A contradictory treatment for lysosomal storage disorders: inhibitors enhance mutant enzyme activity
- 1 July 2003
- journal article
- review article
- Published by Elsevier in Trends in Pharmacological Sciences
- Vol. 24 (7), 355-360
- https://doi.org/10.1016/s0165-6147(03)00158-5
Abstract
No abstract availableKeywords
This publication has 58 references indexed in Scilit:
- Structure-Activity Relations of Successful Pharmacologic Chaperones for Rescue of Naturally Occurring and Manufactured Mutants of the Gonadotropin-Releasing Hormone ReceptorJournal of Pharmacology and Experimental Therapeutics, 2003
- Restoration of the tumor suppressor function to mutant p53 by a low-molecular-weight compoundNature Medicine, 2002
- Recovering Antibody Secretion Using a Hapten Ligand as a Chemical ChaperoneJournal of Biological Chemistry, 2001
- Unfolding retinal dystrophies: a role for molecular chaperones?Trends in Molecular Medicine, 2001
- Galactonojirimycin derivatives restore mutant human β-galactosidase activities expressed in fibroblasts from enzyme-deficient knockout mouseBrain & Development, 2001
- Pharmacological Rescue of Mutant p53 Conformation and FunctionScience, 1999
- Setting the Standards: Quality Control in the Secretory PathwayScience, 1999
- Aggregation of the Inactive Form of Human α-Galactosidase in the Endoplasmic ReticulumBiochemical and Biophysical Research Communications, 1996
- Characterization of a Mutant α-Galactosidase Gene Product for the Late-Onset Cardiac Form of Fabry DiseaseBiochemical and Biophysical Research Communications, 1993
- Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's DiseaseNew England Journal of Medicine, 1991