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Ep. 295: “Roadmap for Cell Therapy Development” Featuring Drs. Jacqueline Barry, Kapil Bharti, and Jack Mosher

The Stem Cell Podcast

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Transforming Lives: Gene Editing Breakthrough

This chapter explores the revolutionary use of CRISPR technology in treating CPS1 deficiency through a patient-specific gene editing case involving a young boy named KJ. It highlights the rapid development, regulatory approval, and broader implications of personalized medicine for rare genetic diseases, underscoring both the triumphs and challenges faced in this groundbreaking journey.

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