Efficacy and safety of LentiGlobin gene therapy in patients with transfusion-dependent beta-thalassaemia and non-beta 0/beta 0 genotypes: Updated results from the completed phase 1/2 Northstar and ongoing phase 3 Northstar-2 studies
BRITISH JOURNAL OF HAEMATOLOGY(2019)
关键词
autologous stem cell transplant,beta-thalassemia,gene therapy
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