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doi: 10.1164/rccm.200604-511OC

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The differences between our own product and trial protocol and those of the Sadelain group include (1) differences in the boundaries of the beta-globin gene, beta-globin promoter and LCR elements, (2) the initial presence of the cHS4 insulator core in our vector, (3) an anti-sickling and chromatography-identifiable AT87Q -globin variant, (4) differences in vector manufacturing and purification, (5) full-dose intravenous busulfan treatment, but without the addition of an immunosuppressive agent, (6) applicability to both beta-thalassemia and sickle cell disease due to our choice of the AT87Q -variant, and (7) initial focus on betaE/beta0-thalassemia major patients [148], [169], [170], [174]