Sohn Myungho, Kim Sanggil, Jeong Hyeon Ju, Ko In Young, Moon Ji Wook, Lee Dowon, Oh Junseo
New Drug Development Center, Osong Medical Innovation Foundation, Osong 28160, Republic of Korea.
Department of Biomedical Sciences, College of Medicine, Korea University, Seoul 02841, Republic of Korea.
Int J Mol Sci. 2024 Dec 27;26(1):137. doi: 10.3390/ijms26010137.
The protein therapeutics market, including antibody and fusion proteins, has experienced steady growth over the past decade, underscoring the importance of optimizing amino acid sequences. In our previous study, we developed a fusion protein, R31, which combines retinol-binding protein (RBP) with albumin domains IIIA and IB, linked by a sequence (AAAA), and includes an additional disulfide bond (N227C-V254C) in IIIA. This fusion protein effectively inhibited hepatic stellate cell activation. In this study, we further optimized the sequence. The G176K mutation at the C-terminus of RBP altered the initiation site of the first α-helix in domain IIIA, shifting it from P182 to K176, and promoted polar interactions between K176 and adjacent residues, enhancing the rigidity of the RBP/IIIA interface. The introduction of an additional disulfide bond (V231C/Y250C) connecting helices 3 and 4 in IIIA resulted in a three-fold increase in productivity and a 2 °C improvement in thermal stability compared to R31. Furthermore, combining the G176K mutation with V231C/Y250C further enhanced both productivity and anti-fibrotic activity. These findings suggest that the enhanced stability of domain IIIA, conferred by V231C/Y250C, along with the increased rigidity of the RBP/IIIA interface, optimizes interdomain distance and alignment, facilitating proper protein folding.
包括抗体和融合蛋白在内的蛋白质治疗药物市场在过去十年中经历了稳步增长,这突出了优化氨基酸序列的重要性。在我们之前的研究中,我们开发了一种融合蛋白R31,它将视黄醇结合蛋白(RBP)与白蛋白结构域IIIA和IB结合,通过一个序列(AAAA)连接,并在IIIA中包含一个额外的二硫键(N227C-V254C)。这种融合蛋白有效地抑制了肝星状细胞的激活。在本研究中,我们进一步优化了序列。RBP C末端的G176K突变改变了IIIA结构域中第一个α螺旋的起始位点,从P182转移到K176,并促进了K176与相邻残基之间的极性相互作用,增强了RBP/IIIA界面的刚性。在IIIA中引入一个连接螺旋3和4的额外二硫键(V231C/Y250C),与R31相比,生产力提高了三倍,热稳定性提高了2°C。此外,将G176K突变与V231C/Y250C相结合,进一步提高了生产力和抗纤维化活性。这些发现表明,V231C/Y250C赋予IIIA结构域更高的稳定性,以及RBP/IIIA界面刚性的增加,优化了结构域间的距离和排列,促进了蛋白质的正确折叠。