Evaluation of 2-thioxo-2, 3, 5, 6, 7, 8-hexahydropyrimido [4, 5-d] pyrimidin-4 (1H)-one analogues as GAA activators

…, O Motabar, N Southall, E Goldin, E Sidransky…

文献索引：Marugan, Juan J.; Zheng, Wei; Motabar, Omid; Southall, Noel; Goldin, Ehud; Sidransky, Ellen; Aungst, Ronald A.; Liu, Ke; Sadhukhan, Subir Kumar; Austin, Christopher P. European Journal of Medicinal Chemistry, 2010 , vol. 45, # 5 p. 1880 - 1897

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被引用次数: 21

摘要

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 ...