Article

CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells

2018-05-31

Abstract excerpt

Sickle Cell Disease and ß-thalassemia, which are caused by defective or deficient adult ß-globin (HBB) respectively, are the most common serious genetic blood diseases in the world. Expression of the fetal ß-like globin, also known as γ-globin, can ameliorate both disorders by serving in place of the adult ß-globin. Here we use CRISPR-Cas9 gene editing to explore a putative γ-globin silencer region identified by c...

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Literature Corpus work: 7bb390ec-2ab1-5b46-9b2d-6d494af551b3
DOI: 10.1101/335729

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