Restricted ketogenic diet enhances the therapeutic action of N-butyldeoxynojirimycin towards brain GM2 accumulation in adult Sandhoff disease mice.

Journal article

Sandhoff disease is an autosomal recessive, neurodegenerative disease involving the storage of brain ganglioside GM2 and asialo-GM2. Previous studies showed that caloric restriction, which augments longevity, and N-butyldeoxynojirimycin (NB-DNJ, Miglustat), an imino sugar that hinders the glucosyltransferase catalyzing the first step in glycosphingolipid biosynthesis, both increase longevity and improve motor behavior in the beta-hexosaminidase (Hexb) knockout (-/-) murine model of Sandhoff d...

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Authors: Denny, C; Heinecke, K; Kim, Y; Baek, R; Loh, K

• Publication status: Published
• Journal: Journal of neurochemistry
• Volume: 113
• Issue: 6
• Pages: 1525-1535
• Publication date: 2010-06-01
• DOI: 10.1111/j.1471-4159.2010.06733.x
• EISSN: 1471-4159
• ISSN: 0022-3042
• Language: English
• Keywords: Eating; beta-N-Acetylhexosaminidases; G(M2) Ganglioside; Chromatography, Thin Layer; Mice; Sandhoff Disease; Animals; Mice, Knockout; Brain; Analysis of Variance; Chromatography, High Pressure Liquid; Body Weight; 3-Hydroxybutyric Acid; 1-Deoxynojirimycin; Myelin Sheath; Ketogenic Diet; Purkinje Cells; Blood Glucose; Lipid Metabolism