Morpholino-induced exon skipping stimulates cell-mediated and humoral responses to dystrophin in mdx mice

Exon skipping is a promising genetic therapeutic strategy for restoring dystrophin expression in the treatment of Duchenne muscular dystrophy (DMD). The potential for newly synthesized dystrophin to trigger an immune response in DMD patients, however, is not well established. We have evaluated the e...

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Detalhes bibliográficos
Publicado no:J Pathol
Main Authors: Vila, Maria Candida, Novak, James S., Klimek, Margaret Benny, Li, Ning, Morales, Melissa, Fritz, Alexander G., Edwards, Katie, Boehler, Jessica F., Hogarth, Marshall W., Kinder, Travis B., Zhang, Aiping, Mazala, Davi, Fiorillo, Alyson A., Douglas, Bonnie, Chen, Yi-Wen, van den Anker, John, Lu, Qi Long, Hathout, Yetrib, Hoffman, Eric P., Partridge, Terence A., Nagaraju, Kanneboyina
Formato: Artigo
Idioma:Inglês
Publicado em: 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6579705/
https://ncbi.nlm.nih.gov/pubmed/30883742
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/path.5263
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