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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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Detalles Bibliográficos
Autores principales: Cotugno, Gabriella, Annunziata, Patrizia, Barone, Maria Vittoria, Karali, Marianthi, Banfi, Sandro, Auricchio, Alberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2012
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3302848/
https://www.ncbi.nlm.nih.gov/pubmed/22428010
http://dx.doi.org/10.1371/journal.pone.0033286