Correction of murine beta-thalassemia by gene transfer into the germ line.
Correction of murine beta-thalassemia by gene transfer into the germ line.
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通过基因转移至种系来纠正小鼠β-地中海贫血。
DOI：
10.1126/science.3461564
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发表时间：
1986
期刊：
影响因子：
--
通讯作者：
Magram,J
中科院分区：
文献类型：
--
作者：
Costantini,F;Chada,K;Magram,J
通过将克隆的β-珠蛋白基因转移到小鼠生殖系中，纠正了小鼠β-地中海贫血。将克隆的小鼠β主要珠蛋白基因或克隆的人β主要珠蛋白基因导入由于β主要珠蛋白基因缺失而导致β主要珠蛋白合成缺陷的小鼠体内。两种引入的基因都产生了功能性的β-珠蛋白链，导致贫血和相关的红细胞异常在一种情况下减少，在另一种情况下消除。
A murine β-thalassemia was corrected by the transfer of cloned β-globin genes into the mouse germ line. The cloned mouse βmaj-globin gene or the cloned human β-globin gene was introduced into mice deficient in β-globin synthesis because of a deletion of the βmaj-globin gene. Both introduced genes produced functional β-globin chains, leading to a reduction in one case, and elimination in another case, of the anemia and associated abnormalities of the red blood cells.