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Adenovirus Vector
Gene therapy increases survival in glycogen storage disease type Ia
October 25th, 2006
Adeno-associated virus vector-mediated gene therapy increases survival in glycogen storage disease type Ia. 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...
Source: Biotech Week (2006-10-25)
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