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Ep97: John Evans on Base Edited Therapeutics

The Long Run with Luke Timmerman

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Hematology and Hemotherapy

Sickle cell has been an incredibly exciting area over the last five or six years. It's also quite crowded, unlike almost all our other ivications. We can basely edit the promoter regions of the fidal hemogum gene itself. And with a single base change that we have identified that gives us the activity, we get higher levels of flicob in up regulation than anyone else. Then one o two is the direct correction back to disease causing letter 'a' So we're literally changing the one letter and doing it very efficiently at 80% accuracy. Both are potential best in class options for curative sickle cells treatment.

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