THE PAST TWO DECADES have brought remarkable progress in our understanding of the
molecular basis of hemophilia A. This disease, which has already been documented as a …

Since increased cholesterol levels are crucial in determining the development of atheroma,
their reduction represents a mainstay in primary and secondary cardiovascular prevention …

The endoplasmic reticulum (ER) is the cellular organelle responsible for protein folding and
assembly, lipid and sterol biosynthesis, and calcium storage. The unfolded protein response …

Recombinant adeno-associated virus (rAAV) vectors encoding human factor VIII (hFVIII)
were systematically evaluated for hemophilia A (HA) gene therapy. A 5.7-kb rAAV …

Factor VIII (FVIII) functions as a cofactor within the intrinsic pathway of blood coagulation.
Quantitative or qualitative deficiencies of FVIII result in the inherited bleeding disorder …

Liver disease is associated with markedly elevated plasma factor VIII (FVIII) levels, whereas
the synthesis of many other coagulation factors and proteins is reduced. In order to define …

Unravelling the structure, function and molecular interactions of factor VIII (FVIII) throughout
its life cycle from biosynthesis to clearance has advanced our understanding of the …

After many reports of successful gene therapy studies in small and large animal models of
haemophilia, we have, at last, seen the first signs of success in human patients. These very …

Gene therapy provides hope for a cure for patients with hemophilia by establishing
continuous endogenous expression of factor VIII or factor IX following transfer of a functional …

Factor VIII (FVIII) is a large glycoprotein that is challenging to express both in vitro and in
vivo. Several studies suggest that high levels of FVIII expression can lead to cellular stress …