Antisickling effects of an endogenous human α-like globin
- 21 March 2004
- journal article
- research article
- Published by Springer Nature in Nature Medicine
- Vol. 10 (4) , 365-367
- https://doi.org/10.1038/nm1022
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.Keywords
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