Clogging up the pipeline: factor VIII aggregates

Abstract

In this issue of Blood, Poothong and colleagues report that increased expression of factor VIII (FVIII) can activate the endoplasmic reticulum (ER) stress response through the formation of amyloid-like fibrils that prevent the further trafficking and secretion of the protein. This study provides insights into the factors that influence the effective secretion of FVIII that may have implications for in vitro protein production systems and in vivo gene therapy.