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Adenovirus-mediated gene therapy in a mouse model of glycogen storage disease type 1a

✍ Scribed by Janice Chou; Adriana Zingone; Chi-Jiunn Pan


Publisher
Springer
Year
2002
Tongue
English
Weight
259 KB
Volume
161
Category
Article
ISSN
0340-6997

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## Abstract ## Background Aspartylglucosaminuria (AGU) is a lysosomal storage disease with severe neurodegenerative clinical features resulting from the deficiency of lysosomal aspartylglucosaminidase (AGA). The AGU knockout mouse is a good model to test different therapy strategies, as it mimics