Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device

Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device

Hemophilia A (HA) is a rare bleeding disorder caused by deficiency/dysfunction of the FVIII protein. As current therapies based on frequent FVIII infusions are not a definitive cure, long-term expression of FVIII in endothelial cells through lentiviral vector (LV)-mediated gene transfer holds the pr...

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Bibliographic Details
Main Authors: Cristina Olgasi, Chiara Borsotti, Simone Merlin, Thorsten Bergmann, Patrick Bittorf, Adeolu Badi Adewoye, Nicholas Wragg, Kelcey Patterson, Andrea Calabria, Fabrizio Benedicenti, Alessia Cucci, Alessandra Borchiellini, Berardino Pollio, Eugenio Montini, Delfina M. Mazzuca, Martin Zierau, Alexandra Stolzing, Philip.M. Toleikis, Joris Braspenning, Antonia Follenzi
Format: Article
Language:English
Published: Elsevier 2021-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hemophilia A
cell and gene therapy
medical device
BOEC
lentiviral vector
FVIII
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001728

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http://www.sciencedirect.com/science/article/pii/S2329050121001728

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