Inhibition of glycosphingolipid biosynthesis: Application to lysosomal storage disorders

被引:288
作者
Butters, TD [1 ]
Dwek, RA [1 ]
Platt, FM [1 ]
机构
[1] Univ Oxford, Dept Biochem, Oxford Glycobiol Inst, Oxford OX1 3QU, England
关键词
D O I
10.1021/cr990292q
中图分类号
O6 [化学];
学科分类号
0703 ;
摘要
A review is given on some of the more recent knowledge of the enzymes involved in the control of glycosphingolipids biosynthesis and how catabolic deficiency leads to the glycosphingolipidoses. Focus is on how small-molecule inhibitors have been instrumental in probing the enzymes in these pathways and how their application has been taken from discovery in the laboratory to therapeutic use in man.
引用
收藏
页码:4683 / +
页数:15
相关论文
共 97 条
  • [1] Reduction of globotriaosylceramide in Fabry disease mice by substrate deprivation
    Abe, A
    Gregory, S
    Lee, L
    Killen, PD
    Brady, RO
    Kulkarni, A
    Shayman, JA
    [J]. JOURNAL OF CLINICAL INVESTIGATION, 2000, 105 (11) : 1563 - 1571
  • [2] Glycosphingolipid depletion in Fabry disease lymphoblasts with potent inhibitors of glucosylceramide synthase
    Abe, A
    Arend, LJ
    Lee, L
    Lingwood, C
    Brady, RO
    Shayman, JA
    [J]. KIDNEY INTERNATIONAL, 2000, 57 (02) : 446 - 454
  • [3] N-butyldeoxygalactonojirimycin:: A more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivo
    Andersson, U
    Butters, TD
    Dwek, RA
    Platt, FM
    [J]. BIOCHEMICAL PHARMACOLOGY, 2000, 59 (07) : 821 - 829
  • [4] Expression cloning of a cDNA encoding a sulfotransferase involved in the biosynthesis of the HNK-1 carbohydrate epitope
    Bakker, H
    Friedmann, I
    Oka, S
    Kawasaki, T
    Nifantev, N
    Schachner, M
    Mantei, N
    [J]. JOURNAL OF BIOLOGICAL CHEMISTRY, 1997, 272 (47) : 29942 - 29946
  • [5] Gene transfer approaches to the lysosomal storage disorders
    Barranger, JA
    Rice, EO
    Swaney, WP
    [J]. NEUROCHEMICAL RESEARCH, 1999, 24 (04) : 601 - 615
  • [6] Gaucher's disease: studies of gene transfer to haematopoietic cells
    Barranger, JA
    Rice, EO
    Dunigan, J
    Sansieri, C
    Takiyama, N
    Beeler, M
    Lancia, J
    Lucot, S
    Scheirer-Fochler, S
    Mohney, T
    Swaney, W
    Bahnson, A
    Ball, E
    [J]. BAILLIERES CLINICAL HAEMATOLOGY, 1997, 10 (04): : 765 - 778
  • [7] The Protein Data Bank
    Berman, HM
    Westbrook, J
    Feng, Z
    Gilliland, G
    Bhat, TN
    Weissig, H
    Shindyalov, IN
    Bourne, PE
    [J]. NUCLEIC ACIDS RESEARCH, 2000, 28 (01) : 235 - 242
  • [8] GAUCHER DISEASE AS A PARADIGM OF CURRENT ISSUES REGARDING SINGLE-GENE MUTATIONS OF HUMANS
    BEUTLER, E
    [J]. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1993, 90 (12) : 5384 - 5390
  • [9] Hematologically important mutations: Gaucher disease
    Beutler, E
    Gelbart, T
    [J]. BLOOD CELLS MOLECULES AND DISEASES, 1997, 23 (01) : 2 - 7
  • [10] BIA J, 1997, BIOCHEMISTRY-US, V36, P8840