Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells

Cell-penetrating peptide-mediated delivery of phosphorodiamidate morpholino oligomers (PMOs) has shown great promise for exon-skipping therapy of Duchenne Muscular Dystrophy (DMD). Pip6a-PMO, a recently developed conjugate, is particularly efficient in a murine DMD model, although mechanisms respons...

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Detalhes bibliográficos
Main Authors: Lehto, Taavi, Castillo Alvarez, Alejandra, Gauck, Sarah, Gait, Michael J., Coursindel, Thibault, Wood, Matthew J. A., Lebleu, Bernard, Boisguerin, Prisca
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2014
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3950666/
https://ncbi.nlm.nih.gov/pubmed/24366877
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkt1220
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