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Article

Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation

10 Nov 2020

Abstract excerpt

Aspartylglucosaminuria (AGU) is an autosomal recessive lysosomal storage disease caused by loss of the enzyme aspartylglucosaminidase (AGA), resulting in AGA substrate accumulation. AGU patients have a slow but progressive neurodegenerative disease course, for which there is no approved disease-modifying treatment. In this study, AAV9/ AGA was administered to Aga −/− mice intravenously (i.v.) or intrathecally...

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PMID: 33186692
DOI: 10.1016/j.ymthe.2020.11.012

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