- 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 ...
Expand abstract - Publication status:
- Published
- Publisher copy:
- 10.1021/jm300342q
-
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- 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
- Language:
- English
- Keywords:
- Copyright date:
- 2012
Journal article
Potent aminocyclitol glucocerebrosidase inhibitors are subnanomolar pharmacological chaperones for treating gaucher disease.
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