Sickle cell disease gene therapy. We are looking at practical barriers that must be overcome before the first successful gene therapy trial for sickle cell disease can be performed.
In vivo selection using chemical inducers of dimerization. Tony Blau working with researchers at Ariad Pharmaceuticals developed a system whereby cell growth can be regulated in vivo. We are continuing to work with Tony to test this system in a mouse model of pyruvate kinase deficiency and in a large animal model.
In vivo selection using chemotherapy protection. A number of groups have demonstrated that the gene for alkyl-guanine transferase, MGMT, can protect hematopoietic cells against a class of chemotherapy, the nitrosureas. This system is now undergoing clinical trails for bone marrow protection in patients with solid tumors. We are investigating novel vectors that can be used for bone marrow protection and in vivo selection.