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Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects

Gene therapy approaches for DMD using recombinant adeno-associated viral (rAAV) vectors to deliver miniaturized (or micro) dystrophin genes to striated muscles have shown significant progress. However, concerns remain about the potential for immune responses against dystrophin in some patients. Utro...

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Detalles Bibliográficos
Autores principales: Banks, Glen B., Chamberlain, Jeffrey S., Odom, Guy L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7682874/
https://www.ncbi.nlm.nih.gov/pubmed/33175853
http://dx.doi.org/10.1371/journal.pgen.1009179