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Article

A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies

4 Feb 2021

Abstract excerpt

Throughout the past decades, the search for a treatment for severe hemoglobinopathies has gained increased interest within the scientific community. The discovery that ɤ-globin expression from intact HBG alleles complements defective HBB alleles underlying β-thalassemia and sickle cell disease, has provided a promising opening for research directed at relieving ɤ-globin repression mechanisms and, thereby, improve...

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PMID: 34713239
DOI: 10.3389/fgeed.2020.617780

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