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Electroporation Enhanced Effect of Dystrophin Splice Switching PNA Oligomers in Normal and Dystrophic Muscle

Peptide nucleic acid (PNA) is a synthetic DNA mimic that has shown potential for discovery of novel splice switching antisense drugs. However, in vivo cellular delivery has been a limiting factor for development, and only few successful studies have been reported. As a possible modality for improvem...

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Principais autores: Camilla Brolin, Takehiko Shiraishi, Pernille Hojman, Thomas O Krag, Peter E Nielsen, Julie Gehl
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2015-01-01
Serier:Molecular Therapy: Nucleic Acids
Fag:
antisense
electroporation
exon skipping
mdx
PNA
splicing modulation
Online adgang:http://www.sciencedirect.com/science/article/pii/S2162253116300531
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