Antisickling effects of an endogenous human α-like globin

被引：14

作者：

He, ZN

Russell, JE ^{[1
]}

机构：

[1] Univ Penn, Sch Med, Dept Med Hematol Oncol, Philadelphia, PA 19104 USA

[2] Univ Penn, Sch Med, Dept Pediat Hematol, Philadelphia, PA 19104 USA

[3] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

来源：

NATURE MEDICINE | 2004年 / 10卷 / 04期

关键词：

D O I：

10.1038/nm1022

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

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(2)(S)) 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.

引用

页码：365 / 367

页数：3

共 12 条

[1]

Bunn H, 1986, HEMOGLOBIN MOL GENET, P502

[2]

Chang J, 1996, BLOOD, V88, P1846

[3] EMBRYONIC ZETA-GLOBIN CHAINS IN ADULTS - A MARKER FOR ALPHA-THALASSEMIA-1 HAPLOTYPE DUE TO A-GREATER-THAN-17.5-KB DELETION [J].