Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler-Najjar Syndrome Type I. [electronic resource]
Producer: 20180326Description: 649-660 p. digitalISSN:- 1476-5462
- Animals
- Bilirubin -- metabolism
- Brain -- metabolism
- Crigler-Najjar Syndrome -- therapy
- Dependovirus -- genetics
- Female
- Gene Transfer Techniques
- Genetic Therapy -- methods
- Genetic Vectors -- genetics
- Glucuronosyltransferase -- genetics
- HEK293 Cells
- Humans
- Liver -- metabolism
- Male
- Mice
- Mice, Inbred C57BL
- Serogroup
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Publication Type: Journal Article; Research Support, Non-U.S. Gov't
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