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Potent aminocyclitol glucocerebrosidase inhibitors are subnanomolar pharmacological chaperones for treating gaucher disease.

Abstract:

Amino-myo-inositol derivatives have been found to be potent inhibitors of glucocerebrosidase (GCase), the β-glucosidase enzyme deficient in Gaucher disease (GD). When tested using lymphoblasts derived from patients with GD homozygous for N370S or L444P mutations, the compounds enhanced GCase activity at very low concentrations. The most potent inhibitor, (1R,2S,3R,4S,5S,6R)-5-(nonylamino)-6-(nonyloxy)cyclohexane-1,2,3,4-tetraol had a K(i) of 1 nM using isolated enzyme and an IC(50) of 4.3 nM ...

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Publication status:
Published

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Publisher copy:
10.1021/jm300342q

Authors


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Institution:
University of Oxford
Department:
Oxford, MSD, Biochemistry
Role:
Author
Journal:
Journal of medicinal chemistry
Volume:
55
Issue:
9
Pages:
4479-4488
Publication date:
2012-05-05
DOI:
EISSN:
1520-4804
ISSN:
0022-2623
URN:
uuid:5302a5db-de08-419a-8c2e-4338f0be0a6c
Source identifiers:
333979
Local pid:
pubs:333979

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