Article

Engineering of the Endogenous <i>HBD</i> promoter increases HbA2

2022-12-20

Abstract excerpt

The β-hemoglobinopathies, such as sickle cell disease and β-thalassemia, are one of the most common genetic diseases worldwide and are caused by mutations affecting the structure or production of β-globin subunits in adult hemoglobin. Many gene editing efforts to treat the β-hemoglobinopathies attempt to correct β-globin mutations or increase γ-globin for fetal hemoglobin production. δ-globin, the subunit of adult...

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Literature Corpus work: dae00e6d-8d4e-5bd5-8be0-0e183daaac95
DOI: 10.1101/2022.12.19.521003

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