Zoom link => https://us02web.zoom.us/j/89588355585
Adeno-associated virus vector-based in vivo gene therapy
– State of the art, challenges and strategies for mitigation
Guangping Gao
Director, Horae Gene Therapy Center
Co-Director, Weibo Li Institute for Rare Diseases Research, University of Massachusetts Medical School
Adeno-associated virus vector-based in vivo gene therapy – State of the art, challenges and strategies for mitigation
Guangping Gao, PhD, Horae Gene Therapy Center & Weibo Li Institute for Rare Diseases Research, University of Massachusetts Medical School.
This presentation will provide an overview on the key principles, state of the art, challenges, and potential strategies to mitigate in AAV in vivo gene therapy. The presentation will also showcase AAV capsid discovery and engineering to modulate target tissue tropism, therapeutic gene expression cassette design and optimization to enhance transduction efficiency, tissue/cell type specify and reduce immunogenicity, and examples of AAV gene therapy development: from proof-of-concept preclinical studies to first-in-human clinical evaluation.
Speaker Short Bio
Guangping Gao, PhD is the Director, Horae Gene Therapy Center and Viral Vector Core, Co-Director, Li Weibo Institute for Rare Diseases Research, 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 American Academy of Microbiology; Past president, American Society of Gene and Cell Therapy.
Dr. Gao is an internationally well recognized gene therapy researcher, instrumental in the discovery and characterization of a new family of adeno-associated virus (AAV) serotypes, which revitalized the field of gene therapy. Dr. Gao’s research primarily focuses on gene therapy platform technologies and product development.
Dr. Gao has published more 310+ research papers, 6 book chapters, and 5 edited books. Dr. Gao holds 191 patents with 401 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 on Editorial Boards of several other gene therapy and virology journals. Dr. Gao was ranked as the World Top 20 Translational Researchers by Nature Biotechnology.
