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Gene Therapy 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). NICHD seeks parties to license this invention towards commercialization.

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Gene Therapy Vector for the Treatment of Glycogen Storage Disease Type Ia (GSD-Ia)