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Development of Therapeutic Splice-Switching Oligonucleotides
Synthetic splice-switching oligonucleotides (SSOs) target nuclear pre-mRNA molecules to change exon splicing and generate an alternative protein isoform. Clinical trials with two competitive SSO drugs are underway to treat Duchenne muscular dystrophy (DMD). Beyond DMD, many additional therapeutic ap...
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| Main Authors: | , , , , , , |
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| Format: | Artigo |
| Language: | Inglês |
| Published: |
Mary Ann Liebert, Inc.
2014
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| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4098814/ https://ncbi.nlm.nih.gov/pubmed/24826963 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2013.234 |
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