Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA

Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA

Preclinical gene therapy strategies using recombinant adeno-associated virus (AAV) vectors in animal models of Duchenne muscular dystrophy have shown dramatic phenotype improvements, but long-lasting efficacy remains questionable. It is believed that in dystrophic muscles, transgene persistence is h...

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Bibliographic Details
Main Authors: Jean-Baptiste Dupont, Benoit Tournaire, Christophe Georger, Béatrice Marolleau, Laurence Jeanson-Leh, Mireille Ledevin, Pierre Lindenbaum, Emilie Lecomte, Benjamin Cogné, Laurence Dubreil, Thibaut Larcher, Bernard Gjata, Laetitia Van Wittenberghe, Caroline Le Guiner, Magalie Penaud-Budloo, Richard O Snyder, Philippe Moullier, Adrien Léger
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050116300225

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http://www.sciencedirect.com/science/article/pii/S2329050116300225

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