Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease. [electronic resource]
Producer: 20180507Description: 66 p. digitalISSN:- 2051-5960
- Animals
- Brain -- metabolism
- Cardiomyopathy, Hypertrophic -- pathology
- Dependovirus -- genetics
- Disease Models, Animal
- Genetic Therapy
- Genetic Vectors
- Glycogen -- metabolism
- Glycogen Storage Disease Type II -- pathology
- HEK293 Cells
- Humans
- Injections, Spinal
- Male
- Muscle Strength -- physiology
- Random Allocation
- Single-Blind Method
- Spinal Cord -- metabolism
- alpha-Glucosidases -- genetics
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Publication Type: Journal Article; Observational Study; Research Support, Non-U.S. Gov't
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