About Center for Gene Therapy Researchers
John A. Zaia, M.D., Chair, Center For Gene Therapy
For 35 years, renowned genetic researcher John A. Zaia, M.D., has been a critical part of the DNA at City of Hope.
Dr. Zaia graduated from Harvard Medical School (HMS) and completed training in pediatrics and pediatric infectious disease. He was an instructor at Harvard Medical School and a clinical associate at Dana-Farber Cancer Institute in Boston before joining City of Hope in 1980. He served as City of Hope's director of Virology and Infectious Diseases within the Department of Pediatrics until 1999 when he assumed overall leadership of our Department of Virology.
Now, as director of the Center for Gene Therapy, Dr. Zaia aims to take the promise of gene and stem cell therapy to the next level, finding new ways to defeat cancer, AIDS and other diseases.
Dr. Zaia, the Aaron D. Miller and Edith Miller Chair in Gene Therapy, also directs the City of Hope Alpha Clinic, funded by the California Institute for Regenerative Medicine. The clinic is dedicated to bringing novel therapies to patients as rapidly as possible.
The holder of a dozen patents and author of over 180 papers, Dr. Zaia is called a “visionary and a teacher” by his peers. His goal: “Not only to provide research to benefit patients in the future, but get these innovative treatments running in real-life clinics to benefit patients now.”
Angelo A. Cardoso, M.D., Ph.D., Research Professor
Clinical Translation of Next-Generation Cell and Gene Engineering
Angelo Cardoso, M.D., Ph.D., is the Director of the Laboratory for Cellular Medicine (LCM) at the COH’s Center for Gene Therapy, and the Research Director of the Alpha Stem Cell Clinic. The LCM is a specialized laboratory with a pipeline of cell and gene therapy projects, at different stages of development. Our activities range from basic research and technology innovation, to scaling and qualification of IND-enabling GMP processes, and implementation of Phase I clinical trials. The LCM clinical activities include the manufacturing of cellular products for the first-in-human trial of genome-edited hematopoietic stem/progenitor cells (HSPC) for trials of lentiviral-based modification of HSPC for HIV, and a novel graft-engineered strategy for induction of mixed chimerism in Sickle Cell Disease (SCD). Our ‘Process Development’ work is currently focused on the development of next-generation platforms for the manufacturing of genome-edited, gene-modified cell products, integrating new technologies into modular, automated, closed systems. It includes gene therapy projects for chemoprotection of HSPC, correction of genetic defects for SCD, chimeric antigen receptor (CAR)-based immunotherapies for long-term remission of HIV, and large-scale graft engineering. The research activities of the LCM are applied to our clinical goals and include the development of validation of taggable platforms for genome-editing, the design of synthetic vectors for more efficient and selective targeting of HSPC, the use of niche-mimetics and strategies to preserve the stemness of human HSC during the genome-editing process, and engineering of bispecific CAR-expressing T cells to fight viral infections.
John C. Burnett, Ph.D., Assistant Professor
John C. Burnett is investigating the molecular factors that regulate HIV latency, including stochastic factors resulting from HIV Tat protein and the occupancy of host factors at the viral promoter. At the Center for Gene Therapy, he has focused on developing novel strategies to eliminate the latent reservoir and establish a functional cure of HIV. Recent advances towards these goals include the discovery of a CCR5-specific aptamer for delivery of anti-HIV siRNAs and the development of strategies to cure HIV using allogeneic hematopoietic stem cells harboring the CCR5Δ32 mutation. He is investigating the efficacy and safety for these strategies in humanized mouse models for HIV infection and latency that he helped to develop.