dependent manner.Figure optionsDownload as PowerPoint slide
Journal: European Journal of Medicinal Chemistry - Volume 45, Issue 5, May 2010, Pages 1880–1897