Discovery of a novel noniminosugar acid α glucosidase chaperone series

DOI: 10.1021/jm3005543

Source and publish data:

Journal of Medicinal Chemistry p. 7546 - 7559,14 (2012)

Update date:2022-08-05

Topics:

Authors:

Xiao, Jingbo

Motabar, Omid

Lea, Wendy A.

Hu, Xin Hu, Xin

Zheng, Wei

Southall, Noel

Liu, Ke Liu, Ke

Simeonov, Anton

Ferrer, Marc

Marugan, Juan J.

Westbroek, Wendy

Velayati, Arash

Gustafson, Ann Marie

Goldin, Ehud

Sidransky, Ellen

Tamargo, Rafael J.

Ribes, Antonia

Matalonga, Leslie

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Article abstract of DOI:10.1021/jm3005543

Pompe disease is an autosomal recessive lysosomal storage disorder (LSD) caused by deficiency of the lysosomal enzyme acid α-glucosidase (GAA). Many disease-causing mutated GAA retain enzymatic activity but are not translocated from endoplasmic reticulum

Full text of DOI:10.1021/jm3005543

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