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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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Detalhes bibliográficos
Publicado no:PLoS Genet
Main Authors: Banks, Glen B., Chamberlain, Jeffrey S., Odom, Guy L.
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7682874/
https://ncbi.nlm.nih.gov/pubmed/33175853
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pgen.1009179
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