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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| Main Authors: | , , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2016-01-01T00:00:00Z.
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| Subjects: | |
| Online Access: | Connect to this object online. |
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