Recombinant Adeno-Associated Virus for Embryonic and In Vivo Genome Editing
Description
Dr. Guangping Gao, University of Massachusetts Medical School
Hosted by: Feng Zhang
Speaker Bio
Guangping Gao, Ph.D.
Dr. Guangping Gao received his Bachelor Degree in Medicine from the West China Medical School of Sichuan University. Dr. Gao completed his Ph.D. training in molecular genetics at Florida International University with his work involving the isolation and characterization of the human aspartoacylase gene and the genetic mutations responsible for Canavan disease, a severe form of inherited neurodegenerative diseases. Dr. Gao joined the University of Pennsylvania in 1994 where he has developed his career in viral vector gene therapy, served as the Director of Vector Program to oversee the vector discovery and development, process development, GMP vector manufacturing and research vector core and quality control testing. He discovered and vectorized a novel primate adeno-associated virus (AAV) family and the development of simian adenovirus vector based genetic vaccine programs.
Dr. Gao joined the University of Massachusetts Medical School (UMMS) in 2008 as the Founding Director of the Hoare Gene Therapy Center and Viral Vector Core, the Scientific Director of the UMMS-China Translational Research Initiative, Professor of Microbiology and Physiological Systems, and the Penelope Booth Rockwell Professor in Biomedical Research.
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 diseases such as Canavan disease, an inherited lethal white matter degenerative disorder.
Dr. Gao has published 213 papers in peer-reviewed journals, edited 5 books and have 121 granted patents some of which have been licensed to more than 10 pharmaceutical companies as well as 215 patents pending. He is a frequently invited speaker at international gene therapy conferences. In October and November of 2003, one of his PNAS papers on novel AAV vector discovery was selected by ISI as among the most cited papers (top 1%) and hottest break-through papers (top 0.1%).
Dr. Gao is the Editor of Human Gene Therapy, the Senior Editor of the Book Series on Gene and Cell Therapy publishing by American Society of Gene and Cell Therapy (ASGCT) and Springer Publisher, and Associate Editor of Signal Transduction and Targeted Therapy. He served on Advisory Board of Advances in Experimental Medicine and Biology. He also serves on the Board of Directors, Vice President (20-17-2018), President-elect (2018-2019), President (2019-2020) of ASGCT, the Editorial Boards of Gene Therapy, Molecular and Cellular Therapy, Journal of Neurological Disorders, Current Opinion in Virology and many study sections and steering committees for NIH and other national and international research organizations, and as vice president of ASGCT. He cofounded Voyager Therapeutics, a Cambridge (MA, USA) based biopharmaceutical company that focuses on developing rAAV gene therapeutics for treating a variety of devastating CNS disorders.