Abstract Alpha‐1‐antitrypsin (AAT) deficiency (AATD) is a genetic disease, caused by
mutation of the AAT gene. Accumulation of mutated AAT protein aggregates in hepatocytes …

Research has been lacking on the natural history, complications, and treatment of
haemophilia B, which is less common than haemophilia A and was recognized as a distinct …

The bleeding disorder hemophilia A (HA) is caused by a single-gene (F8) defect and its
clinical symptom can be substantially improved by a small increase in the plasma …

Abstract Purpose of Review We will review the recent developments of cell sheet technology
as a feasible tissue engineering approach. Specifically, we will focus on the technological …

A large fraction of factor VIII in blood originates from liver sinusoidal endothelial cells
although extrahepatic sources also contribute to plasma factor VIII levels. Identification of …

To better understand cellular basis of hemophilia, cell types capable of producing FVIII need
to be identified. We determined whether bone marrow (BM)–derived cells would produce …

In recent decades, the use of adult multipotent stem cells has paved the way for the
identification of new therapeutic approaches for the treatment of monogenic diseases such …

Approximately 25–30% of the hemophilia A patients develop inhibitory antibodies against
Factor VIII (FVIII) following protein-replacement therapy. This problem is also thought to …

Alpha‐1‐antitrypsin (AAT) deficiency (AATD) is a genetic disease, caused by mutation of the
AAT gene. Accumulation of mutated AAT protein aggregates in hepatocytes leads to …

Liver fibrosis is strongly associated with chronic inflammation. As an alternative to
conventional treatments for fibrosis, mesenchymal stem cells (MSCs) therapy is found to be …