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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Detalles Bibliográficos
Autores principales: Dupont, Jean-Baptiste, Tournaire, Benoit, Georger, Christophe, Marolleau, Béatrice, Jeanson-Leh, Laurence, Ledevin, Mireille, Lindenbaum, Pierre, Lecomte, Emilie, Cogné, Benjamin, Dubreil, Laurence, Larcher, Thibaut, Gjata, Bernard, Van Wittenberghe, Laetitia, Le Guiner, Caroline, Penaud-Budloo, Magalie, Snyder, Richard O, Moullier, Philippe, Léger, Adrien
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2015
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4445007/
https://www.ncbi.nlm.nih.gov/pubmed/26029721
http://dx.doi.org/10.1038/mtm.2015.10

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4445007/
https://www.ncbi.nlm.nih.gov/pubmed/26029721
http://dx.doi.org/10.1038/mtm.2015.10

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