Dr. Gao is the 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.
Dr. Gao is an internationally 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 nearly 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. Particularly Dr. Gao started studying Canavan Disease in 1989, discovered the gene and mutations in 1993, discovered the vector (AAV9) for efficient delivering the therapeutic gene to the brain via intravenous injection in 2004, and developed an optimized effective Canavan gene therapy in 2017, which is ready to enter clinical translation.
Dr. Gao has published 240+ research papers, 6 book chapters, and 4 edited books and serves as Editor of Human Gene Therapy, 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 was recently elected for a three-year term as the Vice President (2017-2018), President elect (2018-2019) and President (2019-2020) for the American Society of Gene and Cell Therapy.
Dr. Gao is an elected fellow of the US National Academy of Inventors (NAI), holding 131 patents with 221 more patent applications pending; some have been licensed to 10+ pharmaceutical companies. Recently, Dr. Gao was ranked as #4 of Top 20 Translational Researchers from 2013-to 2017 by Nature Biotechnology, with his patents US852446 (filed by University of Pennsylvania) and US8734809 (filed by UMass) ranked as #2 and #4 of the Top 20 most cited patents, respectively. Dr. Gao co-founded Voyager Therapeutics, focusing on developing rAAV gene therapeutics for treating a variety of devastating CNS disorders.