Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

Antisense oligonucleotide-mediated exon skipping is able to correct out-of-frame mutations in Duchenne muscular dystrophy and restore truncated yet functional dystrophins. However, its application is limited by low potency and inefficiency in systemic delivery, especially failure to restore dystroph...

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Detalhes bibliográficos
Main Authors: Wu, Bo, Moulton, Hong M., Iversen, Patrick L., Jiang, Jiangang, Li, Juan, Li, Jianbin, Spurney, Christopher F., Sali, Arpana, Guerron, Alfredo D., Nagaraju, Kanneboyina, Doran, Timothy, Lu, Peijuan, Xiao, Xiao, Lu, Qi Long
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2008
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2546441/
https://ncbi.nlm.nih.gov/pubmed/18806224
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0805676105
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