lentiviral vector encoding FVIII

Lentiviral vector encoding FVIII is a gene therapy approach designed for the treatment of Hemophilia A, a genetic bleeding disorder caused by deficiency or dysfunction of coagulation factor VIII (FVIII). This therapeutic strategy involves using a lentiviral vector (LV) to deliver a functional copy of the F8 gene. A specific iteration of this technology, developed by researchers at the Università del Piemonte Orientale, utilizes the liver sinusoidal endothelial cell (LSEC)-specific Stabilin2 (STAB2) promoter. By targeting LSECs—the primary physiological source of FVIII—rather than hepatocytes, the therapy aims to achieve stable and sustained FVIII production while avoiding the endoplasmic reticulum (ER) stress and unfolded protein response (UPR) typically associated with high-level FVIII expression in liver parenchymal cells. Preclinical data suggests this approach maintains liver homeostasis and provides durable phenotypic correction without inducing inhibitor formation.

Other names

LSEC-targeted FVIII gene therapylentiviral vector encoding factor VIII