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Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD
Duchenne muscular dystrophy is a fatal muscle disease, caused by mutations in DMD, leading to loss of dystrophin expression. Phosphorodiamidate morpholino splice-switching oligonucleotides (PMO-SSOs) have been used to elicit the restoration of a partially functional truncated dystrophin by excluding...
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| Main Authors: | , , , , , , |
|---|---|
| Formáid: | Artigo |
| Teanga: | Inglês |
| Foilsithe: |
Elsevier
2018-12-01
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| Sraith: | Molecular Therapy: Nucleic Acids |
| Rochtain Ar Líne: | http://www.sciencedirect.com/science/article/pii/S2162253118302737 |
| Clibeanna: |
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