Table 2.

Increased Levels of γ-Globin and HbF in Erythroblasts Derived from Bone Marrow CD34⁺ Cells of β-Hemoglobinopathy Patients Transduced with IGF2BP1 Lentiviral Vectors

	β-Thalassemia Patient 1				β-Thalassemia Patient 2				SCD Patient
	Mock	GFP	SI2AP	FI2AP	Mock	GFP	SI2AP	FI2AP	Mock	GFP	SI2AP	FI2AP
IGF2BP1 mRNAa	1.4	0.7	8.6	101.8	0.03	0.03	17.3	65.8	0.18	0.06	46	141
% IGF2BP1+b	8	9	59	79	8	13	52	60	32	37	70	74
γ-Globin mRNAc	61.1	73.1	92.3	99.2	76.5	86.6	97.6	98.8	33.0	56.8	91.2	96.5
% HbA or HbSd	27	24	19	9	26	27	9	12	81	74	33	23
% HbA2d	23	23	8	0	15	12	4	0	4	5	0	0
% HbFd	50	53	73	91	59	61	87	88	15	21	67	77

^aIGF2BP1 mRNA as percentage of RNaseP determined by qRT-PCR.

^bPercentage of IGF2BP1-positive cells determined by flow cytometry.

^cRatio of γ-globin to total β-like globin (γ-globin + β-globin) mRNA determined by qRT-PCR.

^dPercentage to total hemoglobin (HbF + HbA/S + HbA₂) determined by HPLC.