31 min listen
It’s In Our Genes: The Potential of Gene Therapy with Dr. Peter Marks
It’s In Our Genes: The Potential of Gene Therapy with Dr. Peter Marks
ratings:
Length:
25 minutes
Released:
Mar 6, 2024
Format:
Podcast episode
Description
Gene Therapy's Potential to Transform Rare Disease TreatmentDr. Peter Marks highlights the groundbreaking advancements in gene therapy, especially for rare diseases with high unmet medical needs. He discusses the FDA's role in fostering these innovations, underscoring the importance of understanding gene therapy's application and ensuring its safe delivery. Dr. Marks emphasizes the challenges in manufacturing gene therapies, noting that overcoming these hurdles is essential for treating large populations and common diseases. This insight reflects on the hope and complexity of gene therapy, aiming to provide one-time treatments that could drastically improve patient outcomes.The Role of AI in Enhancing Gene Therapy DevelopmentDr. Marks points out the significant potential use cases for artificial intelligence (AI) in gene therapy, from product design to clinical development and manufacturing. AI’s ability to predict off-target effects and streamline the manufacturing process could be a game-changer in this space. By harnessing AI, researchers and developers can optimize safety signals and process large quantities of data for continuous improvement. This insight underscores the potential of AI to revolutionize gene therapy, making it more efficient and effective.Emphasizing the Human Impact of Gene TherapiesDr. Marks and Miller share personal stories of patients and families affected by rare diseases, illustrating the transformative power of gene therapy. They recount profound effects of recent gene therapy approvals on individuals and their communities, particularly highlighting Duchenne Muscular Dystrophy. Marks's narrative conveys the deep emotional and physical impact of gene therapy, not just as a scientific achievement but as a means to significantly enhance lives. This insight brings to light the importance of patient-centered approaches in the development and application of gene therapies.
Released:
Mar 6, 2024
Format:
Podcast episode
Titles in the series (38)
From ionic to iconic: The story of Nalgene: The Nalgene water bottle has become a hydration staple for many, but did you know it was born from science? That’s right! The Nalge Company, founded in the 1949, went on to launch Nalgene Outdoor changing the face of high-quality hiking and camping essentials. Eric Hansen and Elissa McGee join host, Ron O’Brien, to discuss how the bottle we all know and love evolved into what it is today. by Science with a Twist