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AAV-Vectors for Treatment of Glycogen Storage Disorders

20:00 EDT 5 Aug 2014 | NIH

Adeno-Associated Virus Vectors for the treatment of glycogen storage disease, particularly glycogen storage disease type Ia, are disclosed. Glycogen storage disease type Ia (GSD-Ia or von Gierke disease) is caused by a deficiency in glucose-6-phosphatase-alpha (G6Pase-alpha or G6PC). Patients affected by GSD-Ia are unable to maintain glucose homeostasis and present with fasting hypoglycemia, growth retardation, hepatomegaly, nephromegaly, hyperlipidemia, hyperuricemia, and lactic academia. There is currently no cure for GSD-Ia deficiency disorder.

Original Article: AAV-Vectors for Treatment of Glycogen Storage Disorders

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