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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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Bibliografiske detaljer
Udgivet i:PLoS Genet
Main Authors: Banks, Glen B., Chamberlain, Jeffrey S., Odom, Guy L.
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2020
Fag:
Online adgang: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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