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doi:10.3109/14767058.2015.1049149 [69] Paesano R, Berlutti F, Pietropaoli M, et al

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]

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