Molecular Genetics and Gene Therapy

Biography

Guangping Gao, Ph.D., is the Director of the Gene Therapy Center and Penelope Booth Rockwell Professor in Biomedical Research, UMass Medical School. He is also a scientific founder of Voyager Therapeutics. He received his Bachelor Degree in Medicine from Sichuan University, China, and Ph.D. in molecular genetics at Florida International University with his work on discovering the human aspartoacylase gene and the genetic mutations responsible for Canavan’s disease. He joined the Institute for Human Gene Therapy (IHGT) at University of Pennsylvania in 1994 and served as the Director of Vector Program of IHGT for vector discovery, process development, vector manufacturing and QC testing. One of his most important contributions to the field of gene therapy is his discovery of a novel family of highly efficient and safe AAV vectors. His primary research interests include molecular mechanisms of AAV evolution, microRNA functional genomics in adult mammals, biology and clinical applications of miRNA therapeutics, and gene therapy of CNS disorders. He has published more than 150 papers in peer-reviewed journals and has 26 patented inventions. He served on several international committees for gene therapy. He is the senior editor of the Book Series on Gene and Cell Therapy publishing by ASGCT and Springer Publisher and serves on Advisory Board of Advances in Experimental Medicine and Biology

Research Interest

Molecular mechanisms of AAV evolution, microRNA functional genomics in adult mammals, biology and clinical applications of miRNA therapeutics, and Gene therapy of CNS disorders