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Oxford University Press

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Molecular pathophysiology in Tay–Sachs and Sandhoff diseases as revealed by gene expression profiling

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Sialidase-mediated depletion of G M2 ganglioside in Tay–Sachs neuroglia cells

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Human Molecular Genetics, Vol. 8, Iss. 6, 1999-06 ,pp. : 1111-1116

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Adenoviral gene therapy of the Tay-Sachs disease in hexosaminidase A-deficient knock-out mice

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A direct gene transfer strategy via brain internal capsule reverses the biochemical defect in Tay–Sachs disease

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