Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device
(2021)
Journal Article
Wragg, N. (2021). Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device. Molecular Therapy - Methods and Clinical Development, 551-566. https://doi.org/10.1016/j.omtm.2021.10.015
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 lentivir... Read More about Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device.