Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial
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DOI: 10.1038/s41591-021-01554-9
Abstract: β-Thalassemias are inherited anemias that are caused by the absent or insufficient production of the β chain of hemoglobin. Here we report 6–8-year follow-up of four adult patients with transfusion-dependent β-thalassemia who were infused with… read more here.
Keywords: reduced intensity; intensity conditioning; lentiviral globin; globin ... See more keywords