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Antisense-based RNA therapy of factor V deficiency: in vitro and ex vivo rescue of a F5 deep-intronic splicing mutation

Francesca Nuzzo, Claudia Radu, Marco Baralle, Luca Spiezia, Tilman M. Hackeng, Paolo Simioni, Elisabetta Castoldi
Journal: Blood
Blood (2013) 122 (23): 3825–3831.
https://doi.org/10.1182/blood-2013-04-499657
Published: 2013
Includes: Supplemental data