The BWC Global Forum: Biotech, Biosecurity & Beyond
Episode #13: Gene Therapy Delivery
Jun 11, 2024
Dr. David Williams discusses cutting-edge gene therapy advancements for pediatric patients. He explains how modified viruses are used as delivery vehicles for targeted cell therapies. The goal is to make treatments more affordable and accessible worldwide. The podcast explores the longevity and effectiveness of gene therapy, as well as the use of innovative delivery methods like chimeric viruses and lipid nanoparticles.
19:26
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Quick takeaways
- Gene therapy using modified viruses can provide long-lasting treatments for severe genetic conditions.
- Advancements in viral vectors like chimeric viruses and lipid nanoparticles aim to expand gene therapy accessibility.
Deep dives
Gene Therapy Applications for Various Diseases
Dr. David Williams, a pediatric hematologist, discusses gene therapy applications for genetic diseases such as severe combined immunodeficiency, sickle cell disease, and cerebral adrenal leukodystrophy. He explains the use of somatic gene therapies to modify cells that produce functional cells like blood cells, emphasizing the success and FDA approval of these approaches.
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