Literature DB >> 22606365

Non-iminosugar glucocerebrosidase small molecule chaperones.

Juan Jose Marugan1, Wenwei Huang, Omid Motabar, Wei Zheng, Jingbo Xiao, Samarjit Patnaik, Noel Southall, Wendy Westbroek, Wendy A Lea, Anton Simeonov, Ehud Goldin, Maria A Debernardi, Ellen Sidransky.   

Abstract

Small molecule chaperones are a promising therapeutic approach for the Lysosomal Storage Disorders (LSDs). Here, we report the discovery of a new series of non-iminosugar glucocerebrosidase inhibitors with chaperone capacity, and describe their structure activity relationship (SAR), selectivity, cell activity phamacokinetics.

Entities:  

Year:  2011        PMID： 22606365      PMCID： PMC3351140          DOI： 10.1039/C1MD00200G

Source DB:  PubMed          Journal:  Medchemcomm        ISSN： 2040-2503            Impact factor:   3.597


  18 in total

Review 1.  A contradictory treatment for lysosomal storage disorders: inhibitors enhance mutant enzyme activity.

Authors:  Jian-Qiang Fan
Journal:  Trends Pharmacol Sci       Date:  2003-07       Impact factor: 14.819

Review 2.  Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.

Authors:  Terry D Butters
Journal:  Expert Opin Pharmacother       Date:  2007-03       Impact factor: 3.889

3.  Isofagomine- and 2,5-anhydro-2,5-imino-D-glucitol-based glucocerebrosidase pharmacological chaperones for Gaucher disease intervention.

Authors:  Zhanqian Yu; Anu R Sawkar; Lisa J Whalen; Chi-Huey Wong; Jeffery W Kelly
Journal:  J Med Chem       Date:  2007-01-11       Impact factor: 7.446

4.  Aminocyclitols as pharmacological chaperones for glucocerebrosidase, a defective enzyme in Gaucher disease.

Authors:  Meritxell Egido-Gabás; Daniel Canals; Josefina Casas; Amadeu Llebaria; Antonio Delgado
Journal:  ChemMedChem       Date:  2007-07       Impact factor: 3.466

5.  Alpha-1-C-octyl-1-deoxynojirimycin as a pharmacological chaperone for Gaucher disease.

Authors:  Liang Yu; Kyoko Ikeda; Atsushi Kato; Isao Adachi; Guillaume Godin; Philippe Compain; Olivier Martin; Naoki Asano
Journal:  Bioorg Med Chem       Date:  2006-08-21       Impact factor: 3.641

Review 6.  Imino sugar inhibitors for treating the lysosomal glycosphingolipidoses.

Authors:  Terry D Butters; Raymond A Dwek; Frances M Platt
Journal:  Glycobiology       Date:  2005-05-18       Impact factor: 4.313

7.  Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.

Authors:  Juan J Marugan; Wei Zheng; Omid Motabar; Noel Southall; Ehud Goldin; Wendy Westbroek; Barbara K Stubblefield; Ellen Sidransky; Ronald A Aungst; Wendy A Lea; Anton Simeonov; William Leister; Christopher P Austin
Journal:  J Med Chem       Date:  2011-01-20       Impact factor: 7.446

8.  Cognitive outcome in treated patients with chronic neuronopathic Gaucher disease.

Authors:  Ozlem Goker-Alpan; Edythe A Wiggs; Michael J Eblan; William Benko; Shira G Ziegler; Ellen Sidransky; Raphael Schiffmann
Journal:  J Pediatr       Date:  2008-02-14       Impact factor: 4.406

Review 9.  Gaucher disease: mutation and polymorphism spectrum in the glucocerebrosidase gene (GBA).

Authors:  Kathleen S Hruska; Mary E LaMarca; C Ronald Scott; Ellen Sidransky
Journal:  Hum Mutat       Date:  2008-05       Impact factor: 4.878

10.  Multivalent iminosugars to modulate affinity and selectivity for glycosidases.

Authors:  Jennifer Diot; M Isabel García-Moreno; Sébastien G Gouin; Carmen Ortiz Mellet; Karsten Haupt; José Kovensky
Journal:  Org Biomol Chem       Date:  2008-11-18       Impact factor: 3.876
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  9 in total

1.  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

2.  Macrophage models of Gaucher disease for evaluating disease pathogenesis and candidate drugs.

Authors:  Elma Aflaki; Barbara K Stubblefield; Emerson Maniwang; Grisel Lopez; Nima Moaven; Ehud Goldin; Juan Marugan; Samarjit Patnaik; Amalia Dutra; Noel Southall; Wei Zheng; Nahid Tayebi; Ellen Sidransky
Journal:  Sci Transl Med       Date:  2014-06-11       Impact factor: 17.956

3.  A New Glucocerebrosidase Chaperone Reduces α-Synuclein and Glycolipid Levels in iPSC-Derived Dopaminergic Neurons from Patients with Gaucher Disease and Parkinsonism.

Authors:  Elma Aflaki; Daniel K Borger; Nima Moaven; Barbara K Stubblefield; Steven A Rogers; Samarjit Patnaik; Frank J Schoenen; Wendy Westbroek; Wei Zheng; Patricia Sullivan; Hideji Fujiwara; Rohini Sidhu; Zayd M Khaliq; Grisel J Lopez; David S Goldstein; Daniel S Ory; Juan Marugan; Ellen Sidransky
Journal:  J Neurosci       Date:  2016-07-13       Impact factor: 6.167

Review 4.  Emptying the stores: lysosomal diseases and therapeutic strategies.

Authors:  Frances M Platt
Journal:  Nat Rev Drug Discov       Date:  2017-11-17       Impact factor: 84.694

5.  Design and Synthesis of Potent Quinazolines as Selective β-Glucocerebrosidase Modulators.

Authors:  Jianbin Zheng; Long Chen; Michael Schwake; Richard B Silverman; Dimitri Krainc
Journal:  J Med Chem       Date:  2016-09-06       Impact factor: 7.446

Review 6.  Second-Generation Pharmacological Chaperones: Beyond Inhibitors.

Authors:  My Lan Tran; Yves Génisson; Stéphanie Ballereau; Cécile Dehoux
Journal:  Molecules       Date:  2020-07-09       Impact factor: 4.411

7.  Ambroxol as a pharmacological chaperone for mutant glucocerebrosidase.

Authors:  Inna Bendikov-Bar; Gali Maor; Mirella Filocamo; Mia Horowitz
Journal:  Blood Cells Mol Dis       Date:  2012-11-14       Impact factor: 3.039

Review 8.  Tuning protein folding in lysosomal storage diseases: the chemistry behind pharmacological chaperones.

Authors:  David M Pereira; Patrícia Valentão; Paula B Andrade
Journal:  Chem Sci       Date:  2018-01-10       Impact factor: 9.825

Review 9.  Advances in the Development of Pharmacological Chaperones for the Mucopolysaccharidoses.

Authors:  Juan Camilo Losada Díaz; Jacobo Cepeda Del Castillo; Edwin Alexander Rodriguez-López; Carlos J Alméciga-Díaz
Journal:  Int J Mol Sci       Date:  2019-12-29       Impact factor: 5.923
  9 in total

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