The Optimized γ-Globin Lentiviral Vector GGHI-mB-3D Leads to Nearly Therapeutic HbF Levels In Vitro in CD34⁺ Cells from Sickle Cell Disease Patients.

RP-HPLC analysis demonstrated that GGHI-mB-3D-transduced cells led to a highly significant increase in mean A γ/α chain ratio ( p = 0.02, n = 9) ( Figure 1 B, left panel); specifically, from 0.23 ± 0.08 observed in the control to 0.28 ± 0.08 ( Table 1 ), with a mean difference of 0.056 ± 0.057 ( Figure S3A ).

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Regarding the HbF levels following transduction with GGHI and GGHI-mB-3D LVs ( Figure 3 C, right panel), both exhibited a marginally significant increase, reaching a mean of 55 ± 17% and 57 ± 20% (mean difference between GGHI and Ctrl of 4.40 ± 3.82, and GGHI-mB-3D and Ctrl of 5.93 ± 4.84, Figure S3B ), compared to 51 ± 18% in mock-transduced cells ( p = 0.062 and p = 0.052, respectively, n = 5).

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When patients were divided according to genotype, transduction of β S β S and β S β + cohorts with both GGHI and GGHI-mB-3D LVs showed a trend, but not a statistically significant increase.

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