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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 DisordersNEXT ARTICLE
According to the National Arthritis Data Workgroup, an estimated 6 million people in the United States report having experienced gout at some point in their lives. In fact, gout is the most common form of inflammatory arthritis in men over the age of 40....