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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5643396/
https://www.ncbi.nlm.nih.gov/pubmed/29038471
http://dx.doi.org/10.1038/s41467-017-00924-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5643396/
https://www.ncbi.nlm.nih.gov/pubmed/29038471
http://dx.doi.org/10.1038/s41467-017-00924-7

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