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Volumn 53, Issue 14, 2010, Pages 5248-5255

Click chemistry approach to new N-substituted aminocyclitols as potential pharmacological chaperones for gaucher disease

Author keywords

[No Author keywords available]

Indexed keywords

1,2,3 TRIAZOLE DERIVATIVE; AMINOCYCLITOL; CHAPERONE;

EID: 77954749600     PISSN: 00222623     EISSN: 15204804     Source Type: Journal    
DOI: 10.1021/jm100198t     Document Type: Article
Times cited : (61)

References (50)
  • 1
    • 0031450742 scopus 로고    scopus 로고
    • Gaucher's disease: Past, present and future
    • Brady, R. O. Gaucher's disease: past, present and future Baillieres Clin. Haematol. 1997, 10, 621-634
    • (1997) Baillieres Clin. Haematol. , vol.10 , pp. 621-634
    • Brady, R.O.1
  • 2
    • 33745293617 scopus 로고    scopus 로고
    • Therapeutic strategies to ameliorate lysosomal storage disorders - A focus on Gaucher disease
    • Sawkar, A. R.; D'Haeze, W.; Kelly, J. W. Therapeutic strategies to ameliorate lysosomal storage disorders - a focus on Gaucher disease Cell. Mol. Life Sci. 2006, 63, 1179-1192
    • (2006) Cell. Mol. Life Sci. , vol.63 , pp. 1179-1192
    • Sawkar, A.R.1    D'Haeze, W.2    Kelly, J.W.3
  • 4
    • 0043235841 scopus 로고    scopus 로고
    • A contradictory treatment for lysosomal storage disorders: Inhibitors enhance mutant enzyme activity
    • Fan, J.-Q. A contradictory treatment for lysosomal storage disorders: inhibitors enhance mutant enzyme activity Trends Pharmacol. Sci. 2003, 24, 355-360
    • (2003) Trends Pharmacol. Sci. , vol.24 , pp. 355-360
    • Fan, J.-Q.1
  • 5
    • 33947221095 scopus 로고    scopus 로고
    • Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders
    • Butters, T. D. Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders Expert Opin. Pharmacother. 2007, 8, 427-435
    • (2007) Expert Opin. Pharmacother. , vol.8 , pp. 427-435
    • Butters, T.D.1
  • 6
    • 41349085850 scopus 로고    scopus 로고
    • Treatment perspectives for the lysosomal storage diseases
    • Grabowski, G. A. Treatment perspectives for the lysosomal storage diseases Expert Opin. Emerging Drugs 2008, 13, 197-211
    • (2008) Expert Opin. Emerging Drugs , vol.13 , pp. 197-211
    • Grabowski, G.A.1
  • 7
  • 10
    • 70849126444 scopus 로고    scopus 로고
    • Characterization of gene-activated human acid-{beta}-glucosidase: Crystal structure, glycan composition and internalization into macrophages
    • Brumshtein, B.; Salinas, P.; Peterson, B.; Chan, V.; Silman, I.; Sussman, J. L.; Savickas, P. J.; Robinson, G. S.; Futerman, A. H. Characterization of gene-activated human acid-{beta}-glucosidase: crystal structure, glycan composition and internalization into macrophages Glycobiology 2010, 20, 24-32
    • (2010) Glycobiology , vol.20 , pp. 24-32
    • Brumshtein, B.1    Salinas, P.2    Peterson, B.3    Chan, V.4    Silman, I.5    Sussman, J.L.6    Savickas, P.J.7    Robinson, G.S.8    Futerman, A.H.9
  • 11
    • 66649137718 scopus 로고    scopus 로고
    • Effects of pH and iminosugar pharmacological chaperones on lysosomal glycosidase structure and stability
    • Lieberman, R. L.; D'Aquino, J, A.; Ringe, D.; Petsko, G. A. Effects of pH and iminosugar pharmacological chaperones on lysosomal glycosidase structure and stability Biochemistry 2009, 48, 4816-4827
    • (2009) Biochemistry , vol.48 , pp. 4816-4827
    • Lieberman, R.L.1    D'Aquino, J.A.2    Ringe, D.3    Petsko, G.A.4
  • 13
    • 21244456941 scopus 로고    scopus 로고
    • X-ray structure of human acid-beta-glucosidase covalently bound to conduritol-B-epoxide. Implications for Gaucher disease
    • Premkumar, L.; Sawkar, A. R.; Boldin-Adamsky, S.; Toker, L.; Silman, I.; Kelly, J. W.; Futerman, A. H.; Sussman, J. L. X-ray structure of human acid-beta-glucosidase covalently bound to conduritol-B-epoxide. Implications for Gaucher disease J. Biol. Chem. 2005, 280, 23815-23819
    • (2005) J. Biol. Chem. , vol.280 , pp. 23815-23819
    • Premkumar, L.1    Sawkar, A.R.2    Boldin-Adamsky, S.3    Toker, L.4    Silman, I.5    Kelly, J.W.6    Futerman, A.H.7    Sussman, J.L.8
  • 14
    • 35348989145 scopus 로고    scopus 로고
    • Crystal structures of complexes of N -butyl- and N -nonyl- deoxynojirimycin bound to acid beta-glucosidase: Insights into the mechanism of chemical chaperone action in Gaucher disease
    • Brumshtein, B.; Greenblatt, H. M.; Butters, T. D.; Shaaltiel, Y.; Aviezer, D.; Silman, I.; Futerman, A. H.; Sussman, J. L. Crystal structures of complexes of N -butyl- and N -nonyl-deoxynojirimycin bound to acid beta-glucosidase: insights into the mechanism of chemical chaperone action in Gaucher disease J. Biol. Chem. 2007, 282, 29052-29058
    • (2007) J. Biol. Chem. , vol.282 , pp. 29052-29058
    • Brumshtein, B.1    Greenblatt, H.M.2    Butters, T.D.3    Shaaltiel, Y.4    Aviezer, D.5    Silman, I.6    Futerman, A.H.7    Sussman, J.L.8
  • 19
    • 34548540634 scopus 로고    scopus 로고
    • Aminocyclitols as pharmacological chaperones for glucocerebrosidase, a defective enzyme in Gaucher disease
    • Egido-Gabas, M.; Canals, D.; Casas, J.; Llebaria, A.; Delgado, A. Aminocyclitols as pharmacological chaperones for glucocerebrosidase, a defective enzyme in Gaucher disease ChemMedChem 2007, 2, 992-994
    • (2007) ChemMedChem , vol.2 , pp. 992-994
    • Egido-Gabas, M.1    Canals, D.2    Casas, J.3    Llebaria, A.4    Delgado, A.5
  • 20
    • 59349084725 scopus 로고    scopus 로고
    • Promising results of the chaperone effect caused by iminosugars and aminocyclitol derivatives on mutant glucocerebrosidases causing Gaucher disease
    • Sanchez-Olle, G.; Duque, J.; Egido-Gabas, M.; Casas, J.; Lluch, M.; Chabas, A.; Grinberg, D.; Vilageliu, L. Promising results of the chaperone effect caused by iminosugars and aminocyclitol derivatives on mutant glucocerebrosidases causing Gaucher disease Blood Cells, Mol. Dis. 2009, 42, 159-166
    • (2009) Blood Cells, Mol. Dis. , vol.42 , pp. 159-166
    • Sanchez-Olle, G.1    Duque, J.2    Egido-Gabas, M.3    Casas, J.4    Lluch, M.5    Chabas, A.6    Grinberg, D.7    Vilageliu, L.8
  • 21
    • 33846649594 scopus 로고    scopus 로고
    • Combinatorial approach to N-substituted aminocyclitol libraries by solution-phase parallel synthesis and preliminary evaluation as glucocerebrosidase inhibitors
    • Serrano, P.; Casas, J.; Zucco, M.; Emeric, G.; Egido-Gabas, M.; Llebaria, A.; Delgado, A. Combinatorial approach to N-substituted aminocyclitol libraries by solution-phase parallel synthesis and preliminary evaluation as glucocerebrosidase inhibitors J. Comb. Chem 2007, 9, 43-52
    • (2007) J. Comb. Chem , vol.9 , pp. 43-52
    • Serrano, P.1    Casas, J.2    Zucco, M.3    Emeric, G.4    Egido-Gabas, M.5    Llebaria, A.6    Delgado, A.7
  • 22
    • 25444489938 scopus 로고    scopus 로고
    • Regio- and stereoselective synthesis of aminoinositols and 1,2-diaminoinositols from conduritol B epoxide
    • Serrano, P.; Llebaria, A.; Delgado, A. Regio- and stereoselective synthesis of aminoinositols and 1,2-diaminoinositols from conduritol B epoxide J. Org. Chem. 2005, 70, 7829-7840
    • (2005) J. Org. Chem. , vol.70 , pp. 7829-7840
    • Serrano, P.1    Llebaria, A.2    Delgado, A.3
  • 23
    • 22944475824 scopus 로고    scopus 로고
    • On the regio- and stereoselective synthesis of aminocyclitols from cyclitol epoxides: The effect of Li as a chelating agent
    • Serrano, P.; Llebaria, A.; Vazquez, J.; de Pablo, J.; Anglada, J. M.; Delgado, A. On the regio- and stereoselective synthesis of aminocyclitols from cyclitol epoxides: the effect of Li as a chelating agent Chem. - Eur. J. 2005, 11, 4465-4472
    • (2005) Chem. - Eur. J. , vol.11 , pp. 4465-4472
    • Serrano, P.1    Llebaria, A.2    Vazquez, J.3    De Pablo, J.4    Anglada, J.M.5    Delgado, A.6
  • 24
    • 77954732942 scopus 로고    scopus 로고
    • The recovery of recombinant GlcCerase activity after thermal denaturation is currently used as an in vitro model assay for potential pharmacological chaperones (see ref 37)
    • The recovery of recombinant GlcCerase activity after thermal denaturation is currently used as an in vitro model assay for potential pharmacological chaperones (see ref 37).
  • 25
    • 0037099395 scopus 로고    scopus 로고
    • A stepwise Huisgen cycloaddition process: Copper(I)-catalyzed regioselective "ligation" of azides and terminal alkynes
    • Rostovtsev, V. V.; Green, L. G.; Fokin, V. V.; Sharpless, K. B. A stepwise Huisgen cycloaddition process: copper(I)-catalyzed regioselective "ligation" of azides and terminal alkynes Angew. Chem., Int. Ed. 2002, 41, 2596-2599
    • (2002) Angew. Chem., Int. Ed. , vol.41 , pp. 2596-2599
    • Rostovtsev, V.V.1    Green, L.G.2    Fokin, V.V.3    Sharpless, K.B.4
  • 26
    • 34250353931 scopus 로고    scopus 로고
    • Click chemistry - What's in a name? Triazole synthesis and beyond
    • Gil, M. V.; Arévalo, M. J.; López, O. Click chemistry - What's in a name? Triazole synthesis and beyond Synthesis 2007, 1589-1620
    • (2007) Synthesis , pp. 1589-1620
    • Gil, M.V.1    Arévalo, M.J.2    López, O.3
  • 27
    • 34447330139 scopus 로고    scopus 로고
    • Practical synthesis of (-)-1-amino-1-deoxy-myo-inositol from achiral precursors
    • Gonzalez-Bulnes, P.; Casas, J.; Delgado, A.; Llebaria, A. Practical synthesis of (-)-1-amino-1-deoxy-myo-inositol from achiral precursors Carbohydr. Res. 2007, 342, 1947-1952
    • (2007) Carbohydr. Res. , vol.342 , pp. 1947-1952
    • Gonzalez-Bulnes, P.1    Casas, J.2    Delgado, A.3    Llebaria, A.4
  • 28
    • 77954716727 scopus 로고    scopus 로고
    • 50 values below 40 μM at pH 5.0 (pH for optimal enzyme activity)
    • 50 values below 40 μM at pH 5.0 (pH for optimal enzyme activity).
  • 29
    • 77954700120 scopus 로고    scopus 로고
    • Only compounds 2b, 2c, and 3a showed GCS inhibition (A549 cells), albeit not higher than 30% at the indicated concentration (250 μM). For a matter of comparison, a 70% inhibition of enzyme activity was found for NBDNJ at 10 μM. For experimental details, see the Supporting Information
    • Only compounds 2b, 2c, and 3a showed GCS inhibition (A549 cells), albeit not higher than 30% at the indicated concentration (250 μM). For a matter of comparison, a 70% inhibition of enzyme activity was found for NBDNJ at 10 μM. For experimental details, see the Supporting Information.
  • 30
    • 77954755744 scopus 로고    scopus 로고
    • Compounds were screened against the following glycosidases: sweet almond β-glucosidase, yeast α-glucosidase, rice α-glucosidase, rice α-galactosidase, and bovine liver β-galactosidase. For further details, see the Supporting Information
    • Compounds were screened against the following glycosidases: sweet almond β-glucosidase, yeast α-glucosidase, rice α-glucosidase, rice α-galactosidase, and bovine liver β-galactosidase. For further details, see the Supporting Information.
  • 31
    • 77954727874 scopus 로고    scopus 로고
    • i ratio as a measure of compound efficacy, the following values are obtained: 1c ≥ 900; 1d ≥ 3300; 1e = 916; 1f = 237
    • i ratio as a measure of compound efficacy, the following values are obtained: 1c ≥ 900; 1d ≥ 3300; 1e = 916; 1f = 237.
  • 33
    • 45749138489 scopus 로고    scopus 로고
    • MM-GB/SA rescoring of docking poses in structure-based lead optimization
    • Guimaraes, C. R.; Cardozo, M. MM-GB/SA rescoring of docking poses in structure-based lead optimization J. Chem. Inf. Model. 2008, 48, 958-970
    • (2008) J. Chem. Inf. Model. , vol.48 , pp. 958-970
    • Guimaraes, C.R.1    Cardozo, M.2
  • 35
    • 37349065872 scopus 로고    scopus 로고
    • The 1,2,3-triazole ring as a peptido- and olefinomimetic element: Discovery of click vanilloids and cannabinoids
    • Appendino, G.; Bacchiega, S.; Minassi, A.; Cascio, M. G.; De Petrocellis, L.; Di Marzo, V. The 1,2,3-triazole ring as a peptido- and olefinomimetic element: discovery of click vanilloids and cannabinoids Angew. Chem., Int. Ed. 2007, 46, 9312-9315
    • (2007) Angew. Chem., Int. Ed. , vol.46 , pp. 9312-9315
    • Appendino, G.1    Bacchiega, S.2    Minassi, A.3    Cascio, M.G.4    De Petrocellis, L.5    Di Marzo, V.6
  • 36
    • 43049130754 scopus 로고    scopus 로고
    • 1,5-Disubstituted 1,2,3-triazoles as cis-restricted analogues of combretastatin A-4: Synthesis, molecular modeling and evaluation as cytotoxic agents and inhibitors of tubulin
    • Odlo, K.; Hentzen, J.; dit Chabert, J. F.; Ducki, S.; Gani, O. A.; Sylte, I.; Skrede, M.; Florenes, V. A.; Hansen, T. V. 1,5-Disubstituted 1,2,3-triazoles as cis-restricted analogues of combretastatin A-4: synthesis, molecular modeling and evaluation as cytotoxic agents and inhibitors of tubulin Bioorg. Med. Chem. 2008, 16, 4829-4838
    • (2008) Bioorg. Med. Chem. , vol.16 , pp. 4829-4838
    • Odlo, K.1    Hentzen, J.2    Dit Chabert, J.F.3    Ducki, S.4    Gani, O.A.5    Sylte, I.6    Skrede, M.7    Florenes, V.A.8    Hansen, T.V.9
  • 37
    • 0037180511 scopus 로고    scopus 로고
    • Chemical chaperones increase the cellular activity of N370S beta-glucosidase: A therapeutic strategy for Gaucher disease
    • Sawkar, A. R.; Cheng, W. C.; Beutler, E.; Wong, C. H.; Balch, W. E.; Kelly, J. W. Chemical chaperones increase the cellular activity of N370S beta-glucosidase: a therapeutic strategy for Gaucher disease Proc. Natl. Acad. Sci. U.S.A. 2002, 99, 15428-15433
    • (2002) Proc. Natl. Acad. Sci. U.S.A. , vol.99 , pp. 15428-15433
    • Sawkar, A.R.1    Cheng, W.C.2    Beutler, E.3    Wong, C.H.4    Balch, W.E.5    Kelly, J.W.6
  • 38
    • 77954704509 scopus 로고    scopus 로고
    • version 8.5; Schrödinger, LLC: New York
    • Maestro, version 8.5; Schrödinger, LLC: New York, 2008.
    • (2008) Maestro
  • 39
    • 70450167464 scopus 로고    scopus 로고
    • version 9.6; Schrödinger, LLC: New York
    • MacroModel, version 9.6; Schrödinger, LLC: New York, 2005.
    • (2005) MacroModel
  • 40
    • 0029912748 scopus 로고    scopus 로고
    • Development and testing of the OPLS all-atom force field on conformational energetics and properties of organic liquids
    • Jorgensen, W. L.; Maxwell, D. S.; TiradoRives, J. Development and testing of the OPLS all-atom force field on conformational energetics and properties of organic liquids J. Am. Chem. Soc. 1996, 118, 11225-11236
    • (1996) J. Am. Chem. Soc. , vol.118 , pp. 11225-11236
    • Jorgensen, W.L.1    Maxwell, D.S.2    Tiradorives, J.3
  • 41
    • 0344778061 scopus 로고
    • Semianalytical treatment of solvation for molecular mechanics and dynamics
    • Still, W. C.; Tempczyk, A.; Hawley, R. C.; Hendrickson, T. Semianalytical treatment of solvation for molecular mechanics and dynamics J. Am. Chem. Soc. 1990, 112, 6127-6129
    • (1990) J. Am. Chem. Soc. , vol.112 , pp. 6127-6129
    • Still, W.C.1    Tempczyk, A.2    Hawley, R.C.3    Hendrickson, T.4
  • 42
    • 77954715732 scopus 로고    scopus 로고
    • version 5.0; Schrödinger, LLC: New York
    • Glide, version 5.0; Schrödinger, LLC: New York, 2008.
    • (2008) Glide
  • 44
    • 1642310340 scopus 로고    scopus 로고
    • Glide: A new approach for rapid, accurate docking and scoring. 2. Enrichment factors in database screening
    • Halgren, T. A.; Murphy, R. B.; Friesner, R. A.; Beard, H. S.; Frye, L. L.; Pollard, W. T.; Banks, J. L. Glide: a new approach for rapid, accurate docking and scoring. 2. Enrichment factors in database screening J. Med. Chem. 2004, 47, 1750-1759
    • (2004) J. Med. Chem. , vol.47 , pp. 1750-1759
    • Halgren, T.A.1    Murphy, R.B.2    Friesner, R.A.3    Beard, H.S.4    Frye, L.L.5    Pollard, W.T.6    Banks, J.L.7
  • 46
    • 0037149134 scopus 로고    scopus 로고
    • Accurate prediction of acidity constants in aqueous solution via density functional theory and self-consistent reaction field methods
    • Klicic, J. J.; Friesner, R. A.; Liu, S. Y.; Guida, W. C. Accurate prediction of acidity constants in aqueous solution via density functional theory and self-consistent reaction field methods J. Phys. Chem. A 2002, 106, 1327-1335
    • (2002) J. Phys. Chem. A , vol.106 , pp. 1327-1335
    • Klicic, J.J.1    Friesner, R.A.2    Liu, S.Y.3    Guida, W.C.4
  • 47
    • 77954703448 scopus 로고    scopus 로고
    • version 7.5; Schrödinger, LLC: New York
    • Jaguar, version 7.5; Schrödinger, LLC: New York, 2008.
    • (2008) Jaguar
  • 49
    • 77954739799 scopus 로고    scopus 로고
    • version 2.2; Schrödinger, LLC: New York
    • LigPrep, version 2.2; Schrödinger, LLC: New York, 2005.
    • (2005) LigPrep
  • 50
    • 0004313709 scopus 로고    scopus 로고
    • version 2008.10; Chemical Computing Group: Montreal, Quebec, Canada
    • Molecular Operating Environment, version 2008.10; Chemical Computing Group: Montreal, Quebec, Canada, 2008.
    • (2008) Molecular Operating Environment


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