“…We note that current trends in gene-therapy development for β-globinopathies favor editing (for potentially improved biosafety) or universal treatment approaches (for R&D profitability). 24 However, improvements in the efficiency of targeted gene addition have started to address biosafety for integrating vectors, 23 and changes in regulatory requirements, manufacturing cost and reimbursement policies may in the future favor more effective, stratified gene-therapy applications, 25 such as that proposed in this study. Figure 3C (n=3), including raw values of band intensities as measured for the unsaturated original images using ImageJ, and including VCN measurements for the three independent biological replicates.…”