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Octa-guanidine Morpholino Restores Dystrophin Expression in Cardiac and Skeletal Muscles and Ameliorates Pathology in Dystrophic mdx Mice

Steric-block antisense oligonucleotides (AONs) are able to target RNAs for destruction and splicing alteration. Reading frame restoration of the dystrophin transcript can be achieved by AON-mediated exon skipping in the dystrophic mdx mouse model. However, simple, unmodified AONs exhibit inefficient...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Wu, Bo, Li, Yongfu, Morcos, Paul A, Doran, Timothy J, Lu, Peijuan, Lu, Qi Long
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group 2009
Pynciau:
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835139/
https://ncbi.nlm.nih.gov/pubmed/19277018
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.38
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