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

Published 2016 View Full Article

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Title
An Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants
Authors
Keywords
Exon-Specific U1snRNA, hemophilia B, ExSpeU1, mice, splicing
Journal
Molecular Therapy-Nucleic Acids
Volume 5, Issue -, Pages e370
Publisher
Elsevier BV
Online
2016-10-05
DOI
10.1038/mtna.2016.77

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