Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy

Author:

Platt F. M.1,Jeyakumar M.1,Andersson U.1,Priestman D. A.1,Dwek R. A.1,Butters T. D.1,Cox T. M.2,Lachmann R. H.2,Hollak C.3,Aerts J. M. F. G.3,Van Weely S.3,Hrebícek M.4,Moyses C.5,Gow I.5,Elstein D.6,Zimran A.6

Affiliation:

1. ; Glycobiology Institute, Department of Biochemistry; University of Oxford; Oxford UK

2. ; Department of Medicine; University of Cambridge; Addenbrooke's Hospital Cambridge UK

3. ; Academic Medical Centre; University of Amsterdam; Amsterdam The Netherlands

4. Institute of Inherited Metabolic Disorders; Prague Czech Republic

5. Oxford GlycoSciences; Abingdon UK

6. ; Shaare Zedek Medical Center; Jerusalem Israel

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Reference46 articles.

1. Plasma and metabolic abnormalities in Gaucher's disease;Aerts;Baillières Clin Haematol,1997

2. N-butyldeoxygalactonojirimycin; a more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivo;Andersson;J Biochem Pharmacol,2000

3. Biology and potential strategies for the treatment of G(M2) gangliosides;Chavany;Mol Med Today,1990

4. Partial enzyme deficiencies: residual activities and the development of neurological disorders;Conzelmann;Dev Neurosci,1983

5. Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis;Cox;Lancet,2000

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