Prevention of Lysosomal Storage in Tay-Sachs Mice Treated with N -Butyldeoxynojirimycin-Reference-Cited by-同舟云学术

Prevention of Lysosomal Storage in Tay-Sachs Mice Treated with N -Butyldeoxynojirimycin

Published:1997-04-18 Issue:5311 Volume:276 Page:428-431
ISSN:0036-8075
Container-title:Science
language:en
Short-container-title:Science

Author:

Platt Frances M.¹²³⁴⁵,Neises Gabrielle R.¹²³⁴⁵,Reinkensmeier Gabriele¹²³⁴⁵,Townsend Mandy J.¹²³⁴⁵,Perry V. Hugh¹²³⁴⁵,Proia Richard L.¹²³⁴⁵,Winchester Bryan¹²³⁴⁵,Dwek Raymond A.¹²³⁴⁵,Butters Terry D.¹²³⁴⁵

Affiliation:

1. F. M. Platt, G. Reinkensmeier, R. A. Dwek, T. D. Butters, Glycobiology Institute, Department of Biochemistry, University of Oxford, South Parks Road, Oxford 0X1 3QU, UK.

2. G. R. Neises, Monsanto Company, 700 Chesterfield Village Parkway, St. Louis, MO 63198, USA.

3. M. J. Townsend and V. H. Perry, Department of Pharmacology, University of Oxford, Mansfield Road, Oxford OX1 3QT, UK.

4. R. L. Proia, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD 20892, USA.

5. B. Winchester, Institute of Child Health, 30 Guilford Street, London WC1N 1EH, UK.

Abstract

The glycosphingolipid (GSL) lysosomal storage diseases result from the inheritance of defects in the genes encoding the enzymes required for catabolism of GSLs within lysosomes. A strategy for the treatment of these diseases, based on an inhibitor of GSL biosynthesis N -butyldeoxynojirimycin, was evaluated in a mouse model of Tay-Sachs disease. When Tay-Sachs mice were treated with N -butyldeoxynojirimycin, the accumulation of G M2 in the brain was prevented, with the number of storage neurons and the quantity of ganglioside stored per cell markedly reduced. Thus, limiting the biosynthesis of the substrate (G M2 ) for the defective enzyme (β-hexosaminidase A) prevents GSL accumulation and the neuropathology associated with its lysosomal storage.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

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