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Using Gene Therapy to Help the Blind See
Oct 17, 2024
In this engaging discussion, Shannon Boye, a leading professor and co-founder of Atsena Therapeutics, dives into the transformative world of gene therapy for blindness. She shares fascinating insights about using viruses to deliver therapeutic genes and highlights breakthroughs, such as the trial for Leber Congenital Amaurosis type 1. Shannon also explores the brain's adaptability in processing vision after treatment and the balance between academic passion and the challenges of entrepreneurship in this groundbreaking field.
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Quick takeaways
- Gene therapy is showing promising results in treating hereditary blindness, particularly through innovative methods like viral gene delivery to restore vision.
- Despite hurdles in funding and transitioning from research to clinical trials, significant patient improvements during trials underscore the transformative potential of gene therapies.
Deep dives
Advancements in Gene Therapy for Blindness
Gene therapy has emerged as a promising approach to treating certain forms of blindness, particularly the genetic condition known as LCA1. Researchers like Shannon Boy utilize a method that involves delivering a healthy gene via a virus, which then targets patient cells to restore vision. Through extensive studies on animal models, including chickens, the effectiveness of this method has been validated, showcasing the potential of gene replacement therapy. The transition from animal studies to human applications represents a significant milestone, highlighting the challenging journey of translating laboratory successes into viable treatments for patients.
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