Infectious and Inflammatory Disease Center, Sanford-Burnham Medical Research Institute, 10901 North Torrey Pines Road, La Jolla, California 92037, United States.
J Med Chem. 2011 Jan 27;54(2):562-71. doi: 10.1021/jm101004d. Epub 2010 Dec 29.
The lymphoid tyrosine phosphatase (Lyp, PTPN22) is a critical negative regulator of T cell antigen receptor (TCR) signaling. A single-nucleotide polymorphism (SNP) in the ptpn22 gene correlates with the incidence of various autoimmune diseases, including type 1 diabetes, rheumatoid arthritis, and systemic lupus erythematosus. Since the disease-associated allele is a more potent inhibitor of TCR signaling, specific Lyp inhibitors may become valuable in treating autoimmunity. Using a structure-based approach, we synthesized a library of 34 compounds that inhibited Lyp with IC(50) values between 0.27 and 6.2 μM. A reporter assay was employed to screen for compounds that enhanced TCR signaling in cells, and several inhibitors displayed a dose-dependent, activating effect. Subsequent probing for Lyp's direct physiological targets by immunoblot analysis confirmed the ability of the compounds to inhibit Lyp in T cells. Selectivity profiling against closely related tyrosine phosphatases and in silico docking studies with the crystal structure of Lyp yielded valuable information for the design of Lyp-specific compounds.
淋巴特异酪氨酸磷酸酶(Lyp,PTPN22)是 T 细胞抗原受体(TCR)信号的关键负调控因子。ptpn22 基因中的单核苷酸多态性(SNP)与多种自身免疫性疾病的发生率相关,包括 1 型糖尿病、类风湿关节炎和系统性红斑狼疮。由于与疾病相关的等位基因是 TCR 信号的更强抑制剂,因此特定的 Lyp 抑制剂可能在治疗自身免疫方面具有重要价值。我们采用基于结构的方法,合成了一个包含 34 种化合物的文库,这些化合物对 Lyp 的抑制作用的 IC50 值在 0.27 到 6.2 μM 之间。我们使用报告基因检测法筛选了可增强细胞中 TCR 信号的化合物,其中几种抑制剂表现出剂量依赖性的激活作用。随后通过免疫印迹分析探测 Lyp 的直接生理靶标,证实了这些化合物在 T 细胞中抑制 Lyp 的能力。针对密切相关的酪氨酸磷酸酶的选择性分析以及与 Lyp 晶体结构的计算机对接研究,为 Lyp 特异性化合物的设计提供了有价值的信息。