Specificity of CRISPR-Cas9 Editing in Exagamglogene Autotemcel
Angela Yen, Zachary Zappala, Rebecca S. Fine, Timothy D. Majarian, Parin Sripakdeevong, David Altshuler
Abstract
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.
Topics & Concepts
CRISPRGenome editingEnhancerComputer scienceCas9Computational biologyBiologyGeneticsGeneGene expressionCRISPR and Genetic EngineeringVirus-based gene therapy researchMosquito-borne diseases and control