Published in
Public Library of Science, PLoS ONE, 3(7), p. e33286, 2012
DOI: 10.1371/journal.pone.0033286
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Impact of Age at Administration, Lysosomal Storage, and Transgene Regulatory Elements on AAV2/8-Mediated Rat Liver Transduction
Journal article published in 2012 by
Gabriella Cotugno,
Patrizia Annunziata,
Maria Vittoria Barone 
,
Marianthi Karali,
Sandro Banfi ,
Alberto Auricchio
,
Marianthi Karali,
Sandro Banfi ,
Alberto Auricchio
This paper is made freely available by the publisher.
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Abstract
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 models and in patients.