The chaperone activity and toxicity of ambroxol on Gaucher cells and normal mice

[1]  Ying Sun,et al.  Isofagomine In Vivo Effects in a Neuronopathic Gaucher Disease Mouse , 2011, PloS one.

[2]  Yoshiyuki Suzuki,et al.  A Fluorescent sp2‐Iminosugar With Pharmacological Chaperone Activity for Gaucher Disease: Synthesis and Intracellular Distribution Studies , 2010, Chembiochem : a European journal of chemical biology.

[3]  Yoshiyuki Suzuki,et al.  The effect of N-octyl-β-valienamine on β-glucosidase activity in tissues of normal mice , 2010, Brain and Development.

[4]  Yoshiyuki Suzuki,et al.  The pharmacological chaperone effect of N-octyl-beta-valienamine on human mutant acid beta-glucosidases. , 2010, Blood cells, molecules & diseases.

[5]  Yoshiyuki Suzuki,et al.  Chaperone Activity of Bicyclic Nojirimycin Analogues for Gaucher Mutations in Comparison with N‐(n‐nonyl)Deoxynojirimycin , 2009, Chembiochem : a European journal of chemical biology.

[6]  Yoshitomo Hamuro,et al.  Identification and Characterization of Ambroxol as an Enzyme Enhancement Agent for Gaucher Disease* , 2009, The Journal of Biological Chemistry.

[7]  M. Horowitz,et al.  ER retention and degradation as the molecular basis underlying Gaucher disease heterogeneity. , 2007, Human molecular genetics.

[8]  Yoshiyuki Suzuki,et al.  Enzyme enhancement activity of N-octyl -β -valienamine on β-glucosidase mutants associated with Gaucher disease , 2007 .

[9]  Mario Maas,et al.  Substrate reduction therapy of glycosphingolipid storage disorders , 2006, Journal of Inherited Metabolic Disease.

[10]  Klaus-Peter Zimmer,et al.  Gaucher disease-associated glucocerebrosidases show mutation-dependent chemical chaperoning profiles. , 2005, Chemistry & biology.

[11]  J. Yodoi,et al.  Helicobacter felis-induced gastritis was suppressed in mice overexpressing thioredoxin-1 , 2005, Laboratory Investigation.

[12]  R. Brady,et al.  Correlation between enzyme activity and substrate storage in a cell culture model system for Gaucher disease , 2004, Journal of Inherited Metabolic Disease.

[13]  Seiichiro Ogawa,et al.  N-Octyl-β-valienamine up-regulates activity of F213I mutant β-glucosidase in cultured cells: a potential chemical chaperone therapy for Gaucher disease , 2004 .

[14]  Ernest Beutler,et al.  Chemical chaperones increase the cellular activity of N370S β-glucosidase: A therapeutic strategy for Gaucher disease , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[15]  R. Dwek,et al.  Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy , 2001, Journal of Inherited Metabolic Disease.

[16]  C. Scriver,et al.  The Metabolic and Molecular Bases of Inherited Disease, 8th Edition 2001 , 2001, Journal of Inherited Metabolic Disease.

[17]  R. Dwek,et al.  Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis , 2000, The Lancet.

[18]  N. Leslie,et al.  Enzyme therapy for Gaucher disease: the first 5 years. , 1998, Blood reviews.

[19]  J. Dambrosia,et al.  Prospective study of neurological responses to treatment with macrophage‐targeted glucocerebrosidase in patients with type 3 Gaucher's disease , 1997, Annals of neurology.

[20]  C. Prows,et al.  Gaucher disease: enzyme therapy in the acute neuronopathic variant. , 1997, American journal of medical genetics.

[21]  K. Yamagishi,et al.  Synthesis of potent β-D-glucocerebrosidase inhibitors: N-alkyl-β-valienamines , 1996 .

[22]  E. Beutler,et al.  Failure of Alglucerase Infused into Gaucher Disease Patients to Localize in Marrow Macrophages , 1995, Molecular medicine.

[23]  J. Dambrosia,et al.  Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease. , 1991, The New England journal of medicine.

[24]  K. Suzuki,et al.  Effect of a heat-stable factor in human placenta on glucosylceramidase, glucosylsphingosine glucosyl hydrolase, and acid beta-glucosidase activities. , 1987, Clinical biochemistry.

[25]  J. Jonkman,et al.  Dose-dependent uricosuric effect of ambroxol , 2004, European Journal of Clinical Pharmacology.

[26]  N. Kondo,et al.  Improvement of neurological symptoms by enzyme replacement therapy for Gaucher disease type IIIb , 2001, European Journal of Pediatrics.

[27]  R. Van Tiggelen,et al.  Gaucher disease , 2019, Haematology.