Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction.

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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Bibliographic Details
Main Authors: Gabriella Cotugno, Patrizia Annunziata, Maria Vittoria Barone, Marianthi Karali, Sandro Banfi, Alberto Auricchio
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2012-01-01
Series:PLoS ONE
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/pmid/22428010/pdf/?tool=EBI

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https://www.ncbi.nlm.nih.gov/pmc/articles/pmid/22428010/pdf/?tool=EBI

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