Co-Director, Li Weibo Institute for Rare Diseases
Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor
of Microbiology and Physiological Systems, Penelope Booth Rockwell Professor
in Biomedical Research, University of Massachusetts Medical School; Elected
fellows, both the US National Academy of Inventors (NAI) and the American Academy
of Microbiology; Past President, American Society of Gene and Cell Therapy.
Dr. Gao is an internationally well-recognized gene therapy researcher who has played a key role
in the discovery and characterization of a 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 30+ years of his scientific research career, Dr. 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 has published 346 research papers, 6 book chapters, and 5 edited books. Dr. Gao holds 233 patents with 480 more patent applications pending. He serves as Executive Editor-In-Chief of
Human Gene Therapy, Senior Editor of the Gene and Cell Therapy book series, Associate Editor of Signal Transduction and Targeted Therapy, and Editorial Boards of several other gene
therapy and virology journals. Dr. Gao has been ranked as the World's Top 20 Translational
Researchers for several years in a row by Nature Biotechnology. Dr. Gao is the scientific co-founder of Adrenas Therapeutics, AspA Therapeutics, and Voyager Therapeutics