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Gene Therapy for Muscular Dystrophy: Lessons Learned and Path Forward
Our Translational Gene Therapy Center has used small molecules for exon skipping and mutation suppression and gene transfer to replace or provide surrogate genes as tools for molecular-based approaches for the treatment of muscular dystrophies. Exon skipping is targeted at the pre-mRNA level allowin...
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| Hauptverfasser: | , , , , , , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
2012
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3492936/ https://ncbi.nlm.nih.gov/pubmed/22609847 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neulet.2012.04.078 |
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