Special Interview with Prof. Guangping Gao
On June 27, 2026, the Editorial Office of Journal of Translational Genetics and Genomics interviewed Dr. Guangping Gao, the Professor and Chair, Department of Genetic and Cellular Medicine, the Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology, Penelope Booth Rockwell Professor in Biomedical Research, UMass Chan Medical School, Massachusetts, USA.
Prof. Gao has long been dedicated to gene therapy research for rare genetic diseases, making internationally recognized contributions to the development of AAV-based vectors and their clinical translation. In this interview, he reflected on the rapid progress of gene therapy over the past three decades, highlighting key drivers such as advances in viral vector engineering, progress in molecular genetics, and the growing maturity of translational pathways supported by orphan drug policies. He also shared his perspectives on the remaining scientific challenges, including long-term safety, immune responses, limited re-dosing capacity, and insufficient tissue-specific targeting. Looking ahead, he emphasized that major challenges in the field include immunological barriers, delivery efficiency, manufacturing scalability, and the difficulty of extending therapies from monogenic rare diseases to more complex disorders. Drawing on his extensive experience, he further discussed emerging directions with strong potential, such as next-generation engineered AAVs through directed evolution, non-viral delivery systems like lipid nanoparticles (LNPs), high-precision genome editing tools including CRISPR base and prime editing, as well as RNA-based therapeutics and AI-assisted design platforms, all of which are expected to drive the next wave of safer, more precise, and more accessible gene therapies.
Watch the full interview with Prof. Guangping Gao:
Interview Questions:
Q1 Over the past three decades, you have dedicated your career to gene therapy for rare genetic diseases and made pioneering contributions to the discovery and development of AAV vectors. Looking back at the evolution of the gene therapy field, what do you think have been the key drivers behind the rapid progress in gene therapies for rare diseases? What important scientific questions still deserve greater attention in the future?
Q2. What are the most significant challenges currently facing the field of human gene therapy? At the same time, which emerging technologies or research directions do you believe hold the greatest promise for creating new opportunities in this field?
About the Interviewee:
Prof. Guangping Gao, Professor and Chair, Department of Genetic and Cellular Medicine, Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology, Penelope Booth Rockwell Professor in Biomedical Research, UMass Chan Medical School, Massachusetts, USA.
Prof. Gao is an internationally recognized gene therapy researcher who has played a key role in the discovery and characterization of new family of adeno-associated virus (AAV) serotypes, which was instrumental in reviving the gene therapy field, hugely impacting many currently untreatable human diseases. For more than 30 years of his scientific research career, Prof. Gao has primarily focused on molecular genetics and viral vector gene therapy of rare genetic diseases, encompassing disease gene cloning, causative mutation identification, pathomechanism investigation, animal modeling, novel viral vector discovery and engineering for in vivo gene delivery, vector biology, preclinical and clinical gene therapy product development, viral vector manufacturing for preclinical and clinical gene therapy applications as well as technology platforms development as novel approaches for human gene therapy.
Dr. GAO is elected fellows of both the U.S. National Academy of Inventors and American Academy of Microbiology. He holds 277 patents with 600 more patent applications pending. He had been repeated ranked among the world’s top 20 translational researchers by Nature Biotechnology and received Chancellor’s Medal for distinguished scholarship. Dr. GAO co-founded several companies for rare disease gene therapies.
Representative Research Achievements
- Elected and served as vice President (2017) President Elect ( 2018), and President (2019) of the American Society of Gene & Cell Therapy (2020), becoming the first Chinese-origin scientist based in the United States to hold this position
- Founded the first Gene and cell therapy academic department in the U.S. A., serving as the Tenured Professor and Chair for the Department of Genetic and Cellular Medicine at University of Massachusetts Chan Medical School and Director of the HORAE Gene Therapy Center
- Served as Executive Editor-in-Chief of Human Gene Therapy, a leading journal in the field of gene and cell therapy
- Made pioneering contributions to adeno-associated virus (AAV) biology and gene therapy, including discovery and characterization of ~1000 novel AAV variants for gene delivery applications
- Advanced translational gene therapy research in collaboration with leading global investigators, including Prof. James Wilson and other key figures in AAV-based therapy development
- Contributed to development and translational research of gene therapy approaches for inherited diseases, particularly AAV-mediated therapeutic strategies
- Investigated AAV vector biology and host - immune interactions, supporting improved safety and durability of systemic gene delivery
- Elected fellows, both the U.S. National Academy of Inventors and American Academy of Microbiology
- Co-founded several Gene Therapy companies
- Ranked among the world’s top 20 translational researchers by Nature Biotechnology
- Awarded Chancellor’s Medal for distinguished scholarship
- Facilitated international academic exchange through organization of Sino - US gene and cell therapy conferences and training programs.
Prof. Gao has published about 430 research papers, 6 book chapters, and 5 edited books and served as Executive Editor in Chief of Human Gene Therapy, Editor in Chief for Med Comm, Senior Editor of the Gene and Cell Therapy book series, Associate Editor of Signal Transduction and Targeted Therapy, and on Editorial Boards of several other gene therapy and virology journals. Dr. Gao served as the Vice President (2017-2018), President elect (2018-2019) and President (2019-2020) for the American Society of Gene and Cell Therapy.
Editor: Iris Yang
Language Editor: Catherine Yang
Production Editor: Xingyue Luo
Respectfully Submitted by the Editorial Office of Journal of Translational Genetics and Genomics
