Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. [electronic resource]
Producer: 20120710Description: 443-55 p. digitalISSN:- 1525-0024
- Amino Acid Sequence
- Animals
- Antibodies, Neutralizing -- immunology
- Antibodies, Viral -- immunology
- Capsid Proteins -- chemistry
- Cell Line
- Child
- Child, Preschool
- Dependovirus -- genetics
- Dystrophin -- genetics
- Genetic Therapy
- Genetic Vectors -- administration & dosage
- Humans
- Male
- Mice
- Mice, Inbred BALB C
- Mice, Inbred C57BL
- Models, Molecular
- Molecular Sequence Data
- Muscle, Skeletal -- metabolism
- Muscular Dystrophy, Duchenne -- genetics
- Protein Conformation
- Sequence Alignment
- T-Lymphocytes -- immunology
- Transduction, Genetic
- Viral Tropism
No physical items for this record
Publication Type: Clinical Trial, Phase I; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
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