Literature DB >> 17894779

Pharmacologic chaperoning as a strategy to treat Gaucher disease.

Zhanqian Yu1, Anu R Sawkar, Jeffery W Kelly.   

Abstract

We briefly introduce the most common lysosomal storage disorder, Gaucher disease, concisely describe the Food and Drug Administration approved strategies to ameliorate Gaucher disease, and then outline the emerging pharmacologic chaperone strategy that offers the promise to remedy this malady.

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Year:  2007        PMID： 17894779     DOI： 10.1111/j.1742-4658.2007.06042.x

Source DB:  PubMed          Journal:  FEBS J        ISSN： 1742-464X            Impact factor:   5.542


  32 in total

1.  Ex vivo and in vivo effects of isofagomine on acid β-glucosidase variants and substrate levels in Gaucher disease.

Authors:  Ying Sun; Benjamin Liou; You-Hai Xu; Brian Quinn; Wujuan Zhang; Rick Hamler; Kenneth D R Setchell; Gregory A Grabowski
Journal:  J Biol Chem       Date:  2011-12-13       Impact factor: 5.157

2.  Binding of 3,4,5,6-tetrahydroxyazepanes to the acid-β-glucosidase active site: implications for pharmacological chaperone design for Gaucher disease.

Authors:  Susan D Orwig; Yun Lei Tan; Neil P Grimster; Zhanqian Yu; Evan T Powers; Jeffery W Kelly; Raquel L Lieberman
Journal:  Biochemistry       Date:  2011-11-14       Impact factor: 3.162

Review 3.  The delicate balance between secreted protein folding and endoplasmic reticulum-associated degradation in human physiology.

Authors:  Christopher J Guerriero; Jeffrey L Brodsky
Journal:  Physiol Rev       Date:  2012-04       Impact factor: 37.312

4.  Molecular probes: Getting lucky in the lysosome.

Authors:  Ethan D Goddard-Borger; Tom Wennekes; Stephen G Withers
Journal:  Nat Chem Biol       Date:  2010-12       Impact factor: 15.040

5.  Pharmacological chaperones facilitate the post-ER transport of recombinant N370S mutant β-glucocerebrosidase in plant cells: evidence that N370S is a folding mutant.

Authors:  Gholamreza Babajani; Michael B Tropak; Don J Mahuran; Allison R Kermode
Journal:  Mol Genet Metab       Date:  2012-04-26       Impact factor: 4.797

6.  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

Review 7.  Lysosomal storage disorders in the newborn.

Authors:  Orna Staretz-Chacham; Tess C Lang; Mary E LaMarca; Donna Krasnewich; Ellen Sidransky
Journal:  Pediatrics       Date:  2009-04       Impact factor: 7.124

Review 8.  Chemical and biological approaches for adapting proteostasis to ameliorate protein misfolding and aggregation diseases: progress and prognosis.

Authors:  Susan L Lindquist; Jeffery W Kelly
Journal:  Cold Spring Harb Perspect Biol       Date:  2011-12-01       Impact factor: 10.005

Review 9.  Expanding the number of 'druggable' targets: non-enzymes and protein-protein interactions.

Authors:  Leah N Makley; Jason E Gestwicki
Journal:  Chem Biol Drug Des       Date:  2013-01       Impact factor: 2.817

10.  Gaucher iPSC-derived macrophages produce elevated levels of inflammatory mediators and serve as a new platform for therapeutic development.

Authors:  Leelamma M Panicker; Diana Miller; Ola Awad; Vivek Bose; Yu Lun; Tea Soon Park; Elias T Zambidis; Judi A Sgambato; Ricardo A Feldman
Journal:  Stem Cells       Date:  2014-09       Impact factor: 6.277
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