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

Research output: Contribution to journalArticleAcademicpeer-review

Original languageEnglish
Pages (from-to)3825-3831
JournalBlood
Volume122
Issue number23
DOIs
Publication statusPublished - 28 Nov 2013

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