Adenovirus Vector
Gene therapy increases survival in glycogen storage disease type Ia
Published in Vaccine Weekly, October 25th, 2006
According to recent research from the United States, "The deficiency of glucose-6-phosphatase (G6Pase) underlies life-threatening hypoglycemia and growth retardation in glycogen storage disease type Ia (GSD-Ia)."
"An adeno-associated virus (AAV) vector encoding G6Pase was pseudotyped as AAV8 and administered to 2-week-old GSD-Ia mice (n=9)," wrote D.D. Koeberl and colleagues at Duke University in Durham. "Median survival was prolonged to 7 months following vector administration, in contrast to untreated GSD-Ia mice that survived for only 2...
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Source: Vaccine Weekly (2006-10-25)
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