Insight Series

Treating Genetic Diseases Prior to Birth from Stem Cells to Gene Delivery

October 19, 2023 | 11:00 AM CT

In utero stem cell transplantation (IUTx) and in utero gene therapy (IUGT) offer the opportunity of treating, and ideally curing, a wide range of genetic disorders. Despite recent progress, there is an urgent need to explore novel cell and gene therapy tools for prenatal application and the ethical considerations surrounding this topic.  


  • Moderators: Afrooz Rashnonejad, PhD - Nationwide Children's Hospital, and William Peranteau, MD - Children's Hospital of Philadelphia

  • Alan Flake, MD - Children's Hospital of Philadelphia
    Using insights from the past and lessons from the present to create the future of prenatal cell therapy

  • Christopher Porada, PhD - Wake Forest Institute for Regenerative Medicine
    Harnessing the potential of in utero gene therapy

  • Tippi MacKenzie, MD - University of California San Francisco
    Clinical trials on fetal cell therapy, briefly touching on regulatory aspect 

  • Saskia Hendriks, MD, PhD - National Institutes of Health
    Ethical considerations for prenatal cell and gene therapies

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

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