Discovery, structure–activity relationship, and biological evaluation of noninhibitory small molecule chaperones of glucocerebrosidase

S Patnaik, W Zheng, JH Choi, O Motabar…

Index: Patnaik, Samarjit; Zheng, Wei; Choi, Jae H.; Motabar, Omid; Southall, Noel; Westbroek, Wendy; Lea, Wendy A.; Velayati, Arash; Goldin, Ehud; Sidransky, Ellen; Leister, William; Marugan, Juan J. Journal of Medicinal Chemistry, 2012 , vol. 55, # 12 p. 5734 - 5748

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Citation Number: 56

Abstract

A major challenge in the field of Gaucher disease has been the development of new therapeutic strategies including molecular chaperones. All previously described chaperones of glucocerebrosidase are enzyme inhibitors, which complicates their clinical development because their chaperone activity must be balanced against the functional inhibition of the enzyme. Using a novel high throughput screening methodology, we identified a chemical ...