Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease. [electronic resource]
Producer: 20200311Description: 57-68 p. digitalISSN:- 1557-7422
- Animals
- Dependovirus -- genetics
- Disease Models, Animal
- Enzyme Activation
- Gene Expression
- Gene Transfer Techniques
- Genetic Therapy -- methods
- Genetic Vectors -- administration & dosage
- Glycogen -- metabolism
- Glycogen Storage Disease Type II -- genetics
- Humans
- Immunohistochemistry
- Mice
- Mice, Transgenic
- Muscle, Skeletal -- metabolism
- Prognosis
- Treatment Outcome
- alpha-Glucosidases -- genetics
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Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
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