Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. [electronic resource]
Producer: 20130627Description: 3335-44 p. digitalISSN:- 1528-0020
- Animals
- Blotting, Western
- Dependovirus -- genetics
- Factor VIII -- genetics
- Genetic Therapy
- Genetic Variation -- genetics
- Genetic Vectors -- administration & dosage
- Glycosylation
- Hemophilia A -- genetics
- Humans
- Immune Tolerance
- Liver -- metabolism
- Macaca mulatta
- Male
- Mice
- Mice, Inbred C57BL
- Mice, Knockout
- Peptide Fragments -- genetics
- Promoter Regions, Genetic -- 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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