Show Notes
Many rare genetic diseases are caused by a “typo” in our DNA. For most, fixing that spelling mistake has been out of reach. But a recent clinical trial has changed everything, opening up the door for possible cures we never thought were possible through a cutting-edge gene-editing tool called prime editing.
In this episode, Ty Sperle takes us through his own story of being cured of a rare disease called chronic granulomatous disease while his doctor, Dr. Stuart Turvey gives Kaylee the 411 on the science behind Ty’s treatment – and what it could mean for rare diseases more broadly. We also make a stop at the PrimeSight Lab, where researchers are exploring how prime editing could help bring the world into focus for people with inherited vision loss.
For a treatment that once sounded like science fiction, could prime editing be a glimpse of the future of medicine?
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Highlights 00:01 - Ty’s World-First Cure 07:11 - Inside the Prime Editing Revolution 23:43 - Can Prime Editing Restore Sight?
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Sources First-in-world BC patient successfully treated for rare immune disease B.C. teen cured of rare disease Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases
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This episode was originally released on August 2, 2022