NEJM and Eric Topol amplify multiple new gene-editing trial results for sickle cell disease, including base editing and Cas12a approaches targeting HBG promoters.
3 new trial results for genome editing of sickle cell disease and β-thalassemia
Original Article: Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease
In the RUBY study, a Cas12a–guide RNA complex was used to target the promoters of HBG1 and HBG2
gene editing with a prime editor was used to treat two persons with chronic granulomatous disease
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