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Article

Precise correction of a spectrum of β-thalassemia mutations in coding and non-coding regions by base editors

3 May 2024

Abstract excerpt

β-thalassemia/HbE results from mutations in the β-globin locus that impede the production of functional adult hemoglobin. Base editors (BEs) could facilitate the correction of the point mutations with minimal or no indel creation, but its efficiency and bystander editing for the correction of β-thalassemia mutations in coding and non-coding regions remains unexplored. Here, we screened BE variants in HUDEP-2...

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CRISPR and Genetic Engineering
Genomics and Rare Diseases
Hemoglobinopathies and Related Disorders

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 38817682
DOI: 10.1016/j.omtn.2024.102205

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