FVIII Storage Site Discovered

Rice University research may impact future gene therapy

It’s been known for years that factor proteins, like factor VIII (FVIII), are produced in the liver. More recently, research has shown that FVIII is produced in endothelial cells, which form the inner lining of blood vessels throughout the body. However, the exact location where FVIII was stored was unknown.

In 2015, Rice Univer­sity researchers bridged that knowledge gap, reporting their findings in a paper published in the journal PLOS One in November 2015. They pinpointed FVIII’s storage site in Weibel-Palade bodies. These structures within endothelial cells also store and release von Willebrand factor (VWF). VWF binds to FVIII, ­keeping it in the circulation longer. 

This discovery has implications for future gene therapy experiments. “Now that we recognize that factor VIII is normally synthesized in endothelial cells and stored in Weibel-Palade bodies, those become the precise, most effective physiological targets for gene delivery,” said Joel L. Moake, MD, hematologist and study co-author. 

Source: Rice University