Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly functional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an attractive strategy to treat DMD. Here we show that loc...

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Detalles Bibliográficos
Autores principales: Le Guiner, Caroline, Servais, Laurent, Montus, Marie, Larcher, Thibaut, Fraysse, Bodvaël, Moullec, Sophie, Allais, Marine, François, Virginie, Dutilleul, Maeva, Malerba, Alberto, Koo, Taeyoung, Thibaut, Jean-Laurent, Matot, Béatrice, Devaux, Marie, Le Duff, Johanne, Deschamps, Jack-Yves, Barthelemy, Inès, Blot, Stéphane, Testault, Isabelle, Wahbi, Karim, Ederhy, Stéphane, Martin, Samia, Veron, Philippe, Georger, Christophe, Athanasopoulos, Takis, Masurier, Carole, Mingozzi, Federico, Carlier, Pierre, Gjata, Bernard, Hogrel, Jean-Yves, Adjali, Oumeya, Mavilio, Fulvio, Voit, Thomas, Moullier, Philippe, Dickson, George
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5537486/
https://www.ncbi.nlm.nih.gov/pubmed/28742067
http://dx.doi.org/10.1038/ncomms16105

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5537486/
https://www.ncbi.nlm.nih.gov/pubmed/28742067
http://dx.doi.org/10.1038/ncomms16105

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