About

Zhenwei Song, Ph.D., is a research assistant professor in the Division of Pharmacotherapy and Experimental Therapeutics. After completing training in induced pluripotent stem cells (iPSCs) at Sun Yat-sen University in China, he pursued postdoctoral training at the University of Utah, and the University of North Carolina at Chapel Hill, specializing in cell and gene therapy. Dr. Song joined the Division in 2023. His primary focus has been on advancing the development of safer and more effective AAV vectors for liver-targeted gene therapy.

His ongoing research involves assessing hepatotoxicity risks using 3D spheroid models for AAV gene therapy. Recombinant adeno-associated virus (rAAV) is a promising vector for gene therapy, and has seen extensive application in both preclinical and clinical investigations. However, serious adverse effects, particularly liver toxicity leading to fatalities in clinical trials, have prompted caution from the FDA. Addressing the challenges of debilitating AAV transduction and hepatotoxicity is crucial for meeting clinical standards, yet remains an unmet area of investigation.

Collaborating with Paul Watkins, MD, and Chengwen Li, PhD, his focus is on developing innovative AAV vector constructs aimed at eliminating liver toxicity, and understanding the underlying mechanisms of cellular responses in primary hepatocytes. Their current approach involves utilizing 3D spheroids derived from both mouse and human primary hepatocytes to assess AAV transduction and hepatotoxicity. Additionally, they are establishing a platform using patient-derived cells to determine personalized dosing strategies for clinical applications.