Although undifferentiated human embryonic stem cells (hESCs) are tumorigenic, this capacity is lost after differentiation, and hESCs are being widely investigated for applications in regenerative medicine. To engineer protection against the unintentional transplantation of undifferentiated cells, we generated hESCs carrying a construct in which the alpha1,3-galactosyltransferase (GalT) open reading frame was transcribed from the hTERT promoter (pmGT). Because the endogenous GalT gene is inactive, GalT expression was limited to undifferentiated cells. A second chimeric construct (pmfGT) differed by replacement of the GalT leader sequence for that of the fucosyltransferase gene. Two subclones containing stable integrations of pmGT and pmfGT (M2 and F11, respectively) were assessed for their response to human serum containing antibodies to the Galalpha1-3Galbeta1-4GlcNAc-R (alpha-gal) epitope. The low-variegation line, M2, and to a lesser extent the more variegated line F11, were sensitive to human serum when exposed in the undifferentiated state. However, M2 cells were largely insensitive after differentiation and retained both a normal karyotype and the ability to differentiate into derivatives of the three germ layers in severe combined immunodeficient mice. These data exemplify a method of protection against residual, undifferentiated hESCs prior to engraftment and may provide ongoing immune surveillance after engraftment against dedifferentiation or against de novo tumorigenesis involving hTERT reactivation. Untransfected H9 cells were not sensitive to the human serum used in this study. Hence, in our system, interactions of hESCs with other circulating antibodies, such as anti-Neu5Gc, were not observed.

译文

尽管未分化的人类胚胎干细胞 (hesc) 具有致瘤性,但这种能力在分化后会丧失,并且hesc正在广泛研究其在再生医学中的应用。为了保护未分化细胞的无意移植,我们产生了带有构建体的hesc,其中 α1,3-半乳糖基转移酶 (GalT) 开放阅读框从hTERT启动子 (pmGT) 转录。由于内源性GalT基因无活性,因此GalT表达仅限于未分化细胞。第二个嵌合构建体 (pmfGT) 的区别在于将GalT前导序列替换为岩藻糖基转移酶基因。评估了两个含有pmGT和pmfGT稳定整合的亚克隆 (分别为M2和F11) 对含有Galalpha1-3Galbeta1-4GlcNAc-R (α-gal) 表位抗体的人血清的反应。在未分化状态下暴露时,低杂色系M2和较小程度的杂色系F11对人血清敏感。然而,在严重的联合免疫缺陷小鼠中,M2细胞在分化后基本不敏感,并保留了正常的核型和分化为三个胚层衍生物的能力。这些数据举例说明了在植入前针对残留的,未分化的hesc的保护方法,并且可以在植入后针对去分化或针对涉及hTERT再激活的从头肿瘤发生提供持续的免疫监视。未转染的H9细胞对本研究中使用的人血清不敏感。因此,在我们的系统中,未观察到hesc与其他循环抗体 (例如anti-Neu5Gc) 的相互作用。

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