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Article

Specificity of CRISPR-Cas9 Editing in Exagamglogene Autotemcel

24 Apr 2024

Abstract excerpt

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 likelihood of off-target editing.

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Identifiers

PMID: 38657268
DOI: 10.1056/nejmc2313119

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