Article

Precise modelling and correction of a spectrum of β-thalassemic mutations in human erythroid cells by base editors

2022-06-01

Abstract excerpt

β-thalassemia and HbE result from mutations in the β-globin locus that impedes the production of functional β-hemoglobin and represents one of the most common genetic disorders worldwide. Recent advances in genome engineering have opened up new therapeutic opportunities to directly correct these pathogenic mutations using base editors that install transition mutations (A>G and C>T) in the target region with minima...

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Literature Corpus work: bf322c6c-2614-5791-8f65-636b150deb5e
DOI: 10.1101/2022.06.01.494256

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