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    Adenovirus-mediated gene transfer results in decreased lysosomal storage in brain and total correction in liver of aspartylglucosaminuria (AGU) mouse

    Aspartylglucosaminuria (AGU) is a lysosomal storage disease leading to mental retardation, which is caused by deficiency of aspartylglucosaminidase (AGA). AGU is strongly enriched in the Finnish population in ...

    M Peltola, A Kyttälä, O Heinonen, J Rapola, T Paunio, F Revah, L Peltonen… in Gene Therapy (1998)