Article
Adenovirus-mediated gene therapy in a mouse model of glycogen storage disease type 1a.
European journal of pediatrics - 1 Oct 2002
Chou Janice Yang, Zingone Adriana, Pan Chi-Jiunn
Abstract excerpt
UNLABELLED: Glycogen storage disease type 1a (GSD-1a), characterized by growth retardation, hypoglycemia, hepatomegaly, kidney enlargement, hyperlipidemia, hyperuricemia, and renal dysfunction, is caused by deficiencies in glucose-6-phosphatase (G6Pase), a key enzyme in glucose homeostasis. Over the last 20 years, dietary therapies have greatly improved the prognosis of GSD-1a patients. However, the underlying...
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