Research Highlights

The Center for Gene Therapy is a world-class leader in research on stem cell-based therapies, and is 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 and prevent 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 to provide therapeutic options to conventional treatments. For example:
  • In three separate trials, we are testing gene therapy of blood stem cells for AIDS patients. In one of these, a method of disruption of genes by zinc finger nuclease represents a first-in-human trial.
  • The center is exploring, with other members of the Department of Pediatrics, how best to collect and correct blood stem cells from patients with sickle cell disease.
  • In partnership with biotechs, the center is testing new clinical-grade manufacturing systems to improve the efficiency of gene modification and stem cell engraftment. 

 

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 genetic medicine.
  • Kevin  Morris, Ph.D., is associate director of the Center for Gene Therapy, has spent the last 15 years determining and developing 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 a research professor in the Center for Gene Therapy, and has 20 years of experience in immunology. He 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.

 

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.