Extended Data Fig. 2: β-globin expression in Patient 2 at 12 months post infusion. | Nature Medicine

Extended Data Fig. 2: β-globin expression in Patient 2 at 12 months post infusion.

From: Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial

Extended Data Fig. 2

a, HPLC chromatograms illustrating globin production of erythroid cells from four individual BFU-Es derived from bone marrow obtained from Patient 2 at 12 months post infusion. Top chromatograms: two representative examples from two individual, non-transduced BFU-E from Patient 2; lower chromatograms: two representative examples from two individual, transduced BFU-E from Patient 2. b, β-globin to α-globin ration in erythroids derived from untransduced and transduced HSCs obtained from Patient 2 at 12 months post infusion. The β/α expression ratio determined by HPLC in single BFU-Es increased from a mean of 0.11 to 0.38 in BFU-Es harboring a single copy of the integrated vector, representing a gain of 0.27. Results from BFU-Es derived from untransduced HSCs (n = 2; VCN = 0) and from BFU-Es derived from transduced HSCs (n = 4; VCN = 1). All data are mean ± s.e.m.

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