Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effect...
Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects
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United States: Public Library of Science
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Language
English
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United States: Public Library of Science
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Contents
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. Utrophin, a developmental paralogue of dystrophin, may...
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Full title
Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects
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TN_cdi_plos_journals_2479450410
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_2479450410
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ISSN
1553-7404,1553-7390
E-ISSN
1553-7404
DOI
10.1371/journal.pgen.1009179
