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Long-term Exon Skipping Studies With 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models

  • Published:2012 Issue: Volume:1 Page:e44
  • ISSN:2162-2531
  • Container-title:Molecular Therapy - Nucleic Acids
  • language:en
  • Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Tanganyika-de Winter Christa L,Heemskerk Hans,Karnaoukh Tatyana G,van Putten Maaike,de Kimpe Sjef J,van Deutekom Judith,Aartsma-Rus Annemieke

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference38 articles.

1. Progress in therapeutic antisense applications for neuromuscular disorders;Aartsma-Rus;Eur J Hum Genet,2010

2. The therapeutic potential of antisense-mediated exon skipping;van Ommen;Curr Opin Mol Ther,2008

3. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus;Monaco;Genomics,1988

4. The muscular dystrophies;Emery;Lancet,2002

5. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations;Aartsma-Rus;Hum Mutat,2009

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