Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy. [electronic resource]
Producer: 20101012Description: 35ra42 p. digitalISSN:- 1946-6242
- Animals
- Animals, Newborn
- Base Sequence
- Codon -- genetics
- Dependovirus -- genetics
- Disease Models, Animal
- Fibroblasts -- metabolism
- Gene Transfer Techniques
- Genetic Therapy
- Mice
- Molecular Sequence Data
- Motor Activity -- physiology
- Muscular Atrophy, Spinal -- genetics
- Mutation -- genetics
- Phenotype
- Survival Analysis
- Survival of Motor Neuron 1 Protein -- genetics
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Publication Type: Journal Article
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