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Exons 45–55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene
Mutations in the dystrophin (DMD) gene and consequent loss of dystrophin cause Duchenne muscular dystrophy (DMD). A promising therapy for DMD, single-exon skipping using antisense phosphorodiamidate morpholino oligomers (PMOs), currently confronts major issues in that an antisense drug induces the p...
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| Udgivet i: | Mol Ther |
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| Main Authors: | , , , , , , , , , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
American Society of Gene & Cell Therapy
2019
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6838919/ https://ncbi.nlm.nih.gov/pubmed/31416775 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.07.012 |
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