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Development of a Single Vector System that Enhances Trans-Splicing of SMN2 Transcripts
RNA modalities are developing as a powerful means to re-direct pathogenic pre-mRNA splicing events. Improving the efficiency of these molecules in vivo is critical as they move towards clinical applications. Spinal muscular atrophy (SMA) is caused by loss of SMN1. A nearly identical copy gene called...
| Autores principales: | , , , , |
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| Formato: | Texto |
| Lenguaje: | English |
| Publicado: |
Public Library of Science
2008
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2565107/ https://www.ncbi.nlm.nih.gov/pubmed/18941511 http://dx.doi.org/10.1371/journal.pone.0003468 |