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The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery
Many genetic diseases are induced by mutations disturbing the maturation of pre-mRNAs, often affecting splicing. Antisense oligoribonucleotides (AONs) have been used to modulate splicing thereby circumventing the deleterious effects of mutations. Stable delivery of antisense sequences is achieved by...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Nature Publishing Group
2012
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3390224/ https://www.ncbi.nlm.nih.gov/pubmed/23344083 http://dx.doi.org/10.1038/mtna.2012.24 |