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ASGCT Podcast Network

07 - Luigi Naldini, MD, PhD - Giants of Gene Therapy

May 30, 2023
In this enlightening conversation, Luigi Naldini, a pioneer in gene therapy and a professor at San Raffaele University, shares his remarkable journey from Torino to groundbreaking research. He discusses the innovative use of lentiviral vectors and their potential to cure genetic disorders and cancer. Naldini addresses challenges in making gene therapies accessible in low-income countries and emphasizes the importance of diverse representation in genomic research. He also offers insights for aspiring scientists and explores the joy of balancing work with personal passions like art and music.
37:08

Episode guests

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Quick takeaways

  • Dr. Luigi Naldini's pioneering work on lentiviral vectors has fundamentally transformed gene therapy by improving gene transfer efficiency and clinical outcomes.
  • The establishment of the San Raffaele Telethon Institute exemplifies Dr. Naldini's commitment to bridging scientific research with practical medical applications for gene therapy.

Deep dives

Pioneering Advances in Lentiviral Vector-Based Gene Therapy

Dr. Louis G. Naldini is renowned for his pioneering work in lentiviral vector-based gene therapy, which has significantly influenced clinical trials for various genetic diseases and cancer. His innovations have enhanced the efficiency of gene transfer, particularly in non-dividing cells, by leveraging the natural mechanisms of the HIV virus. Naldini's groundbreaking design included self-inactivating vectors, which mitigated genotoxic risks associated with earlier gene transfer methods. This research not only improved therapeutic outcomes but also laid a foundation for safe and effective gene therapies used in clinical settings today.

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