Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, Providence, RI, USA.
mBio. 2013 Jun 11;4(3):e00247-13. doi: 10.1128/mBio.00247-13.
The human JC polyomavirus (JCPyV) is the causative agent of the fatal, demyelinating disease progressive multifocal leukoencephalopathy (PML). The Mad-1 prototype strain of JCPyV uses the glycan lactoseries tetrasaccharide c (LSTc) and serotonin receptor 5-HT2A to attach to and enter into host cells, respectively. Specific residues in the viral capsid protein VP1 are responsible for direct interactions with the α2,6-linked sialic acid of LSTc. Viral isolates from individuals with PML often contain mutations in the sialic acid-binding pocket of VP1 that are hypothesized to arise from positive selection. We reconstituted these mutations in the Mad-1 strain of JCPyV and found that they were not capable of growth. The mutations were then introduced into recombinant VP1 and reconstituted as pentamers in order to conduct binding studies and structural analyses. VP1 pentamers carrying PML-associated mutations were not capable of binding to permissive cells. High-resolution structure determination revealed that these pentamers are well folded but no longer bind to LSTc due to steric clashes in the sialic acid-binding site. Reconstitution of the mutations into JCPyV pseudoviruses allowed us to directly quantify the infectivity of the mutants in several cell lines. The JCPyV pseudoviruses with PML-associated mutations were not infectious, nor were they able to engage sialic acid as measured by hemagglutination of human red blood cells. These results demonstrate that viruses from PML patients with single point mutations in VP1 disrupt binding to sialic acid motifs and render these viruses noninfectious. IMPORTANCE Infection with human JC polyomavirus (JCPyV) is common and asymptomatic in healthy individuals, but during immunosuppression, JCPyV can spread from the kidney to the central nervous system (CNS) and cause a fatal, demyelinating disease, progressive multifocal leukoencephalopathy (PML). Individuals infected with HIV, those who have AIDS, or those receiving immunomodulatory therapies for autoimmune diseases are at serious risk for PML. Recent reports have demonstrated that viral isolates from PML patients often have distinct changes within the major capsid protein. Our structural-functional approach highlights that these mutations result in abolished engagement of the carbohydrate receptor motif LSTc that is necessary for infection. Viruses with PML-associated mutations are not infectious in glial cells, suggesting that they may play an alternative role in PML pathogenesis.
人类 JC 多瘤病毒(JCPyV)是致命的脱髓鞘疾病进行性多灶性白质脑病(PML)的病原体。JCPyV 的 Mad-1 原型株使用聚糖乳糖系列四糖 c(LSTc)和 5-羟色胺受体 5-HT2A 分别附着和进入宿主细胞。病毒衣壳蛋白 VP1 中的特定残基负责与 LSTc 的α2,6-连接唾液酸直接相互作用。来自 PML 患者的病毒分离株通常在 VP1 的唾液酸结合口袋中含有突变,据推测这些突变是由正选择引起的。我们在 JCPyV 的 Mad-1 株中重新构建了这些突变,并发现它们不能生长。然后将这些突变引入重组 VP1 中,并重新组装成五聚体以进行结合研究和结构分析。携带 PML 相关突变的 VP1 五聚体不能与允许的细胞结合。高分辨率结构测定表明,这些五聚体折叠良好,但由于唾液酸结合位点的空间位阻,不再与 LSTc 结合。将突变重新构建到 JCPyV 假病毒中,使我们能够直接在几种细胞系中定量测定突变体的感染性。带有 PML 相关突变的 JCPyV 假病毒没有感染性,也不能像通过人红细胞的血凝作用测量的那样与唾液酸结合。这些结果表明,VP1 单点突变的 PML 患者的病毒破坏了与唾液酸基序的结合,并使这些病毒失去感染性。重要性 健康个体中人类 JC 多瘤病毒(JCPyV)的感染很常见且无症状,但在免疫抑制期间,JCPyV 可以从肾脏传播到中枢神经系统(CNS)并导致致命的脱髓鞘疾病进行性多灶性白质脑病(PML)。感染 HIV 的个体、患有艾滋病的个体或接受自身免疫性疾病免疫调节治疗的个体患 PML 的风险很高。最近的报告表明,来自 PML 患者的病毒分离株通常在主要衣壳蛋白内具有明显的变化。我们的结构功能方法强调,这些突变导致感染所需的碳水化合物受体基序 LSTc 的结合被废除。带有 PML 相关突变的病毒在神经胶质细胞中没有感染性,这表明它们可能在 PML 发病机制中发挥替代作用。