Specificity of CRISPR-Cas9 Editing in Exagamglogene Autotemcel,The New England Journal of Medicine

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Specificity of CRISPR-Cas9 Editing in Exagamglogene Autotemcel
The New England Journal of Medicine ( IF 158.5 ) Pub Date : 2024-04-24 , DOI: 10.1056/nejmc2313119
Angela Yen ₁ , Zachary Zappala ₁ , Rebecca S. Fine ₁ , Timothy D. Majarian ₁ , Parin Sripakdeevong ₂ , David Altshuler ₁

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The autologous cellular therapy exagamglogene autotemcel is generated by editing an erythroid-specific enhancer of BCL11A. Could another site be edited unintentionally? This study gauged the likeli...

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