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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Bibliographic Details
Published in:Mol Ther Nucleic Acids
Main Authors: Balestra, Dario, Scalet, Daniela, Pagani, Franco, Rogalska, Malgorzata Ewa, Mari, Rosella, Bernardi, Francesco, Pinotti, Mirko
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2016
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5095682/
https://ncbi.nlm.nih.gov/pubmed/27701399
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2016.77
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