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...

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Detalles Bibliográficos
Autores principales: Coady, Tristan H., Baughan, Travis D., Shababi, Monir, Passini, Marco A., Lorson, Christian L.
Formato: Texto
Lenguaje:English
Publicado: Public Library of Science 2008
Materias:
Research Article
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

Internet

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

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