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ASGCT Podcast Network

New Technologies for Gene Targeting + Gene Correction with David Segal, PhD

May 7, 2024
David Segal, PhD, an abstract session co-chair at the ASGCT Annual Meeting, sheds light on groundbreaking advancements in gene targeting and correction. He discusses the transition of academic innovations like CRISPR from labs to industry, aiming to benefit rare disease patients. Segal highlights the growing influence of biotech in genetic therapies and the crucial challenges of effective delivery systems. The conversation also addresses the FDA's impactful approval of the first gene editing therapy, signaling exciting times ahead for genetic medicine.
05:14

Episode guests

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Quick takeaways

  • Significant advancements in gene editing technologies, particularly CRISPR and epigenetic methods, are crucial for developing new RNA therapies and gene correction.
  • The collaboration between academia and biotech industries is enhancing the translation of research into effective treatments for various genetic diseases.

Deep dives

Advancements in Gene Editing Technologies

Recent discussions highlight significant advancements in gene editing technologies, particularly focusing on CRISPR and epigenetic editing methods. These innovations are paving the way for new RNA therapies and the application of artificial intelligence to facilitate gene correction. Notably, the FDA's approval of the first gene editing gene therapy, known as Cass Gevi, marks a pivotal moment in the field, showcasing the potential therapeutic benefits of these technologies. The increasing variety and sophistication of these approaches reflect the vibrant research landscape aimed at addressing genetic diseases, ranging from common conditions like cancer to rare genetic disorders.

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