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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Pubblicato in:J Pathol
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2019
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Article
Accesso online: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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