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

• Prif Awduron: Dario Balestra (Awdur), Daniela Scalet (Awdur), Franco Pagani (Awdur), Malgorzata Ewa Rogalska (Awdur), Rosella Mari (Awdur), Francesco Bernardi (Awdur), Mirko Pinotti (Awdur)
• Fformat: Llyfr
• Cyhoeddwyd: Elsevier, 2016-01-01T00:00:00Z.
• Pynciau: article
• Mynediad Ar-lein: Connect to this object online.

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...