Dr. Gao received his Ph.D. degree in biological sciences from the Florida International University, with his work involving the discovery of the human aspartoacylase gene and mutations responsible for Canavan disease, an inherited rare leukodystrophy. Dr. Gao developed his career in gene therapy at the University of Pennsylvania. Dr. Gao’s primary research interests include molecular mechanisms of AAV evolution and diversity, AAV capsid structure-tissue tropism-host response correlations, AAV capsid discovery, modifications and vector genome engineering for novel vector development, next generation recombinant AAV (rAAV) manufacturing and Quality Control testing pipeline development, gene replacement, addition, silencing and editing for in vivo somatic gene therapy, molecular and immunological interactions between endogenous AAV, rAAV and host, microRNA functional genomics and therapeutic development, animal modeling for human genetic diseases, preclinical, translational and clinical development of rAAV gene therapy for rare neurological diseases. Dr. Gao has published 217 papers with 121 granted patents and 215 patents pending. Dr. Gao cofounded Voyager Therapeutics, a biopharmaceutical company focusing on rAAV gene therapeutics for CNS disorders. Dr. Gao serves as the Editor of Human Gene Therapy and on editorial board of several other gene therapy, neurological disease and virology journals. He was recently elected as the Vice President (2017-2018), President-elect (2018-2019), President (2019-2020) of American Society of Gene and Cell Therapy.
