Abstract
Gene replacement or gene reactivation therapies for sickle-cell disease (SCD) typically target the mutant βS-globin subunits of hemoglobin-S (α2βS2) for substitution by nonpathological β-like globins. Here we show, in vitro and in vivo in a transgenic mouse model of SCD, that the adverse properties of hemoglobin-S can be reversed by exchanging its normal α-globin subunits for ζ-globin, an endogenous, developmentally silenced, non-β-like globin.
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Acknowledgements
This research was supported by National Institutes of Health grants HL61399 and HL67118.
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He, Z., Russell, J. Antisickling effects of an endogenous human α-like globin. Nat Med 10, 365–367 (2004). https://doi.org/10.1038/nm1022
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DOI: https://doi.org/10.1038/nm1022
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