Insight Series

Risk Assessment of Lentiviral and AAV Gene Therapy Vectors

October 17, 2023 | 11:00 AM CT

A key factor to successful gene therapy is efforts to monitor the fate of gene-corrected cells. Lentivirus and AAV vectors can efficiently introduce new genetic material into human cells; however, risks exist for genotoxicity and malignant transformation. This panel of experts will discuss state-of-the-art technologies and assays for integration site profiling, clonality, and oncogenesis.


  • Moderators: Jessica Lynch, PhD - Johnson and Johnson, and Andrew Wilber, PhD - Southern Illinois University School of Medicine

  • Frederic Bushman, PhD - Perelman School of Medicine at the University of Pennsylvania
    Genomic analysis of retroviral and AAV gene therapy vectors and consequences for the human genome

  • Markus Grompe, MD - Oregon Health and Science University
    Preclinical evaluation of AAV for safety and efficacy in treatment of metabolic disorders

  • Silvana Libertini, PhD - Novartis
    Safety of cell therapy products - methods to assess tumorigenicity

Watch on demand

More ASGCT Insights

Register for more sessions during ASGCT Insights Week, Oct. 16-20. Members and registrants can watch on demand!


Register for the ASGCT Policy Summit

September 23-24, 2024 | Washington, D.C.

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