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Optimized Gene Therapy Vector for the Treatment of Glycogen Storage Disease Type Ia

20:00 EDT 30 Sep 2015 | NIH

NIH researchers have developed an adeno-associated viral (AAV) vector for the treatment of glycogen storage disease type Ia (GSD-Ia). GSD-Ia is an inherited disorder of metabolism associated with life-threatening hypoglycemia, hepatic malignancy, and renal failure caused by the deficiency of glucose-6-phosphatase-alpha (G6Pase-alpha or G6PC).

Original Article: Optimized Gene Therapy Vector for the Treatment of Glycogen Storage Disease Type Ia

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