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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Bibliografske podrobnosti
izdano v: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
Format: Artigo
Jezik:Inglês
Izdano: 2019
Teme:
Article
Online dostop: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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