Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction.
Liver-directed gene transfer is being investigated for the treatment of systemic or liver-specific diseases. Recombinant vectors based on adeno-associated virus serotype 8 (AAV2/8) efficiently transduce liver cells allowing long term transgene expression after a single administration in animal model...
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Auteurs principaux: | , , , , , |
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Format: | article |
Langue: | EN |
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Public Library of Science (PLoS)
2012
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Accès en ligne: | https://doaj.org/article/a03f2ebf161546d1b2099161ca58d6a7 |
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