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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Bibliographische Detailangaben
Hauptverfasser: Wu, Bo, Benrashid, Ehsan, Lu, Peijuan, Cloer, Caryn, Zillmer, Allen, Shaban, Mona, Lu, Qi Long
Format: Artigo
Sprache:Inglês
Veröffentlicht: Public Library of Science 2011
Schlagworte:
Research Article
Online Zugang: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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