An Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants

In cellular models we have demonstrated that a unique U1snRNA targeting an intronic region downstream of a defective exon (Exon-specific U1snRNA, ExSpeU1) can rescue multiple exon-skipping mutations, a relevant cause of genetic disease. Here, we explored in mice the ExSpeU1 U1fix9 toward two model H...

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Detalles Bibliográficos
Autores principales: Balestra, Dario, Scalet, Daniela, Pagani, Franco, Rogalska, Malgorzata Ewa, Mari, Rosella, Bernardi, Francesco, Pinotti, Mirko
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5095682/
https://www.ncbi.nlm.nih.gov/pubmed/27701399
http://dx.doi.org/10.1038/mtna.2016.77

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5095682/
https://www.ncbi.nlm.nih.gov/pubmed/27701399
http://dx.doi.org/10.1038/mtna.2016.77

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