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Targeted Skipping of Human Dystrophin Exons in Transgenic Mouse Model Systemically for Antisense Drug Development

Antisense therapy has recently been demonstrated with great potential for targeted exon skipping and restoration of dystrophin production in cultured muscle cells and in muscles of Duchenne Muscular Dystrophy (DMD) patients. Therapeutic values of exon skipping critically depend on efficacy of the dr...

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Detalhes bibliográficos
Main Authors: Wu, Bo, Benrashid, Ehsan, Lu, Peijuan, Cloer, Caryn, Zillmer, Allen, Shaban, Mona, Lu, Qi Long
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2011
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3096650/
https://ncbi.nlm.nih.gov/pubmed/21611204
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0019906
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