22min chapter

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06 - Terry Flotte, MD - Giants of Gene Therapy

ASGCT Podcast Network

CHAPTER

Pioneering Gene Therapy: A Journey Through Challenges and Triumphs

This chapter recounts the speaker's early career and pivotal experiences in pediatric pulmonary medicine that led to the first human trial of AAV gene therapy for cystic fibrosis. It emphasizes the evolution of gene therapy, addressing the technological challenges, safety concerns, and the successes achieved with AAV as a vector. The narrative showcases significant advancements in treating genetic disorders and the ongoing efforts to refine gene therapy techniques for broader applications.

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