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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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Bibliographic Details
Main Authors: Wu, Bo, Benrashid, Ehsan, Lu, Peijuan, Cloer, Caryn, Zillmer, Allen, Shaban, Mona, Lu, Qi Long
Format: Artigo
Language:Inglês
Published: Public Library of Science 2011
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Online Access: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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