Stem cells engineered with lentivirus expressing human fix for the treatment of hemophilia B

Abstract

The aim of this study was to investigate an alternative treatment combining cellular and biomaterial therapy. Human mesenchymal stem cells (hMSC) have increasingly been investigated for cellular therapies due to their immunomodulatory properties and differentiation potential [2]. Enclosure of cells in alginate microcapsules offers immunoprotection to the cells and the added benefit of potential retrievability, enhancing the strategy’s safety.