We are part of the MCB program
The main focus of our lab is to study stem cell biology and stem cell gene transfer with the goal of developing novel stem cell-based treatment strategies for patients with genetic, infectious stem cells (HSCs) although more recently we have also initiated studies using embryonic stem (ES) cells and induced pluripotent stem (iPS) cells. Hematopoietic stem cells are attractive targets for gene therapy because of their ability to permanently reconstitute the hematopoietic and immune systems. However, in order to efficiently modify HSCs one has to be able to identify and isolate HSCs and identify vector systems that allow for efficient transduction of HSCs. In recent studies we have demonstrated efficient gene transfer with lentivirus and foamy virus based vector systems. We have shown successful expansion and in vivo selection of hematopoietic repopulating cells. Based on these studies we have currently an open gene therapy study using chemoprotected stem cells for the treatment of patients with glioblastoma. We are also working on studies for the correction of genetic diseases using lentivirus vectors and gene repair strategies. We are exploring anti-HIV stem cell gene therapy strategies using lentivirus and foamy virus vectors and zinc finger technology. In addition to the hematopoietic stem cell work we are pursuing the generation of iPS cells from large animal models to study iPS – based treatment approaches. We are also exploring novel cancer treatment strategies using oncolytic viruses.
Thus current research in our laboratory focuses on studies to: