University of Cambridge, Department of Biochemistry, Tennis Court Road, CB2 1QW Cambridge, UK.
Methods. 2012 Sep;58(1):69-78. doi: 10.1016/j.ymeth.2012.07.008. Epub 2012 Jul 27.
Notch signalling occurs via direct cell-cell interactions and plays an important role in linking the fates of neighbouring cells. There are four different mammalian Notch receptors that can be activated by five cell surface ligands. The ability to inhibit specific Notch receptors would help identify the roles of individual family members and potentially provide a means to study and control cell differentiation. Anti-Notch antibodies in the form of single chain Fvs were generated from an antibody phage display library by selection on either the ligand binding domain or the negative regulatory region (NRR) of Notch1 and Notch2. Six antibodies targeting the NRR of Notch1 and four antibodies recognising the NRR of Notch2 were found to prevent receptor activation in cell-based luciferase reporter assays. These antibodies were potent, highly specific inhibitors of individual Notch receptors and interfered with endogenous signalling in stem cell systems of both human and mouse origin. Antibody-mediated inhibition of Notch efficiently down-regulated transcription of the immediate Notch target gene hairy and enhancer of split 5 (Hes5) in both mouse and human neural stem cells and revealed a redundant regulation of Hes5 in these cells as complete down-regulation was seen only after simultaneous blocking of Notch1 and Notch2. In addition, these antibodies promoted differentiation of neural stem cells towards a neuronal fate. In contrast to the widely used small molecule γ-secretase inhibitors, which block all 4 Notch receptors (and a multitude of other signalling pathways), antibodies allow blockade of individual Notch family members in a highly specific way. Specific inhibition will allow examination of the effect of individual Notch receptors in complex differentiation schemes regulated by the co-ordinated action of multiple signalling pathways.
Notch 信号通路通过细胞间的直接相互作用发挥作用,在连接相邻细胞的命运方面发挥着重要作用。哺乳动物中有四种不同的 Notch 受体,它们可以被五种细胞表面配体激活。抑制特定 Notch 受体的能力将有助于确定各个家族成员的作用,并有可能提供一种研究和控制细胞分化的方法。通过选择 Notch1 和 Notch2 的配体结合域或负调控区 (NRR),从抗体噬菌体展示文库中生成了 Notch1 和 Notch2 的单链 Fv 形式的抗 Notch 抗体。发现六种针对 Notch1 的 NRR 的抗体和四种识别 Notch2 的 NRR 的抗体能够在基于细胞的荧光素酶报告基因测定中阻止受体激活。这些抗体是针对单个 Notch 受体的有效、高度特异性抑制剂,并且在人源和鼠源干细胞系统中干扰内源性信号。抗体介导的 Notch 抑制有效地下调了小鼠和人神经干细胞中瞬时 Notch 靶基因 hairy 和 enhancer of split 5 (Hes5) 的转录,并揭示了这些细胞中 Hes5 的冗余调节,因为只有在同时阻断 Notch1 和 Notch2 后才能观察到完全下调。此外,这些抗体促进了神经干细胞向神经元命运的分化。与广泛使用的阻断所有 4 种 Notch 受体(和许多其他信号通路)的小分子 γ-分泌酶抑制剂不同,抗体以高度特异性的方式允许阻断单个 Notch 家族成员。特异性抑制将允许检查单个 Notch 受体在受多个信号通路协调作用调节的复杂分化方案中的作用。