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Long-Term Morpholino Oligomers in Hexose Elicits Long-Lasting Therapeutic Improvements in mdx Mice
Approval of antisense oligonucleotide eteplirsen highlights the promise of exon-skipping therapeutics for Duchenne muscular dystrophy patients. However, the limited efficacy of eteplirsen underscores the importance to improve systemic delivery and efficacy. Recently, we demonstrated that a glucose a...
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| Udgivet i: | Mol Ther Nucleic Acids |
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| Main Authors: | , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
American Society of Gene & Cell Therapy
2018
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6070676/ https://ncbi.nlm.nih.gov/pubmed/30195785 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.06.005 |
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