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Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
- Source :
- Nature medicine. 28(1)
- Publication Year :
- 2020
-
Abstract
- Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are the most prevalent monogenic disorders worldwide. Trial HGB-205 ( NCT02151526 ) aimed at evaluating gene therapy by autologous CD34
Details
- ISSN :
- 1546170X
- Volume :
- 28
- Issue :
- 1
- Database :
- OpenAIRE
- Journal :
- Nature medicine
- Accession number :
- edsair.doi.dedup.....930387735e417bd01aeb26e215185d89