Curing Genetic Disease Before Life Begins with Tippi MacKenzie

What if we could cure genetic disease before a baby is even born? In this episode, pediatric and fetal surgeon Tippi MacKenzie shares how in utero gene therapy and stem cell transplants could stop devastating diseases before life begins.

Dr. MacKenzie is a pioneer in fetal genome surgery and in utero stem cell therapy, with a mission to give every child a healthy start. As Director of the UCSF Broad Stem Cell Center, she leads groundbreaking clinical trials treating disorders like alpha thalassemia while pushing the boundaries of what’s possible in regenerative medicine.

She also opens up about her personal journey—from Juilliard-trained pianist to surgeon-scientist—and the New England Journal of Medicine article that changed the course of her career.

We discuss the unique biology of the fetus, why fetal immune tolerance presents a once-in-a-lifetime treatment window, and how her team is using lipid nanoparticles to develop future-ready gene editing strategies. We also explore how to better support the next generation of clinician-scientists and why stronger partnerships between academia and industry are key to bringing therapies from lab to patient.

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