Hemostasis Today
NewsFeed
Headlines Hematology Health Medicine VTE ASH Plasma Donation
November, 2026
November 2026
M T W T F S S
 1
2345678
9101112131415
16171819202122
23242526272829
30  
Wolfgang Miesbach: Targeting Hepatic Endothelial Cells for Lentiviral Gene Therapy in Haemophilia A
Wolfgang Miesbach and Floriya Antony/LinkedIn
Nov 4, 2026, 15:47

Wolfgang Miesbach: Targeting Hepatic Endothelial Cells for Lentiviral Gene Therapy in Haemophilia A

Wolfgang Miesbach, Head of Hemostasis/Hemophilia Center at Medical Clinic 2 at University Hospital Frankfurt, shared a post on LinkedIn:

“FVIII was never a hepatocyte’s job.

ESGCT 26, Floriya Antony on the hepatic endothelium as a target for lentiviral gene therapy in haemophilia A:

The liver’s natural source of FVIII is the sinusoidal endothelial cell, not the hepatocyte.

Forcing a hepatocyte to overproduce a large, awkward protein loads the endoplasmic reticulum, and sustained ER stress can turn the unfolded protein response from pro-survival to pro-apoptotic. A lentiviral vector driven by the endothelial stabilin-2 promoter puts the gene back where the biology expects it.

  • Sustained FVIII activity of about 10% for up to 24 weeks in haemophilia A mice
  • Lower long-term ER stress markers and less hepatic lipid accumulation than a ubiquitous promoter
  • No relevant anti-FVIII antibodies, no significant liver toxicity in adult or newborn mice
  • Efficacy persisted after deliberate endothelial injury; neonatal treatment promoted vascular recovery

Taken together, these preclinical findings support the hepatic endothelium as a promising target for safe and durable lentiviral gene therapy in haemophilia A.”

Wolfgang Miesbach

Other posts featuring Wolfgang Miesbach on Hemostasis Today.

bleeding bleeding disorders Blood ESGCT 26 Floriya Antony FVIII haemophilia A Health Hematology Hemophilia Hemostasis Hemostasis News Hemostasis Today hepatocyte lentiviral vector Liver Medicine sinusoidal endothelial cell Wolfgang Miesbach
NewsFeed