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

• Main Authors: Christa L Tanganyika-de Winter (Author), Hans Heemskerk (Author), Tatyana G Karnaoukh (Author), Maaike van Putten (Author), Sjef J de Kimpe (Author), Judith van Deutekom (Author), Annemieke Aartsma-Rus (Author)
• Format: Book
• Published: Elsevier, 2012-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Antisense-mediated exon skipping for Duchenne muscular dystrophy (DMD) is currently tested in phase 3 clinical trials. The aim of this approach is to modulate splicing by skipping a specific exon to reframe disrupted dystrophin transcripts, allowing the synthesis of a partly functional dystrophin pr...