Hyman J. Zimmerman Hepatotoxicity State of the Art: Hepatotoxicity Arising From Gene Therapy

Gene therapy encompasses a range of therapeutic strategies that modify or regulate genetic material (DNA or RNA) to treat or prevent disease. Despite substantial clinical advances and multiple U.S. Food and Drug Administration (FDA) approvals, hepatotoxicity has emerged as a significant toxicity across several gene therapy platforms. This presentation will review the spectrum of hepatotoxicity associated with contemporary gene therapy modalities, evaluate current evidence regarding the underlying mechanisms of liver injury, and discuss established as well as emerging strategies for the prevention and management of gene therapy-associated hepatotoxicity. .