Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle

Exon skipping is a promising therapeutic strategy for Duchenne muscular dystrophy (DMD), employing morpholino antisense oligonucleotides (PMO-AO) to exclude disruptive exons from the mutant DMD transcript and elicit production of truncated dystrophin protein. Clinical trials for PMO show variable an...

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Publicado no:Nat Commun
Main Authors: Novak, James S., Hogarth, Marshall W., Boehler, Jessica F., Nearing, Marie, Vila, Maria C., Heredia, Raul, Fiorillo, Alyson A., Zhang, Aiping, Hathout, Yetrib, Hoffman, Eric P., Jaiswal, Jyoti K., Nagaraju, Kanneboyina, Cirak, Sebahattin, Partridge, Terence A.
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2017
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5643396/
https://ncbi.nlm.nih.gov/pubmed/29038471
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-017-00924-7
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