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Antisense-mediated splice intervention to treat human disease: the odyssey continues
Recent approvals of oligonucleotide analogue drugs to alter gene expression have been welcomed by patient communities but not universally supported. These compounds represent a class of drugs that are designed to target a specific gene transcript, and they include a number of chemical entities to ev...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
F1000 Research Limited
2019
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6534073/ https://www.ncbi.nlm.nih.gov/pubmed/31164976 http://dx.doi.org/10.12688/f1000research.18466.1 |