Stemming the tide: Glycosphingolipid synthesis inhibitors as therapy for storage diseases

Glycobiology

Volumn 10, Issue 12, 2000, Pages 1249-1258

  Tifft, Cynthia J ^b   Proia, Richard L ^a,c  

^a NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES   (United States)

^b CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^c NATIONAL INSTITUTES OF HEALTH   (United States)

Author keywords

Glycolipid function; Lysosomal storage diseases; Neudegenerative diseases; Sphingolipids; Substrate deprivation

Indexed keywords

BETA GALACTOSIDASE; BETA GLUCOSIDASE; BETA N ACETYLHEXOSAMINIDASE A; BETA N ACETYLHEXOSAMINIDASE B; BLOCKING AGENT; CERAMIDE; CERAMIDE DERIVATIVE; CHOLESTEROL; DEXTRO 4' HYDROXY 1 PHENYL 2 PALMITOYLAMINO 2 PYRROLIDINO 1 PROPANOL; DEXTRO THREO 1 (3',4' ETHYLENEDIOXY)PHENYL 2 PALMITOYLAMINO 3 PYRROLIDINO 1 PROPANOL; DEXTRO THREO 1 PHENYL 2 DECANOYLAMINO 3 MORPHOLINO 1 PROPANOL; GALABIOSYLCERAMIDE; GALACTOSYLCERAMIDE; GANGLIOSIDE; GANGLIOSIDE GA2; GANGLIOSIDE GM2; GANGLIOSIDE GM3; GLOBOTRIAOSYLCERAMIDE; GLYCAN; GLYCOSPHINGOLIPID; GLYCOSPHINGOLIPID SYNTHESIS INHIBITOR; KERATAN SULFATE; LACTOSYLCERAMIDE; MIGLUSTAT; N BUTYLDEOXYGALACTONOJIRIMYCIN; OLIGOSACCHARIDE; PROPANOL; SPHINGOMYELIN; SPHINGOSINE; SULFATIDE; UNCLASSIFIED DRUG; UNINDEXED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; BIOSYNTHESIS; CELL MEMBRANE; CENTRAL NERVOUS SYSTEM; CHEMICAL COMPOSITION; CHEMICAL STRUCTURE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DIARRHEA; DISEASE SEVERITY; DRUG METABOLISM; DRUG STRUCTURE; EMBRYO DEVELOPMENT; EUKARYOTIC CELL; FABRY DISEASE; GAUCHER DISEASE; GENETICS; GM1 GANGLIOSIDOSIS; HUMAN; HYDROPHOBICITY; INHERITANCE; LIFESPAN; LYSOSOME STORAGE DISEASE; MOUSE; NERVE DEGENERATION; NEUROPATHY; NIEMANN PICK DISEASE; NONHUMAN; PATHOGENESIS; PRIORITY JOURNAL; REVIEW; SANDHOFF DISEASE; STORAGE DISEASE; TAY SACHS DISEASE; WEIGHT REDUCTION;

ANIMALIA; EUKARYOTA;

EID: 0034495363     PISSN: 09596658     EISSN: None     Source Type: Journal    
DOI: 10.1093/glycob/10.12.1249     Document Type: Review

References (63)

1. Purification and characterization of 1-O-acylceramide synthase, a novel phospholipase A2 with transacylase activity
2. Improved inhibitors of glucosylceramide synthase
3. Structural and stereo-chemical studies of potent inhibitors of glucosylceramide synthase and tumor cell growth
4. Induction of glucosylceramide synthase by synthase inhibitors and ceramide
5. Glycosphingolipid depletion in fabry disease lymphoblasts with potent inhibitors of glucosylceramide synthase
6. Reduction of globotriaosylceramide in fabry disease mice by substrate deprivation
7. Gaucher disease
8. Functional breakdown of the lipid bilayer of the myelin membrane in central and peripheral nervous system by disrupted galactocerebroside synthesis
9. Myelination in the absence of galactocerebroside and sulfatide: Normal structure with abnormal function and regional instability
10. Biochemical basis of late-onset neurolipidoses
11. Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis
12. α-Galactosidase A deficiency: Fabry disease
13. Myelin galactolipids are essential for proper node of Ranvier formation in the CNS
14. 3
15. Inhibition of HIV replication by amino-sugar derivatives
16. Crucial role for the myelin-associated glycoprotein in the maintenance of axon-myelin integrity
17. The GM2 gangliosidoses
18. Bifunctional role of glycosphingolipids. Modulators for transmembrane signaling and mediators for cellular interactions
19. New insights in glycosphingolipid function: "Glycosignaling domain", a cell surface assembly of glycosphingolipids with signal transducer molecules, involved in cell adhesion coupled with signaling
20. Sphingolipid activator protein deficiency in a 16-week-old atypical Gaucher disease patient and his fetal sibling: Biochemical signs of combined sphingolipidoses
21. Physiology and pathophysiology of sphingolipid metabolism and signaling
22. Preparation of the active isomer of 1-phenyl-2-decanoylamino-3-morpholino-1-propanol, inhibitor of murine glucocerebroside synthetase
23. Reconstitution of membranes simulating "glycosignaling domain" and their susceptibility to lyso-GM3
24. Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin
25. Aminosugar derivatives as potential anti-human immunodeficiency virus agents
26. Association of Src family tyrosine kinase Lyn with ganglioside GD3 in rat brain. Possible regulation of Lyn by glycosphingolipid in caveolae- like domains
27. Improved inhibitors of glucosylceramide synthase
28. Quantitative correlation between the residual activity of beta-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease
29. A genetic model of substrate deprivation therapy for a glycosphingolipid storage disorder
30. Alleviation of neuronal ganglioside storage does not improve the clinical course of the Niemann-Pick C disease mouse
31. Alpha-Galactosidase A deficient mice: A model of Fabry disease
32. Aging accentuates and bone marrow transplantation ameliorates metabolic defects in Fabry disease mice
33. Niemann-Pick disease type C: A cellular cholesterol lipidosis
34. New therapeutic prospects for the glycosphingolipid lysosomal storage diseases
35. N-Butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis
36. N-butyldeoxygalactonojirimycin inhibits glycolipid biosynthesis but does not affect N-linked oligosaccharide processing
37. Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin
38. Extensive glycosphingolipid depletion in the liver and lymphoid organs of mice treated with N-butyldeoxynojirimycin
39. Gaucher disease: Enzyme therapy in the acute neuronopathic variant
40. Inhibitors and stimulators of glucocerebroside metabolism
41. Metabolic effects of inhibiting glucosylceramide synthesis with PDMP and other substances
42. Cell cycle arrest induced by an inhibitor of glucosylceramide synthase. Correlation with cyclin-dependent kinases
43. Sphingolipid activator proteins
44. Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism
45. Glycosphingolipids in cell surface recognition
46. The glycosphingolipidoses-from disease to basic principles of metabolism
47. Perspectives in basic science: Sphingolipids
48. Mice lacking complex gangliosides develop Wallerian degeneration and myelination defects
49. Growth of ectopic dendrites on cortical pyramidal neurons in neuronal storage diseases correlates with abnormal accumulation of GM2 ganglioside
50. Myelin glycolipids and their functions
51. Hydrops fetalis: Lysosomal storage disorders in extremis
52. β-Galactosidase deficiency (β-galactosidosis): GM1 gangliosidosis and Morquio B disease
53. Designation and shematic structure of gangliosides and allied glycosphingolipids
54. Mice with disrupted GM2/GD2 synthase gene lack complex gangliosides but exhibit only subtle defects in their nervous system
55. Complex gangliosides are essential in spermatogenesis of mice: Possible roles in the transport of testosterone
56. Recent advances in elucidating Niemann-Pick C disease
57. GM2 gangliosidase and pyramidal neuron dendritogenesis
58. GM2 gangliosidase as a regulator of pyramidal neuron dendritogenesis
59. Caloric intake and aging: Mechanisms in rodents and a study in nonhuman primates
60. Targeted disruption of the Hexa gene results in mice with biochemical and pathologic features of Tay-Sachs disease
61. A vital role for glycosphingolipid synthesis during development and differentiation
62. Myelin-associated glycoprotein is a myelin signal that modulates the caliber of myelinated axons
63. Attenuation of interleukin 2 signal in the spleen cells of complex gangliosidase-lacking mice