Pászty C, Brion C M, Manci E, Witkowska H E, Stevens M E, Mohandas N, Rubin E M
Human Genome Center and Department of Subcellular Structure, Lawrence Berkeley National Laboratory, 1 Cyclotron Road (MS 74-157), University of California, Berkeley, CA 94720, USA.
Science. 1997 Oct 31;278(5339):876-8. doi: 10.1126/science.278.5339.876.
To create mice expressing exclusively human sickle hemoglobin (HbS), transgenic mice expressing human alpha-, gamma-, and betaS-globin were generated and bred with knockout mice that had deletions of the murine alpha- and beta-globin genes. These sickle cell mice have the major features (irreversibly sickled red cells, anemia, multiorgan pathology) found in humans with sickle cell disease and, as such, represent a useful in vivo system to accelerate the development of improved therapies for this common genetic disease.
为了培育仅表达人类镰状血红蛋白（HbS）的小鼠，研究人员构建了表达人类α-、γ-和βS-珠蛋白的转基因小鼠，并将其与敲除了小鼠α-和β-珠蛋白基因的基因敲除小鼠进行杂交。这些镰状细胞小鼠具有人类镰状细胞病患者所具有的主要特征（不可逆性镰状红细胞、贫血、多器官病变），因此，它们是一种有用的体内系统，有助于加速开发针对这种常见遗传病的改良疗法。
Zotero 插件