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    Widespread distribution of β-hexosaminidase activity in the brain of a Sandhoff mouse model after coinjection of adenoviral vector and mannitol

    Sandhoff disease is a severe inherited neurodegenerative disorder resulting from deficiency of the β-subunit of hexosaminidases A and B, lysosomal hydrolases involved in the degradation of GM2 ganglioside and rel...

    C Bourgoin, C Emiliani, E J Kremer, A Gelot, B Tancini, R A Gravel… in Gene Therapy (2003)