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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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Bibliografische gegevens
Gepubliceerd in:PLoS Genet
Hoofdauteurs: Banks, Glen B., Chamberlain, Jeffrey S., Odom, Guy L.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Public Library of Science 2020
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Online toegang: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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