Institute of Structural and Molecular Biology, Division of Biosciences, University College London, London, United Kingdom; Novo Nordisk A/S, Novo Nordisk Park, Måløv, Denmark.
Department of Chemistry, Technical University of Denmark, Kongens Lyngby, Denmark.
Biophys J. 2019 Aug 6;117(3):479-489. doi: 10.1016/j.bpj.2019.07.007. Epub 2019 Jul 11.
The von Willebrand factor (VWF) and coagulation factor VIII (FVIII) are intricately involved in hemostasis. A tight, noncovalent complex between VWF and FVIII prolongs the half-life of FVIII in plasma, and failure to form this complex leads to rapid clearance of FVIII and bleeding diatheses such as hemophilia A and von Willebrand disease (VWD) type 2N. High-resolution insight into the complex between VWF and FVIII has so far been strikingly lacking. This is particularly the case for the flexible a3 region of FVIII, which is imperative for high-affinity binding. Here, a structural and biophysical characterization of the interaction between VWF and FVIII is presented with focus on two of the domains that have been proven pivotal for mediating the interaction, namely the a3 region of FVIII and the TIL'E' domains of VWF. Binding between the FVIII a3 region and VWF TIL'E' was here observed using NMR spectroscopy, where chemical shift changes were localized to two β-sheet regions on the edge of TIL'E' upon FVIII a3 region binding. Isothermal titration calorimetry and NMR spectroscopy were used to characterize the interaction between FVIII and TIL'E' as well as mutants of TIL'E', which further highlights the importance of the β-sheet region of TIL'E' for high-affinity binding. Overall, the results presented provide new insight into the role the FVIII a3 region plays for complex formation between VWF and FVIII and the β-sheet region of TIL'E' is shown to be important for FVIII binding. Thus, the results pave the way for further high-resolution insights into this imperative complex.
血管性血友病因子(VWF)和凝血因子 VIII(FVIII)在止血中密切相关。VWF 和 FVIII 之间紧密的非共价复合物延长了 FVIII 在血浆中的半衰期,如果不能形成这种复合物,会导致 FVIII 迅速清除,从而出现血友病 A 和血管性血友病(VWD)2N 型等出血倾向。迄今为止,人们对 VWF 和 FVIII 之间的复合物缺乏深入的了解。这在 FVIII 的柔性 a3 区域尤其如此,该区域对于高亲和力结合至关重要。本文重点介绍了介导相互作用的两个关键结构域,即 FVIII 的 a3 区域和 VWF 的 TIL'E' 结构域,对 VWF 和 FVIII 之间的相互作用进行了结构和生物物理特性的分析。使用 NMR 光谱学观察到 FVIII a3 区域和 VWF TIL'E' 之间的结合,其中 FVIII a3 区域结合后,TIL'E' 边缘的两个β-折叠区域的化学位移发生变化。等温滴定量热法和 NMR 光谱学用于表征 FVIII 与 TIL'E' 以及 TIL'E' 突变体之间的相互作用,这进一步强调了 TIL'E' 的β-折叠区域对于高亲和力结合的重要性。总的来说,这些结果提供了对 FVIII a3 区域在 VWF 和 FVIII 复合物形成中所起作用的新认识,并且表明 TIL'E' 的β-折叠区域对于 FVIII 结合很重要。因此,这些结果为进一步深入了解这一关键复合物铺平了道路。