Article

Recent advancements in gene therapy for sickle cell disease and β-thalassemia

2024-09-27

Abstract excerpt

β-hemoglobinopathies, including sickle cell disease (SCD) and β-thalassemia, are prevalent monogenic disorders causing abnormal hemoglobin structure or production that affect millions globally. Current available therapies for SCD and β-thalassemia are primarily symptomatic treatments and allogeneic hematopoietic stem cell transplant (HSCT). Allo-HSCT is the only curative treatment, which has limitations. Gene ther...

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Literature Corpus work: 94aab55a-d645-5af1-a8b9-c958381c5dc7
DOI: 10.3389/frhem.2024.1468952

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