Article

Editing of highly homologous gamma-globin genes by nickase deficient Base Editor mitigates large intergenic deletions

2023-12-04

Abstract excerpt

Base editing in gamma-globin promoter is a promising approach for reactivation of fetal-hemoglobin. Recent studies have shown that base editing could result in genotoxic events at the gamma globin locus including 4.9 kb large deletion of intervening region due to nicking in the paralogous HBG 1 and HBG 2 genes. Although the deletion frequency is less than what is observed with Cas9, it could diminish the therape...

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Literature Corpus work: 5f3d6523-2386-5fbf-bddc-5ef575c8d2bc
DOI: 10.1101/2023.12.04.569931

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