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

3 Nov 2021

Abstract excerpt

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 promise of a one-time treatment. Thus, here we sought to determine whether LV-corrected blood outgrowth endothelial...

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PMID: 34853801
DOI: 10.1016/j.omtm.2021.10.015

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