Research Highlights

The Center for Gene Therapy is a world-class leader in research on stem cell-based therapies, dedicated to converting that research into real-world treatments and cures as rapidly as possible by:

  • Using a patient’s own blood stem cells to introduce new genes or small noncoding RNAs that will combat cancer, HIV/AIDS, hemophilia, sickle cell disease and other deadly diseases
  • Being at the forefront of technologic innovation that integrates new process development for the clinical scale manufacturing of these therapies
 

Novel Approaches

The research group focuses on new ways to edit the human genome that provide therapeutic options for conventional treatments. For example:
  • In three separate clinical trials, we are testing genetically-modified blood stem cells in HIV/AIDS. One of these studies is a first-in-human trial of blood stem cells modified at the CCR5 gene using zinc finger nucleases.
  • In collaboration with the University of California, San Diego, we are working on engineering HIV individual’s immune T cells to express chimeric antigen receptors (CAR) that can recognize and specifically kill HIV-infected cells. The goal is to achieve long-term remission in the absence of antiretroviral therapy (ART).
  • In collaboration with the Fred Hutchinson Cancer Research Center, the University Hospitals Case Medical Center and Mayo Clinic Hospital Arizona, we are working on making blood stem cells resistant to the toxic side effects of chemotherapy used to fight glioblastoma, while sensitizing the brain tumor cells to chemotherapy by using a more aggressive drug regimen.
  • In collaboration with members of the Department of Pediatrics, the center is exploring how best to collect and then genetically correct the defect in blood stem cells from patients with sickle cell disease.
  • In partnership with biotech companies, the center is testing new clinical-grade manufacturing systems to improve the efficiency of gene modification and stem cell engraftment for various diseases.
     

Leadership

The center combines clinical and research experts in the field of stem cell and gene therapies. 

  • John Zaia, M.D., director of the Center for Gene Therapy, has over 30 years of experience in translational research involving viruses and the immune system. He is a clinical virologist with a major commitment to development of new therapies, particularly in the area of vaccines and gene therapy for HIV/AIDS.
  • Kevin Morris, Ph.D., associate director of the Center for Gene Therapy, has spent the last 15 years developing and determining the role of both small and long noncoding RNAs that can transcriptionally silence HIV-1, CCR5 and other genes. He is dedicated to finding a cure for diseases with unmet needs, including cystic fibrosis, cardiovascular disease and AIDS.
  • Angelo Cardoso, M.D., Ph.D., director of the Laboratory for Cellular Medicine and research professor in the Center for Gene Therapy, has over 20 years of experience in immunology and is responsible for overseeing the manufacturing of gene-modified cell products for ongoing clinical trials. He is committed to developing the next-generation of clinical manufacturing devices and addressing the technical bottlenecks limiting the efficiency of cell therapies.
  • John Burnett, Ph.D., assistant professor in the Center for Gene Therapy, focuses on developing strategies for targeting HIV latency using genome engineering. Another aspect of his work is the application of oligonucleotide-based and targeted genome editing technologies to treat diseases caused by mitochondrial DNA mutations, inherited diseases that currently have no treatments or cures.

 

Looking Ahead

The goal of the Center for Gene Therapy is to continue the clinical translation of alternative therapeutic approaches using leading-edge gene editing approaches. If successful, these therapies could become new standard of care and significantly improve disease outcomes and patient quality of life.