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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...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Public Library of Science
2020
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| 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 |