| First Author | He Z | Year | 2004 |
| Journal | Nat Med | Volume | 10 |
| Issue | 4 | Pages | 365-7 |
| PubMed ID | 15034572 | Mgi Jnum | J:131270 |
| Mgi Id | MGI:3773417 | Doi | 10.1038/nm1022 |
| Citation | He Z, et al. (2004) Antisickling effects of an endogenous human alpha-like globin. Nat Med 10(4):365-7 |
| abstractText | Gene replacement or gene reactivation therapies for sickle-cell disease (SCD) typically target the mutant beta(S)-globin subunits of hemoglobin-S (alpha(2)beta(S)(2)) for substitution by nonpathological beta-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 alpha-globin subunits for zeta-globin, an endogenous, developmentally silenced, non-beta-like globin. |
