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[High-level production of ?-globin, ?-globin, or therapeutic mutant globins in the RBC lineage by hematopoietic stem cell gene therapy ameliorates or cures the hemoglobinopathies sickle cell disease and beta thalassemia, which are major causes of morbidity and mortality worldwide.]. Sentence from MEDLINE/PubMed, a database of the U.S. National Library of Medicine.
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