T-Cell Gene Transfer and Therapy by the Sleeping Beauty Transposon System
The objective of this application is to test whether a novel, naked DNA based vehicle, Sleeping Beauty transposon, can express therapeutic molecules in human T cells for the treatment of leukemia and lymphoma. The rationale for the proposed research is based on Dr. Zhou’s recent finding that the Sleeping Beauty vehicle can transfer and permanently express marker genes in human T cells. To accomplish this goal, blood T cells will be engineered by the Sleeping Beauty vehicle carrying a therapeutic gene and a suicide gene, and tested for efficacy to kill leukemia and lymphoma cells in laboratory tubes and animals. The engineered T cells can also be eliminated by activating the suicide gene if necessary, creating a safe guard for this therapy. Dr. Zhou’s research is expected to develop a novel, simple, inexpensive, efficient, and safe gene therapy using T cells for the treatment of patients with relapsed leukemia and lymphoma after chemotherapy and/or bone marrow transplantation.