Literature DB >> 19397268

Rational design and synthesis of highly potent pharmacological chaperones for treatment of N370S mutant Gaucher disease.

Guan-Nan Wang1, Gabriele Reinkensmeier, Si-Wei Zhang, Jian Zhou, Liang-Ren Zhang, Li-He Zhang, Terry D Butters, Xin-Shan Ye.   

Abstract

Highly potent N-substituted delta-lactams have been rationally designed and synthesized by a concise route with a one-pot tandem reaction as key step. These iminosugars show weak inhibition of wild-type beta-glucocerebrosidase but 3- to 6-fold increases in mutant enzyme activity (N370S).

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Year:  2009        PMID： 19397268     DOI： 10.1021/jm801506m

Source DB:  PubMed          Journal:  J Med Chem        ISSN： 0022-2623            Impact factor:   7.446


  12 in total

1.  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

2.  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

3.  Orphan diseases: state of the drug discovery art.

Authors:  Claude-Henry Volmar; Claes Wahlestedt; Shaun P Brothers
Journal:  Wien Med Wochenschr       Date:  2016-01-27

4.  Rapid assembly of a library of lipophilic iminosugars via the thiol-ene reaction yields promising pharmacological chaperones for the treatment of Gaucher disease.

Authors:  Ethan D Goddard-Borger; Michael B Tropak; Sayuri Yonekawa; Christina Tysoe; Don J Mahuran; Stephen G Withers
Journal:  J Med Chem       Date:  2012-03-06       Impact factor: 7.446

5.  Synthetic N-alkylated iminosugars as new potential immunosuppressive agents.

Authors:  Guan-Nan Wang; Yulan Xiong; Jia Ye; Li-He Zhang; Xin-Shan Ye
Journal:  ACS Med Chem Lett       Date:  2011-07-05       Impact factor: 4.345

Review 6.  Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders.

Authors:  Kenneth J Valenzano; Richie Khanna; Allan C Powe; Robert Boyd; Gary Lee; John J Flanagan; Elfrida R Benjamin
Journal:  Assay Drug Dev Technol       Date:  2011-06       Impact factor: 1.738

Review 7.  Small molecules as therapeutic agents for inborn errors of metabolism.

Authors:  Leslie Matalonga; Laura Gort; Antonia Ribes
Journal:  J Inherit Metab Dis       Date:  2016-12-13       Impact factor: 4.982

8.  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

9.  Synthesis of lipophilic 1-deoxygalactonojirimycin derivatives as D-galactosidase inhibitors.

Authors:  Georg Schitter; Elisabeth Scheucher; Andreas J Steiner; Arnold E Stütz; Martin Thonhofer; Chris A Tarling; Stephen G Withers; Jacqueline Wicki; Katrin Fantur; Eduard Paschke; Don J Mahuran; Brigitte A Rigat; Michael Tropak; Tanja M Wrodnigg
Journal:  Beilstein J Org Chem       Date:  2010-03-01       Impact factor: 2.883

10.  Orthoester functionalized N-guanidino derivatives of 1,5-dideoxy-1,5-imino-d-xylitol as pH-responsive inhibitors of β-glucocerebrosidase.

Authors:  Alen Sevšek; Javier Sastre Toraño; Linda Quarles van Ufford; Ed E Moret; Roland J Pieters; Nathaniel I Martin
Journal:  Medchemcomm       Date:  2017-10-10       Impact factor: 3.597
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