J. Lipid Res.
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J. Lipid Res., doi:10.1194/jlr.M400411-JLR200
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Submitted on October 19, 2004
Revised on January 5, 2005
Accepted on January 19, 2005

Substrate reduction therapy reduces ganglioside content in postnatal cerebrum-brainstem and cerebellum in a mouse model of GM1 gangliosidosis

Julie L. Kasperzyk, Alessandra d'Azzo, Frances M. Platt, Joseph Alroy, and Thomas N. Seyfried

Department of Biology, Boston College, Chestnut Hill, MA 02467

Corresponding Author: thomas.seyfried{at}bc.edu

GM1 gangliosidosis is an incurable lysosomal storage disease caused by a deficiency in acid beta -galactosidase (beta -gal), resulting in accumulation of ganglioside GM1 and its asialo-form (GA1) primarily in the brain. In this study, we investigated the effects of N-butyldeoxygalacto- nojirimycin (NB-DGJ), an imino sugar that inhibits ganglioside biosynthesis, in normal C57BL/6J (B6) and in beta -gal knockout (-/-) mice from p-9 to p-15. This is a period of active cerebellar development and CNS myelinogenesis in the mouse and would be comparable to late stage embryonic and early neonatal development in humans. NB-DGJ significantly reduced total ganglioside and GM1 content in cerebrum-brainstem (C-BS) and in cerebellum of normal and beta -gal -/- mice. NB-DGJ had no adverse effects on body weight or C-BS/cerebellar weight, water content, or thickness of the external cerebellar granule cell layer. Sphingomyelin was elevated in C-BS and cerebellum, but no changes were found for cerebroside (a myelin enriched glycosphingolipid), neutral phospholipids, or GA1 in the treated mice. Our findings indicate that the effects of NB-DGJ in the CNS are largely specific to gangliosides and suggest NB-DGJ may be an effective early intervention therapy for GM1 gangliosidosis and other ganglioside storage disorders.


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