Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

Author:

Mah C,Cresawn K O,Fraites T J,Pacak C A,Lewis M A,Zolotukhin I,Byrne B J

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference36 articles.

1. Baudhin P, Hers HG . An electron microscopic and biochemical study of type II glycogenosis. Lab Invest 1964; 13: 1139–1152.

2. Hirschhorn R, Reuser AJJ . Glycogen storage disease II: acid-alpha glucosidase (acid maltase) deficiency. In: Scriver et al (eds). The Metabolic and Molecular Bases of Inherited Diseases. McGraw Hill: New York, 2000, pp 3389–3420.

3. Hers HG . Alpha-glucosidase deficiency in generalized glycogen storage disease (Pompe's disease). Biochem J 1963; 86: 11–16.

4. Raben N, Plotz P, Byrne BJ . Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease). Curr Mol Med 2002; 2: 145–166.

5. Reuser AJ et al. Glycogenosis type II (acid maltase deficiency). Muscle Nerve 1995; 3: S61–S69.

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