AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice. [electronic resource]
Producer: 20150427Description: 1553-61 p. digitalISSN:- 1528-0020
- Amino Acid Substitution
- Animals
- Capsid -- immunology
- Cytokines -- blood
- Dependovirus -- genetics
- Disease Models, Animal
- Dogs
- Factor IX -- antagonists & inhibitors
- Gene Expression
- Genetic Therapy -- methods
- Genetic Vectors -- adverse effects
- Hemophilia B -- genetics
- Humans
- Male
- Mice
- Mice, Inbred C57BL
- Mice, Mutant Strains
- Mutant Proteins -- genetics
- Recombinant Proteins -- genetics
- Thrombosis -- prevention & control
- Translational Research, Biomedical
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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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