Walensky Loren D, Kung Andrew L, Escher Iris, Malia Thomas J, Barbuto Scott, Wright Renee D, Wagner Gerhard, Verdine Gregory L, Korsmeyer Stanley J
Howard Hughes Medical Institute, Department of Pediatric Hematology/Oncology and Children's Hospital Boston, Massachusetts, USA.
Science. 2004 Sep 3;305(5689):1466-70. doi: 10.1126/science.1099191.
BCL-2 family proteins constitute a critical control point for the regulation of apoptosis. Protein interaction between BCL-2 members is a prominent mechanism of control and is mediated through the amphipathic alpha-helical BH3 segment, an essential death domain. We used a chemical strategy, termed hydrocarbon stapling, to generate BH3 peptides with improved pharmacologic properties. The stapled peptides, called "stabilized alpha-helix of BCL-2 domains" (SAHBs), proved to be helical, protease-resistant, and cell-permeable molecules that bound with increased affinity to multidomain BCL-2 member pockets. A SAHB of the BH3 domain from the BID protein specifically activated the apoptotic pathway to kill leukemia cells. In addition, SAHB effectively inhibited the growth of human leukemia xenografts in vivo. Hydrocarbon stapling of native peptides may provide a useful strategy for experimental and therapeutic modulation of protein-protein interactions in many signaling pathways.
BCL-2家族蛋白构成了细胞凋亡调控的关键控制点。BCL-2成员之间的蛋白质相互作用是一种重要的调控机制,通过两亲性α-螺旋BH3片段介导,这是一个必需的死亡结构域。我们采用一种称为烃链固定的化学策略来生成具有改善药理特性的BH3肽。这些被称为“BCL-2结构域稳定α-螺旋”(SAHBs)的链固定肽被证明是螺旋状、抗蛋白酶且可穿透细胞的分子,它们与多结构域BCL-2成员口袋的结合亲和力增强。来自BID蛋白的BH3结构域的SAHB特异性激活凋亡途径以杀死白血病细胞。此外,SAHB在体内有效抑制人白血病异种移植瘤的生长。天然肽的烃链固定可能为实验性和治疗性调节许多信号通路中的蛋白质 - 蛋白质相互作用提供一种有用的策略。