Article

Gene editing without <i>ex vivo</i> culture evades genotoxicity in human hematopoietic stem cells

2023-05-27

Abstract excerpt

<h4>SUMMARY</h4> Gene editing the BCL11A erythroid enhancer is a validated approach to fetal hemoglobin (HbF) induction for β-hemoglobinopathy therapy, though heterogeneity in edit allele distribution and HbF response may impact its safety and efficacy. Here we compared combined CRISPR-Cas9 endonuclease editing of the BCL11A +58 and +55 enhancers with leading gene modification approaches under clinical investig...

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Literature Corpus work: c9c60156-4425-5be3-b954-4b669a75a01d
DOI: 10.1101/2023.05.27.542323

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Gene editing without <i>ex vivo</i> culture evades genotoxicity in human hematopoietic stem cellsDOI 10.1101/2023.05.27.542323Read

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