Literature DB >> 12871668

A contradictory treatment for lysosomal storage disorders: inhibitors enhance mutant enzyme activity.

Jian-Qiang Fan1.   

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Year:  2003        PMID: 12871668     DOI: 10.1016/S0165-6147(03)00158-5

Source DB:  PubMed          Journal:  Trends Pharmacol Sci        ISSN: 0165-6147            Impact factor:   14.819


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  56 in total

1.  Molecular basis of reduced glucosylceramidase activity in the most common Gaucher disease mutant, N370S.

Authors:  Marc N Offman; Marcin Krol; Israel Silman; Joel L Sussman; Anthony H Futerman
Journal:  J Biol Chem       Date:  2010-10-27       Impact factor: 5.157

2.  Discovery, structure-activity relationship, and biological evaluation of noninhibitory small molecule chaperones of glucocerebrosidase.

Authors:  Samarjit Patnaik; Wei Zheng; Jae H Choi; Omid Motabar; Noel Southall; Wendy Westbroek; Wendy A Lea; Arash Velayati; Ehud Goldin; Ellen Sidransky; William Leister; Juan J Marugan
Journal:  J Med Chem       Date:  2012-06-08       Impact factor: 7.446

Review 3.  Protein misfolding disorders: pathogenesis and intervention.

Authors:  N Gregersen
Journal:  J Inherit Metab Dis       Date:  2006 Apr-Jun       Impact factor: 4.982

4.  Three classes of glucocerebrosidase inhibitors identified by quantitative high-throughput screening are chaperone leads for Gaucher disease.

Authors:  Wei Zheng; Janak Padia; Daniel J Urban; Ajit Jadhav; Ozlem Goker-Alpan; Anton Simeonov; Ehud Goldin; Douglas Auld; Mary E LaMarca; James Inglese; Christopher P Austin; Ellen Sidransky
Journal:  Proc Natl Acad Sci U S A       Date:  2007-08-01       Impact factor: 11.205

5.  N4-phenyl modifications of N2-(2-hydroxyl)ethyl-6-(pyrrolidin-1-yl)-1,3,5-triazine-2,4-diamines enhance glucocerebrosidase inhibition by small molecules with potential as chemical chaperones for Gaucher disease.

Authors:  Wenwei Huang; Wei Zheng; Daniel J Urban; James Inglese; Ellen Sidransky; Christopher P Austin; Craig J Thomas
Journal:  Bioorg Med Chem Lett       Date:  2007-08-28       Impact factor: 2.823

6.  Inhibiting HIV-1 integrase by shifting its oligomerization equilibrium.

Authors:  Zvi Hayouka; Joseph Rosenbluh; Aviad Levin; Shoshana Loya; Mario Lebendiker; Dmitry Veprintsev; Moshe Kotler; Amnon Hizi; Abraham Loyter; Assaf Friedler
Journal:  Proc Natl Acad Sci U S A       Date:  2007-05-08       Impact factor: 11.205

7.  Effects of pH and iminosugar pharmacological chaperones on lysosomal glycosidase structure and stability.

Authors:  Raquel L Lieberman; J Alejandro D'aquino; Dagmar Ringe; Gregory A Petsko
Journal:  Biochemistry       Date:  2009-06-09       Impact factor: 3.162

8.  Optimization and validation of two miniaturized glucocerebrosidase enzyme assays for high throughput screening.

Authors:  Daniel J Urban; Wei Zheng; Ozlem Goker-Alpan; Ajit Jadhav; Mary E Lamarca; James Inglese; Ellen Sidransky; Christopher P Austin
Journal:  Comb Chem High Throughput Screen       Date:  2008-12       Impact factor: 1.339

9.  Distinguishing the differences in β-glycosylceramidase folds, dynamics, and actions informs therapeutic uses.

Authors:  Fredj Ben Bdira; Marta Artola; Herman S Overkleeft; Marcellus Ubbink; Johannes M F G Aerts
Journal:  J Lipid Res       Date:  2018-10-02       Impact factor: 5.922

10.  The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry disease.

Authors:  Richie Khanna; Rebecca Soska; Yi Lun; Jessie Feng; Michelle Frascella; Brandy Young; Nastry Brignol; Lee Pellegrino; Sheela A Sitaraman; Robert J Desnick; Elfrida R Benjamin; David J Lockhart; Kenneth J Valenzano
Journal:  Mol Ther       Date:  2009-09-22       Impact factor: 11.454

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