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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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Detalles Bibliográficos
Autores principales: Wu, Bo, Benrashid, Ehsan, Lu, Peijuan, Cloer, Caryn, Zillmer, Allen, Shaban, Mona, Lu, Qi Long
Formato: Texto
Lenguaje:English
Publicado: Public Library of Science 2011
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3096650/
https://www.ncbi.nlm.nih.gov/pubmed/21611204
http://dx.doi.org/10.1371/journal.pone.0019906