Department of Plasma Proteins, Sanquin Research, 1066 CX Amsterdam, The Netherlands.
Department of Plasma Proteins, Sanquin Research, 1066 CX Amsterdam, The Netherlands
Biochem J. 2018 Sep 11;475(17):2819-2830. doi: 10.1042/BCJ20180431.
The D'-D3 fragment of von Willebrand factor (VWF) can be divided into TIL'-E'-VWD3-C8_3-TIL3-E3 subdomains of which TIL'-E'-VWD3 comprises the main factor VIII (FVIII)-binding region. Yet, von Willebrand disease (VWD) Type 2 Normandy (2N) mutations, associated with impaired FVIII interaction, have been identified in C8_3-TIL3-E3. We now assessed the role of the VWF (sub)domains for FVIII binding using isolated D', D3 and monomeric C-terminal subdomain truncation variants of D'-D3. Competitive binding assays and surface plasmon resonance analysis revealed that D' requires the presence of D3 for effective interaction with FVIII. The isolated D3 domain, however, did not show any FVIII binding. Results indicated that the E3 subdomain is dispensable for FVIII binding. Subsequent deletion of the other subdomains from D3 resulted in a progressive decrease in FVIII-binding affinity. Chemical footprinting mass spectrometry suggested increased conformational changes at the N-terminal side of D3 upon subsequent subdomain deletions at the C-terminal side of the D3. A D'-D3 variant with a VWD type 2N mutation in VWD3 (D879N) or C8_3 (C1060R) also revealed conformational changes in D3, which were proportional to a decrease in FVIII-binding affinity. A D'-D3 variant with a putative VWD type 2N mutation in the E3 subdomain (C1225G) showed, however, normal binding. This implies that the designation VWD type 2N is incorrect for this variant. Results together imply that a structurally intact D3 in D'-D3 is indispensable for effective interaction between D' and FVIII explaining why specific mutations in D3 can impair FVIII binding.
血管性血友病因子(VWF)的 D'-D3 片段可分为 TIL'-E'-VWD3-C8_3-TIL3-E3 亚结构域,其中 TIL'-E'-VWD3 包含主要的因子 VIII(FVIII)结合区。然而,与 FVIII 相互作用受损相关的血管性血友病(VWD)2 型诺曼底(2N)突变已在 C8_3-TIL3-E3 中被发现。我们现在使用分离的 D'、D3 和单体 C 末端亚结构域截断变体评估了 VWF(亚)结构域对 FVIII 结合的作用。竞争性结合测定和表面等离子体共振分析表明,D' 有效与 FVIII 相互作用需要 D3 的存在。然而,分离的 D3 结构域没有显示任何 FVIII 结合。结果表明,E3 亚结构域对于 FVIII 结合是可有可无的。随后从 D3 中删除其他亚结构域导致 FVIII 结合亲和力逐渐降低。化学足迹质谱法表明,在 D3 的 C 末端侧随后的亚结构域缺失后,D3 的 N 末端侧发生了构象变化。在 D3 的 VWD3 (D879N)或 C8_3 (C1060R)处具有 2N 型 VWD 突变的 D'-D3 变体也显示了 D3 中的构象变化,这与 FVIII 结合亲和力的降低成正比。然而,具有 E3 亚结构域假定的 2N 型 VWD 突变的 D'-D3 变体(C1225G)显示出正常结合。这意味着该变体的 2N 型 VWD 指定是不正确的。结果共同表明,D'-D3 中的结构完整的 D3 对于 D' 和 FVIII 之间的有效相互作用是必不可少的,这解释了为什么 D3 中的特定突变会损害 FVIII 结合。