Dr. Lili Wang is a research associate professor and a research director at the Gene Therapy Program at the University of Pennsylvania. She has been in the field AAV gene therapy for over 25 years with extensive experience in the development of in vivo gene therapy for rare genetic diseases, including vector design & vector optimization, preclinical vector production, in vivo gene therapy in small and large animal models. Several of the preclinical programs she led have advanced to phase I – III clinical trials. Over the past 8 years, she has expanded her research interest to in vivo genome editing with CRISPR/Cas9 and meganuclease and demonstrated efficacy of in vivo genome editing/gene targeting as novel treatment approaches for liver metabolic diseases in mouse models and nonhuman primates. In her talk, she will discuss her work on developing AAV gene therapy and in vivo genome editing to treat metabolic diseases in liver, such as OTC deficiency.
