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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Detalles Bibliográficos
Autores principales: Olgasi, Cristina, Borsotti, Chiara, Merlin, Simone, Bergmann, Thorsten, Bittorf, Patrick, Adewoye, Adeolu Badi, Wragg, Nicholas, Patterson, Kelcey, Calabria, Andrea, Benedicenti, Fabrizio, Cucci, Alessia, Borchiellini, Alessandra, Pollio, Berardino, Montini, Eugenio, Mazzuca, Delfina M., Zierau, Martin, Stolzing, Alexandra, Toleikis, Philip.M., Braspenning, Joris, Follenzi, Antonia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8606349/
https://www.ncbi.nlm.nih.gov/pubmed/34853801
http://dx.doi.org/10.1016/j.omtm.2021.10.015

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