Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models
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...
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| Main Authors: | , , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2012-01-01T00:00:00Z.
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| Online Access: | Connect to this object online. |
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