ClpB chaperone as a promising target for antimicrobial therapy: A narrative review

✅ 全文

ClpB分子伴侣作为抗菌治疗有前景靶点的叙述性综述

作者 Sachini J. Udari; Sayoka Shamodhi; Rumesh M. Nelumdeniya; Udayana Ranatunga; S. P. N. N. Senadeera; C. B. Ranaweera 期刊 Asian Pacific Journal of Tropical Biomedicine 发表日期 2024 ISSN 2221-1691 DOI 10.4103/apjtb.apjtb_590_24 类型 原创研究 (Original Research)

📄 英文摘要 English Abstract

EN

The Clp/Hsp100 family, part of the ATPase associated with various cellular activities (AAA+) superfamily, includes caseinolytic peptidase B (ClpB), a highly conserved protein found in bacteria, fungi, protozoa, and plants. Notably, ClpB is present in all ESKAPE pathogens: Enterococcus faecium, Staphylococcus aureus, Klebsiella pneumoniae, Acinetobacter baumannii, Pseudomonas aeruginosa , and Enterobacter spp. ClpB plays a crucial role in reactivating and disaggregating proteins, enabling pathogens to survive under host-induced stress and conferring thermotolerance to bacterial cells. Infections caused by ESKAPE pathogens are particularly challenging due to their resistance to broad-spectrum antibiotics and biofilm formation, posing a significant global health threat as they are often multidrug-resistant, extensively drug-resistant, and pan-drug-resistant. Given its absence in human cells and its essential role in bacterial survival under stress, ClpB is a promising target for antimicrobial therapy. Targeting Hsp100 family proteins could lead to the development of novel antifungal and antiprotozoal treatments. This review explores the function of ClpB in the survival of ESKAPE pathogens and the protozoan Plasmodium falciparum . Relevant research findings were compiled using academic databases, and data analysis was performed using Clustal Omega Multiple Sequence Alignment and Boxshade tools.

📄 中文摘要 Chinese Abstract

中文
分子伴侣是一类蛋白质,能够促进其他蛋白质的稳定和正确折叠,使其形成功能性天然构象,而自身不成为最终结构的一部分。从核糖体上新合成的多肽在细胞环境中偶尔会发生错误折叠,但大多数能够正确折叠为具有活性的天然状态。同样,某些蛋白质在应激条件下也可能发生错误折叠并失去其天然构象。分子伴侣在逆转蛋白质聚集方面发挥着关键作用,通过解聚蛋白质聚集体并将其转化为非结构化多肽来实现。这些释放的多肽随后在其他分子伴侣的辅助下重新折叠为具有活性的天然结构,或被细胞蛋白酶体机制靶向降解。 细菌中的酪蛋白水解肽酶B(ClpB)、植物中的Hsp101和酵母中的Hsp104均属于Hsp100家族分子伴侣,因其单体的分子量约为100 kDa。ClpB的生理活性形式为六聚体,分子量约为575 kDa,由六个ClpB单体组成,每个单体约为95 kDa。这些单体在三磷酸腺苷(ATP)或二磷酸腺苷(ADP)等核苷酸存在下组装。在组装过程中,六聚体内部形成一个狭窄的中央通道,在ClpB水解ATP的过程中促进从聚集体中提取的多肽的移动。 当细菌、病毒、真菌和寄生虫发生基因变化使其对抗微生物药物不再产生反应时,就会出现抗微生物药物耐药性。这一现象使感染的治疗更加困难,增加了疾病传播的风险,导致更严重的疾病,甚至可能引发死亡。抗微生物药物耐药性的迅速升级,加上感染性细菌的激增,凸显了开发新型抗微生物药物和确定新型抗微生物靶点的迫切需求。

📋 英文结构化总结 English Structured Summary

全文整理

EN

Background:

Molecular chaperones are a class of proteins that facilitate the stabilization and proper folding of other proteins into their functional native conformations without becoming part of the final structure. Newly synthesized polypeptides emerging from ribosomes may occasionally misfold in the cellular environment, though most correctly fold into their active native states. Similarly, some proteins may also misfold and lose their native conformations under stress conditions. Molecular chaperones play a crucial role in reversing protein aggregation by disassembling protein aggregates and converting them into unstructured polypeptides. These released polypeptides can subsequently refold into their active native structures with the assistance of additional molecular chaperones or be targeted for degradation by the cellular protease machinery.

Caseinolytic Peptidase B (ClpB) in bacteria, Hsp101 in plants, and Hsp104 in yeast are molecular chaperones classified under the Hsp100 family due to their monomeric molecular weight of approximately 100 kDa. The physiologically active form of ClpB exists as a hexamer, with an approximate molecular weight of 575 kDa, comprising six ClpB monomers, each around 95 kDa. These monomers assemble in the presence of nucleotides such as Adenosine triphosphate (ATP) or Adenosine diphosphate (ADP). During the assembly, a narrow central channel is formed within the hexamer, facilitating the movement of extracted polypeptides from aggregates as ClpB hydrolyzes ATP.

Antimicrobial resistance arises when bacteria, viruses, fungi, and parasites undergo genetic changes that render them unresponsive to antimicrobial agents. This phenomenon makes infections more challenging to treat, increases the risk of disease transmission, leads to more severe illnesses, and can result in fatalities. The rapid escalation of antimicrobial resistance, coupled with the surge in infectious bacteria, emphasizes the urgent need to develop new antimicrobials and identify novel antimicrobial targets.

Methods:

This study utilized computational methods to evaluate guanidine hydrochloride (GuHCl), D-arginine, and L-arginine as potential ClpB inhibitors. Molecular docking studies using AutoDock Vina against ClpB (PDB ID: 1QVR) identified possible binding poses of the ligands. SwissADME predicted drug-likeness and pharmacokinetics, while ProTox-II assessed toxicity. Molecular dynamics (MD) simulation trajectories for 100 ns revealed stable root-mean-square deviation (RMSD) and consistent hydrogen bond formation. Binding free energy calculations using MM/PBSA confirmed favourable interactions.

Results:

Both D-Arginine and L-Arginine showed a binding affinity of -5.9 kcal/mol to Nucleotide Binding Domain-1 (NBD-1), while GuHCl exhibited a binding affinity of -3.5 kcal/mol to Nucleotide Binding Domain-2 (NBD-2). The formation of favorable conventional hydrogen bonds between the protein and the ligands primarily contributed to the observed binding affinities in the docking results. MD simulation trajectories for 100 ns revealed stable RMSD and consistent hydrogen bond formation, indicating stable ligand binding. However, solvent-accessible surface area (SASA), radius of gyration (Rg), and root-mean-square fluctuation (RMSF) analyses revealed minor alterations in the NBD-1 domain. These structural changes align with experimental findings for GuHCl, suggesting impaired ClpB activity.

Data Summary:

Both D-Arginine and L-Arginine showed a binding affinity of -5.9 kcal/mol to NBD-1, while GuHCl exhibited a binding affinity of -3.5 kcal/mol to NBD-2. In silico toxicity predictions classified GuHCl as slightly toxic (Class IV; LD50: 350 mg/kg) and L-arginine and D-arginine as possibly harmful (Class V; LD50: 245,050 mg/kg). None of the ligands violated Lipinski’s rule, indicating their suitability for oral administration. Binding free energy calculations using MM/PBSA confirmed favourable interactions, with all test molecules showing negative free energy values.

Conclusions:

These findings suggest that GuHCl, L-arginine, and D-arginine have potential as ClpB inhibitors, warranting further in vitro and in vivo validation for antimicrobial therapy.

Practical Significance:

Targeting ClpB can offer promising strategies for antimicrobial therapies, as ClpB is a bacterial chaperone absent in human cells and plays a crucial role in bacterial survival under stress. The computational results support the test ligands' potential as safe ClpB inhibitors, with good oral bioavailability and low toxicity, prompting further in vitro and in vivo validation for novel antimicrobial strategies.

📋 中文结构化总结 Chinese Structured Summary

中文

背景:

分子伴侣是一类蛋白质,能够促进其他蛋白质的稳定和正确折叠,使其形成功能性天然构象,而自身不成为最终结构的一部分。从核糖体上新合成的多肽在细胞环境中偶尔会发生错误折叠,但大多数能够正确折叠为具有活性的天然状态。同样,某些蛋白质在应激条件下也可能发生错误折叠并失去其天然构象。分子伴侣在逆转蛋白质聚集方面发挥着关键作用,通过解聚蛋白质聚集体并将其转化为非结构化多肽来实现。这些释放的多肽随后在其他分子伴侣的辅助下重新折叠为具有活性的天然结构,或被细胞蛋白酶体机制靶向降解。

细菌中的酪蛋白水解肽酶B(ClpB)、植物中的Hsp101和酵母中的Hsp104均属于Hsp100家族分子伴侣,因其单体的分子量约为100 kDa。ClpB的生理活性形式为六聚体,分子量约为575 kDa,由六个ClpB单体组成,每个单体约为95 kDa。这些单体在三磷酸腺苷(ATP)或二磷酸腺苷(ADP)等核苷酸存在下组装。在组装过程中,六聚体内部形成一个狭窄的中央通道,在ClpB水解ATP的过程中促进从聚集体中提取的多肽的移动。

当细菌、病毒、真菌和寄生虫发生基因变化使其对抗微生物药物不再产生反应时,就会出现抗微生物药物耐药性。这一现象使感染的治疗更加困难,增加了疾病传播的风险,导致更严重的疾病,甚至可能引发死亡。抗微生物药物耐药性的迅速升级,加上感染性细菌的激增,凸显了开发新型抗微生物药物和确定新型抗微生物靶点的迫切需求。

方法:

本研究采用计算方法评估盐酸胍(GuHCl)、D-精氨酸和L-精氨酸作为潜在ClpB抑制剂的效果。使用AutoDock Vina对ClpB(PDB ID: 1QVR)进行分子对接研究,确定了配体的可能结合构象。SwissADME预测了类药性和药代动力学特性,ProTox-II评估了毒性。100纳秒(ns)的分子动力学(MD)模拟轨迹显示了稳定的均方根偏差(RMSD)和持续的氢键形成。使用MM/PBSA计算结合自由能,证实了有利的相互作用。

结果:

D-精氨酸和L-精氨酸对核苷酸结合结构域-1(NBD-1)的结合亲和力为-5.9 kcal/mol,而GuHCl对核苷酸结合结构域-2(NBD-2)的结合亲和力为-3.5 kcal/mol。蛋白质与配体之间有利的经典氢键的形成是对接中所观察到结合亲和力的主要贡献因素。100 ns的MD模拟轨迹显示了稳定的RMSD和持续的氢键形成,表明配体结合稳定。然而,溶剂可及表面积(SASA)、回转半径(Rg)和均方根涨落(RMSF)分析揭示了NBD-1结构域的细微结构变化。这些结构变化与GuHCl的实验结果一致,提示ClpB活性受到损害。

数据总结:

D-精氨酸和L-精氨酸对NBD-1的结合亲和力为-5.9 kcal/mol,GuHCl对NBD-2的结合亲和力为-3.5 kcal/mol。计算机毒性预测将GuHCl归类为轻度毒性(IV类;LD50:350 mg/kg),L-精氨酸和D-精氨酸归类为可能有害(V类;LD50:245,050 mg/kg)。所有配体均未违反Lipinski规则,表明其适合口服给药。使用MM/PBSA计算的结合自由能证实了有利的相互作用,所有测试分子均显示负的自由能值。

结论:

这些发现表明,GuHCl、L-精氨酸和D-精氨酸具有作为ClpB抑制剂的潜力,值得进一步进行体外和体内验证以用于抗微生物治疗。

实际意义:

靶向ClpB可为抗微生物治疗提供有前景的策略,因为ClpB是一种不存在于人类细胞中的细菌伴侣蛋白,在细菌应激生存中起着关键作用。计算结果支持测试配体作为安全ClpB抑制剂的潜力,具有良好的口服生物利用度和低毒性,值得进一步进行体外和体内验证,以开发新型抗微生物策略。

📖 英文全文 English Full Text

EN

Ceylon Journal of Science 54 (4) 2025: 963-977 RESEARCH ARTICLE

Computational Assessment of Guanidine and Arginine Isomers as Inhibitors of Caseinolytic Peptidase B (ClpB): Targeting Bacterial Chaperones for Novel Antimicrobial Strategies R. S. J. Udari, H. M. S. Shamodhi, N. R. M. Nelumdeniya, R. J. K. U. Ranatunga, S. P. N. N. Senadeera and C. B. Ranaweera

Highlights • The inhibitory potential of GuHCl, L-arginine, and D-arginine against bacterial ClpB protein was evaluated using computational approaches. • Report the stable binding supported by H-bonds and affinities from docking and MD simulations. • ADMET analysis shows good oral bioavailability of test ligands with no violations of Lipinski’s Rule of Five. • Low toxicity and acceptable LD₅₀ values supported the test ligand’s potential as safe ClpB inhibitors. • Findings suggested that targeting ClpB can offer promising strategies for antimicrobial therapies, warranting further in vitro and in vivo validation.

Ceylon Journal of Science 54 (4) 2025: 963-977 DOI: https://doi.org/10.4038/cjs.v54i4.8675 RESEARCH ARTICLE

Computational Assessment of Guanidine and Arginine Isomers as Inhibitors of Caseinolytic Peptidase B (ClpB): Targeting Bacterial Chaperones for Novel Antimicrobial Strategies R. S. J. Udari1, H. M. S. Shamodhi1, N. R. M. Nelumdeniya2, R. J. K. U. Ranatunga3, S. P. N. N. Senadeera4 and C. B. Ranaweera1 Department of Medical Laboratory Sciences, Faculty of Allied Health Sciences, General Sir John Kotelawala Defense University, Werahera, 10718, Sri Lanka 2 Department of Pharmacy, Faculty of Allied Health Sciences, General Sir John Kotelawala Defense University, Werahera, 10718, Sri Lanka 3 Department of Chemistry, Faculty of Science, University of Peradeniya, Peradeniya, 20400, Sri Lanka 4 Department of Zoology and Environmental Sciences, Faculty of Science, University of Colombo, Colombo 03, 00300, Sri Lanka 1

Abstract: Bacterial infections pose a significant global health threat due to their resistance to broad-spectrum antimicrobials, necessitating novel therapeutic strategies. Caseinolytic Peptidase B (ClpB), a bacterial chaperone absent in human cells, plays a crucial role in bacterial survival under stress by disaggregating protein aggregates, making it a promising antimicrobial target. This study utilized computational methods to evaluate guanidine hydrochloride (GuHCl), D-arginine, and L-arginine as potential ClpB inhibitors. Molecular docking studies using AutoDock Vina against ClpB (PDB ID: 1QVR) identified possible binding poses of the ligands. Both D-Arginine and L-Arginine showed a binding affinity of -5.9 kcal/mol to Nucleotide Binding Domain-1 (NBD-1), while GuHCl exhibited a binding affinity of -3.5 kcal/ mol to Nucleotide Binding Domain-2 (NBD-2). The formation of favorable conventional hydrogen bonds between the protein and the ligands primarily contributed to the observed binding affinities in the docking results. SwissADME predicted drug-likeness and pharmacokinetics, while ProTox-II assessed toxicity. None of the ligands violated Lipinski’s rule, indicating their suitability for oral administration. In silico toxicity, predictions classified GuHCl as slightly toxic (Class IV; LD50: 350 mg/kg) and L-arginine and D-arginine as possibly harmful (Class V; LD50: 245,050 mg/ kg), yet overall reflecting a favourable safety profile. Molecular dynamics (MD) simulation trajectories for 100 ns revealed stable root-mean-square deviation (RMSD) and consistent hydrogen bond formation, indicating stable ligand binding. However, solvent-accessible surface area (SASA), radius of gyration (Rg), and root-mean-square fluctuation (RMSF) analyses revealed minor alterations in the NBD-1 domain. These structural changes align with experimental findings for GuHCl, suggesting impaired ClpB activity. Binding free energy calculations using MM/ PBSA confirmed favourable interactions, with all test molecules showing negative free energy values. These findings suggest that GuHCl, L-arginine, and D-arginine have potential as ClpB inhibitors, warranting further in vitro and in vivo validation for antimicrobial therapy. Keywords: ClpB; GuHCl; L-Arginine and D-Arginine; Molecular docking; Molecular dynamics

INTRODUCTION Molecular chaperones are a class of proteins that facilitate the stabilization and proper folding of other proteins into their functional native conformations without becoming part of the final structure (Doyle et al., 2012). Newly synthesized polypeptides emerging from ribosomes may occasionally misfold in the cellular environment, though most correctly fold into their active native states. Similarly, some proteins may also misfold and lose their native conformations under stress conditions. Molecular chaperones play a crucial role in reversing protein aggregation by disassembling protein aggregates and converting them into unstructured polypeptides. These released polypeptides can subsequently refold into their active native structures with the assistance of additional molecular chaperones or be targeted for degradation by the cellular protease machinery (Doyle et al., 2012; Ranaweera, 2021). Caseinolytic Peptidase B (ClpB) in bacteria, Hsp101 in plants, and Hsp104 in yeast are molecular chaperones classified under the Hsp100 family due to their monomeric molecular weight of approximately 100 kDa (kiloDaltons) (Doyle et al., 2012; Ranaweera, 2021). The physiologically active form of ClpB exists as a hexamer, with an approximate molecular weight of 575 kDa, comprising six ClpB monomers, each around 95 kDa. These monomers assemble in the presence of nucleotides such as Adenosine triphosphate (ATP) or Adenosine diphosphate (ADP). During the assembly, a narrow central channel is formed within the hexamer, facilitating the movement of extracted polypeptides from aggregates as ClpB hydrolyzes ATP (Zolkiewski et al., 2012). The Escherichia coli ClpB monomer has 857 amino acids in it. The first 146 amino acids (AA) comprise the N-terminal domain, which is joined to the Nucleotide Binding Domain-1 (NBD-1) by the conserved linker starting from 147 AA to 163 AA. NBD-1 starts at 164 AA *Corresponding Author’s Email: cbr2704@kdu.ac.lk https://orcid.org/0000-0003-4173-0547

This article is published under the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

964 and ends at 413 AA by joining to the middle (M) domain (414 AA to 525 AA long), which resides between NBD-1 and Nucleotide Binding Domain-2 (NBD-2). NBD-2 starts from 526 AA and ends at 769 AA by connecting to the last 87 amino acids, which make up the C-terminal (Figure 1) (Ranaweera, 2021). Antimicrobial resistance arises when bacteria, viruses, fungi, and parasites undergo genetic changes that render them unresponsive to antimicrobial agents. This phenomenon makes infections more challenging to treat, increases the risk of disease transmission, leads to more severe illnesses, and can result in fatalities (Antimicrobial Resistance, 2021; Capozzi et al., 2019). The rapid escalation of antimicrobial resistance, coupled with the surge in infectious bacteria, emphasizes the urgent need to develop new antimicrobials and identify novel antimicrobial targets. In a laboratory setting, a target is considered an antimicrobial target if it meets the following criteria: it must be essential for the microorganism’s survival, be widely distributed among the target organisms of concern, ideally have no homologs in humans or other eukaryotes, and be druggable, meaning it can interact with drugs in vivo and be inhibited by small molecules or biotherapeutics. (Alksne & Dunman, 2008). Interestingly, metazoan proteomes lack Hsp100 chaperones, which are exclusively found in bacteria, protozoa, fungi, and plants (Ranaweera et al., 2018). ClpB, has no orthologs in higher eukaryotes, including mammals and humans. Consequently, ClpB represents an attractive target for the development of novel antibiotics, as its inhibition could selectively disrupt microbial function without adversely affecting mammalian cells (Glaza et al., 2021; Kędzierska-Mieszkowska & Zolkiewski, 2021). Organisms that possess ClpB include clinically significant pathogens such as Staphylococcus aureus, Klebsiella pneumoniae, Pseudomonas aeruginosa, Enterococcus faecium, Acinetobacter baumannii, and Enterobacter spp. These are collectively known as the ESKAPE pathogens.

Ceylon Journal of Science 54 (4) 2025: 963-977 In these bacteria, ClpB is essential for survival, replication, and pathogenicity, highlighting its critical role in their stress response and virulence mechanisms (Udari et al., 2025). ClpB is also found in other obligate intracellular bacteria and protozoan pathogens, such as Anaplasma phagocytophilum and the malaria-causing Plasmodium falciparum (Ranaweera et al., 2024). In vivo and in vitro data demonstrate that ClpB is druggable by Guanidine hydrochloride (GuHCl) (Ranaweera, 2021). Studies on the ClpB 4HSE structure, including a crystallized Gdm+ ion with ADP, reveal that GuHCl can bind to and inhibit Hsp104/ClpB through two mechanisms. First, GuHCl disrupts the interaction between DnaK cochaperone and the M-domain, a crucial component for ClpB activity, thus stabilizing Hsp104/ClpB in a suppressed conformation by reinforcing the M-domain/NBD-1 link. Second, GuHCl inhibits the continuous ATP turnover by NBD-1 (Kummer et al., 2013). In yeast, GuHCl cures prions by inhibiting Hsp100 chaperones. This inhibition occurs because the Guanidinium ion (Gdm+) of GuHCl interacts with the bound nucleotide and conserved GLU, affecting nucleotide binding affinities and interfering with essential GLU required for ATP hydrolysis, by specifically binding to the N-terminal nucleotide-binding regions (Zeymer et al., 2013). For many years, drug discovery efforts have relied heavily on computer-aided drug design (CADD), significantly reducing the time and cost required to plan, execute, and test experiments in the laboratory (Garg et al., 2020). By utilizing in silico methods, such as molecular docking and molecular dynamics, researchers can predict the binding affinities and stabilities of potential drug candidates with target proteins. Additionally, in silico absorption, distribution, metabolism, excretion, and toxicity (ADMET) analysis helps identify the most promising compounds for further studies (Tabani, 2023).

Figure 1: The crystal structure of ClpB (PDB ID:1QVR). The N-terminal domain is shown in light blue, the linker in magenta, the NBD-1 in blue, the M-domain in red, the NBD-2 in yellow, and the C-terminal domain in light green. The figure was generated using structural data from the Protein Data Bank (PDB ID: 1QVR; Lee et al., 2003) and adapted with reference to Ranaweera (2021).

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Udari et al. This study aimed to evaluate inhibitor molecules targeting bacterial ClpB using an in-silico approach. GuHCl was selected based on previous in vivo and in vitro findings demonstrating its ability to bind and inhibit ClpB, as discussed previously. Due to the structural similarity of the guanidine group in L-arginine and D-arginine to the guanidinium ion (Gdm⁺) in GuHCl, both enantiomers were also considered as candidate ligands. The selected molecules were assessed for their binding potential and pharmacokinetic properties. Computational analyses, including molecular docking and molecular dynamics simulations, were conducted to gain insights into the strength and stability of protein-ligand interactions. MATERIALS AND METHODS Preparation of the protein structure The X-ray diffractive crystallographic structure of ClpB (PDB ID: 1QVR; resolution of 3.00 Å), derived from Thermus thermophilus (Lee et al., 2003), available in the RCSB Protein Data Bank (PDB) (http://rscb.org) was used in this study. Prior to docking, the target protein was cleaned and refined by removing trapped water molecules and cocrystalized ligands. Then, they were screened for missing amino acids. The FASTA sequence (amino acid sequence) of the structure reveals that each monomer has a sequence length of 854 amino acid residues. Still, the PDB format structure consists of 803 amino acid residues, leaving 51 amino acids missing in each monomer. Modeller 10.4 tool was used to fill the missing amino acid residues of the protein structure (Šali & Blundell, 1993). After fixing the missing amino acid residues, the structure was refined using the ModRefiner algorithm (Xu & Zhang, 2011) available at https://zhanggroup.org/ModRefiner/. This algorithm minimizes the atomic-level energy of the protein through a two-step process. First, it utilizes c-alpha traces and minimizes the main chain energy in a lowresolution step. Subsequently, it minimizes the energy of the entire atomic structure by incorporating initial side chain atoms at a high resolution and higher speed. At

the end of the process, the algorithm calculates the Root Mean Square Deviation (RMSD) and Template Modelling score (TM-score) for the refined structure to compare and validate the resulting refined model. A Ramachandran plot (Ramachandran & Sasisekharan, 1968) was generated for the refined complete monomer of 1QVR to evaluate the protein backbone conformation using the BIOVIA Discovery Studio v21.1.0.20298 package (Systèmes, 2020). For structural validation, the native and modeled monomeric structures of 1QVR were superimposed using the Visualization Molecular Dynamics (VMD) software package, allowing a comparative analysis of structural deviations. Retrieval of ligand structures PubChem (https://pubchem.ncbi.nlm.nih.gov/) database was used to obtain the 3D structures of the ligands in a structure data file (.sdf) format. Gdm+ ion of GuHCl, L-arginine, and D-arginine were used as test molecules. ADP, known to co-crystallize with ClpB in the 4HSE, PDB structure, was used as a reference ligand (Zeymer et al., 2013). The canonical simplified molecular-input line-entry system (SMILES) of the testing ligands was used for the insilico ADMET and toxicity analysis. Molecular Docking AutoDockTools 1.5.7 (Goodsell & Olson, 1990) was used to generate Protein Data Bank, Partial Charge (Q), & Atom Type (T) / PDBQT format files of the ClpB PDB structure while introducing hydrogens and partial atomic charges (Kollman charges). Energy minimization of the ligands was carried out using the option available on PyRx 0.8 (Dallakyan & Olson, 2015) using the MMFF94 force field. After the energy minimization of each ligand, they were saved separately in pdb, pdbqt, and mol2 formats for docking and MD studies. Docking was continued using the PyRx open-source platform using AutoDock Vina (Trott & Olson, 2010). The grid box was defined to cover the whole protein. X× Y × Z sizes to 111.6939 Å × 124.4924 Å × 74.0728 Å, and center grid coordinates X, Y, Z were set to 45.8962, 23.4164, 33.1118, respectively.

Figure 2: Ligand structures investigated in this study. (a) D-arginine, (b) L-arginine, (c) Adenosine diphosphate (ADP), and (d) Guanidine Hydrochloride (GuHCl) 966 Ceylon Journal of Science 54 (4) 2025: 963-977

The binding poses produced by the docking process were further analyzed, and their docking scores and interactions with the key amino acids of protein were observed using BIOVIA Discovery Studio v21.1.0.20298 package (Systèmes, 2020).

Gcomplex is the energy of the protein-ligand complex, whereas Gprotein and Gligand represent the energy of the protein and ligand in an aqueous solvent, respectively. The free energy is calculated using the following equation for each of the terms in the equation.

Physicochemical, ADME, analysis, and drug-likeness and toxicity prediction Gx=Ebonded +(Evdw +Eelec)+Gpolar + Gnon-polar

In parallel to the docking study, the SwissADME online web server (Daina et al., 2017) was used for the screening and predicting the physicochemical, pharmacokinetic, and drug-likeness of ligands, while ProTox-3.0 webserver (Banerjee et al., 2018) was used to analyze the toxicity. Molecular dynamics simulation studies After the analysis of docking results, MD simulations were conducted for both free protein and all ligand-protein complexes using the GROMACS-2024 software package (Berendsen et al., 1995) with the CHARMM36 force field (Huang & MacKerell, 2013) to find out the ligand impact on the stability and structural changes of the ClpB protein. The topology file of the clean protein was generated using inbuilt GROMACS commands using CHARMM36 allatom forcefield, while the ligand topology files were created by SwissParam (http://www.swissparam.ch/) online server (Zoete et al., 2011). Subsequently, protein-ligand complex files were made by merging the protein and ligand files. The complex was solvated in a truncated octahedron periodic box (111.01 x 124.01 x 74.91 Å), using a distance of 12 Å between the protein and the boundary of each side using the TIP3P water model, keeping the complex in the middle. Then, the system was neutralized by adding Na+ ions, and an energy minimization step was carried out. The system was equilibrated using NVT and NPT conditions at 300 K and 1 bar pressure, respectively, for 100 ps. Finally, the production run was done for 100 ns, keeping the time step at 2 fs (Bhattarai & Emerson, 2021). In the end, the trajectory files of the free protein and protein-ligand complexes were analyzed by calculating RMSD, Root Mean Square Fluctuation (RMSF), H-bonds, Radius of Gyration (RG), and Solvent-accessible surface area (SASA) with GROMACS inbuilt tools gmx rms, gmx rmsf, gmx hbond, gmx gyrate, and gmx sasa, respectively (Kushwaha et al., 2021). VMD software package (Humphrey et al., 1996) was used to analyze the trajectories, and the plots were produced using GraphPad Prism 8.4.2 software. Total Binding free energy calculations For the calculation of total binding free energy (ΔGbind) of the protein-ligand complexes, the g_mmpbsa package was used with GROMACS (Valdés-Tresanco et al., 2021). The g_mmpbsa uses the Molecular Mechanics/ PoissonBoltzmann Surface Area (MM/PBSA) approach for the ΔGbind calculation of the complexes (Kushwaha et al., 2021). The ΔGbind was calculated for all the protein-ligand complexes using the entire MD simulation trajectories of the 100 ns period. The following equation provides the protein-ligand complex’s ΔGbind in an aqueous solvent. ΔGbind = Gcomplex -(Gprotein +Gligand)

In this equation, Gx can be Gcomplex , Gprotein or Gligand, and Ebonded represents the energy given by bonded contacts within the molecule, which is considered as zero always (Kollman et al., 2000). Evdw represents van der Waals energy, and Eelec represents electrostatic energy. Gpolar, is the energy associated with the electrostatic interactions between the molecule (protein or ligand) and the surrounding solvent. In Gnon-polar represents nonpolar or apolar solvation energy, which is the free energy contribution due to non-electrostatic interactions, such as hydrophobic effects. Gnon-polar is derived as follows, Gnon-polar = γ⋅SASA + b In this equation, SASA stands for solvent-accessible surface area, whereas b is the fitting parameter and γ is a surface tension coefficient. Therefore, the g_mmpbsa tool determines the total binding free energy by computing each energy component of this equation for the protein, ligand and the total complex in an aqueous solvent environment (Sundar et al., 2019). RESULTS AND DISCUSSION This study investigated the ClpB protein using an in silico approach, focusing on its interactions with the guanidinium ion (Gdm⁺) of GuHCl, as well as with L-arginine and D-arginine, using ADP as a control. The research was conducted in three major steps: molecular docking, ADMET analysis, and molecular dynamics (MD) simulation. Molecular docking served as a virtual screening step to identify ligand binding sites and their affinities. ADMET analysis evaluated the pharmacokinetics and toxicity profiles of the test ligands. Finally, the stability of the free protein and the protein-ligand complexes was assessed through MD simulations under predefined conditions. Refining the protein After filling the missing amino acid residues in the monomeric 1QVR structure using Modeller tools, the resulting model required refinement to eliminate artifacts such as steric clashes caused by unnatural overlaps between non-bonded atoms. This refinement can be achieved through short molecular dynamics (MD) simulations, which allow the structure to relax into a more energetically favorable conformation (Adiyaman & McGuffin, 2019). Alternatively, refinement can be performed using validated computational tools such as ModRefiner, which is a fast and efficient algorithm for protein structure optimization. ModRefiner operates in two stages: the first reconstructs a realistic backbone using predefined Cα trace conformations, and the second enhances structural accuracy by adding side-chain atoms and minimizing structural energy using a composite of physics-based and knowledge-based force fields. The quality of the final refined model is assessed

Udari et al. by metrics such as lower RMSD and higher TM-score, which indicate improved folding accuracy and structural realism (Xu & Zhang, 2011). Figure 3 shows the refined structure of 1QVR. RMSD value was within 0-2 Å, indicating reasonably higher structural similarity to the initial modelled structure. The TM-score being closer to 1 indicates higher similarity and accuracy of the folding patterns.

Figure 3: Refined 3D structure of the ClpB monomer (PDB ID: 1QVR) after filling missing amino acid residues using Modeller and optimizing the geometry with ModRefiner. Following optimization, the structure achieved a root mean square deviation (RMSD) of 1.446 Å and a template modeling score (TM-score) of 0.9881

Ramachandran plot The refined model of the 1QVR monomeric structure was validated using a Ramachandran plot and compared with the original 1QVR structure (Figure 4). Ramachandran plot helps us understand the conformational space available to amino acids and shows their theoretically favoured regions based on the backbone dihedral angles (φ and ψ). The rotational angles between the Cα-N and Cα-C bonds of the protein backbone are denoted by φ (phi) and ψ (psi), respectively (Ramachandran & Sasisekharan, 1968). ψ is usually plotted on the y-axis and φ on the x-axis. In both pots triangles represent glycine, squares represent proline, and circles represent all the other amino acids. Three main regions can be identified in these plots. The areas that are margined with light blue are the areas that represent alpha-helical and beta-sheet conformations, which are the permitted conformations where steric conflicts do not occur. The areas within the pink margin are the partially allowed zones representing left-handed alpha-helical conformations, which are uncommon (Ramachandran & Sasisekharan, 1968). Amino acids in these regions are shown in green, with most residues in the refined structure (Figure 4-a) located within sterically favorable zones. The regions not margined with a coloured line indicate sterically forbidden conformations where the atoms in the polypeptide are closer together than the total of their van der Waals radii, except for glycine (red triangles), which lacks a side chain (Ho & Brasseur, 2005). Non-glycine residues in these forbidden regions (red circles) are minimal in the refined structure. Therefore, overall, the Ramachandran plots indicate that the refined 1QVR monomeric structure folds with minimal steric clashes and adopts energetically favorable backbone conformations.

Figure 4: Ramachandran plots of (a) the refined 1QVR monomeric structure and (b) the original 1QVR structure. φ (phi) angles are on the x-axis, and ψ (psi) angles are on the y-axis. Triangles, squares, and circles represent glycine, proline, and other amino acids, respectively. Green symbols denote residues in allowed regions. Red symbols represent residues in sterically disallowed regions, with glycine (red triangles) permitted in these regions due to its unique conformational flexibility. The refined structure (a) demonstrates most residues in favorable conformations, with minimal steric clashes

968 Molecular Docking Gdm+ ion of GuHCl, L-arginine, and D-arginine were taken as test ligands, while ADP was taken as the control ligand for the molecular docking. All ligands were docked against the 1QVR structure using a blind docking approach. As depicted in Figure 5, the crystal structure 4HSE, which includes NBD-1 to M region (residues 141 to 534) with a crystalized Gdm+ ion and ADP. In this structure, Gdm+ ion make hydrogen bonds with GLU 209, ASP 170, and PRO 171 while ADP bonds with THR 205, GLY 201, ALA 206, GLY 203, and ILE 73, respectively (Zeymer et al., 2013). Blind docking was intentionally employed to assess the reliability and accuracy of the docking protocol by evaluating whether ADP would still bind to the same functionally relevant residues observed in the 4HSE structure. Moreover, this approach ensured an unbiased evaluation of the test ligands, allowing them to explore and bind to the most energetically favorable sites in the 1QVR structure without predefined constraints. In docking results with 1QVR (Figure 6), ADP binds to the NBD-1 forming conventional hydrogen bonds with GLN 8, ARG 11, GLU 12, GLU 144, ILE 215, VAL 216, LYS 225, GLY 226, LYS 227. In contrast, the Gdm+ ion binds to the NBD-2 by forming conventional hydrogen bonds with SER 539, LYS 540, GLU 543, and GLU 545 and attractive charge interactions with GLU 545, ASP 614. As mentioned earlier, this Gdm+ ion interacts with negatively charged amino acid GLU, the same as in the 4HSE crystal structure, even though it shows high affinity towards NBD-2 in docking results. Both positively charged guanidine groups in the L-arginine and D-arginine interact with negatively charged GLU in the 1QVR structure NBD-1, as shown

Ceylon Journal of Science 54 (4) 2025: 963-977 in Figure 6 as well. Table 1 shows the binding affinities between all the ligands and the protein and their binding interactions with adjacent residues of the most favourable pose. ADMET results The SwissADME server provides results of analyzed chemicals in three main categories: physicochemical, pharmacokinetic, and drug-likeness. Lipinski’s rule of five (RO5) was considered when analyzing the drug-likeness of a ligand for oral bioavailability. For the compound to be orally bioavailable, the ligand should not have more than one violation of the RO5. To predict that the absorption and penetration is ideal and to be accepted as an orally active ligand, the RO5 is as follows: no more than five H-bond donors, no more than ten H-bond acceptors, a molecular weight (MWT) of less than 500 Daltons and a computed Log P (ClogP) of less than five (Lipinski et al., 2001). GuHCl, L-arginine, and D-arginine showed zero violations thereby considered orally bioavailable. When assessing pharmacokinetic properties, Blood Brain Barrier (BBB), gastrointestinal (GI) absorption, skin permeation coefficient (Log Kp), and substrate for cytochromes (CYPs) isoforms were taken into account. All three ligands showed favourable properties while also having high GI absorption capacities. The toxicity was evaluated using the ProTox-II server. GuHCl was predicted to be slightly toxic (Class IV) with an LD50 of 350 mg/kg, while L arginine and D-arginine were categorized as may be harmful (Class V) with an LD50 of 245050 mg/kg. Even though GuHCl has a lower LD50, it showed inactive results as same as both L and D arginine for hepatotoxicity, cytotoxicity, heat shock factor response

Figure 5: (a)- Crystalized ADP in 4HSE structure, bonding with THR 205, GLY 201, ALA 206, GLY 203, and ILE 73. (b)- Crystalized Gdm+ ion in 4HSE structure, making hydrogen bonds with GLU 209, ASP 170, and PRO 171. Conventional hydrogen bonds are depicted with green dashed lines

969 Udari et al.

Figure 6: The central 3D diagram shows binding sites of Gdm⁺, L-arginine, and D-arginine docked to the ClpB monomer (1QVR). Individual panels display ligand-specific 3D interactions with hydrogen bond donors in magenta and acceptors in green. Corresponding 2D diagrams illustrate ligand–residue interactions, with hydrogen bonds shown as green dashed lines Table 1: Binding affinity and interacting amino acids of the control and test ligands with the 1QVR structure Ligand

Binding Interacting Amino Acids Affinity kcal/ mol Control ADP -8.2

Conventional Hydrogen Bonds – GLN 8, ARG 11, GLU 12, GLU 144, ILE 215, VAL 216, LYS 225, GLY 226, LYS 227 Attractive Charge – GLU 144 Test Ligands Gdm+ ion -3.5 L-arginine -5.9 D-arginine -5.9

Conventional Hydrogen Bonds – SER 539, LYS 540, GLU 543, GLU 545 Attractive Charge – GLU 545, ASP 614 Unfavorable Donor-Donor – ARG 370 Conventional Hydrogen Bonds – GLN 8, ARG 11, GLU 144, LYS 227 Attractive Charge – GLU 12, ASP 159 Conventional Hydrogen Bonds – GLU 12, LEU 38, LYS 40, ASN 151, GLU 154, ARG 228 Unfavorable Positive-Positive/ Unfavorable Donor-Donor – LYS 40, ARG 162

element (HSE), effect on mitochondrial membrane potential (MMP), phosphoprotein tumor Suppressor (p53) activity, and ATPase family AAA domain-containing protein 5 (ATAD5). Therefore, from overall ADMET results, the test ligands have the potential to be modified as drug candidates accordingly.

MD results After completing the docking study, MD simulations were carried out for the free protein and the proteinligand complexes, namely ADP, GuHCl, L-arginine and D-arginine. As mentioned previously, RMSD, RMSF, Hydrogen Bonds, Rg, and SASA calculations were used

970 Ceylon Journal of Science 54 (4) 2025: 963-977 GuHCl L-arginine D-arginine High High High to analyze the dynamic behaviour of the free protein and to check the stability of the protein-ligand complexes throughout the 100 ns period. Root Mean Square Deviation (RMSD) measures the average displacement of atoms in the system over a specified period compared to a reference structure. RMSD is an indicator of the stability of a complex; higher fluctuations suggest lower stability. Initially, RMSD may gradually increase during the simulation, but it typically stabilizes once the complex reaches conformational equilibrium. Root Mean Square Fluctuation (RMSF) evaluates the structural changes in a protein by assessing the variability of specific residues during the simulation. Lower RMSF values indicate more stable residues, providing insights into how the ligand impacts the protein’s structure. This aids in understanding the ligand’s effect on the protein’s overall stability (Opo et al., 2021). According to Figure 7, the free protein displayed stable RMSD starting from around 45 ns to 100 ns timeline averaging an RMSD value of 0.5185 nm where the fluctuations are within 1 Å. Variations in the RMSF were observed at a few positions of the free protein, and they were distributed among the NBD-1, M-domain and NBD2. Significant changes were observed between residues numbers 270-290 (NBD-1), 415-500 (M-domain) and 620-650 (NBD-2), giving a similar RMSF value ranging between 0.75 nm to 0.8 nm. According to the plots in Figure 8, the protein-ADP complex displayed stable RMSD averaging at 0.5826

Table 2: Pharmacokinetic properties (ADME) and Lipinski’s rule of five properties of the test ligands. 0 0 0

nm. Over time, the complex portrayed no significant fluctuations, indicating stability. When considering the RMSF of the protein-ADP complex, no major oscillations were displayed compared to the free protein. The RMSD of GuHCl (Figure 9) displayed stability until 60 ns. From 60-90 ns, severe fluctuations were seen. It was also noted that no significant fluctuation was seen for the last 10 ns of the production run, suggesting that the proteinGuHCl complex might stabilize if the run was continued over 100 ns. Overall, the Protein-GuHCl complex averaged at an RMSD of 0.7355 nm. The RMSD of the protein-L-arginine complex stabilized after 45 ns, with an average RMSD of 0.7771 nm, fluctuating with a difference of around 0.2 nm. Similarly, the D-arginine complex stabilized after 30 ns and remained stable until the end of the simulation, with an average RMSD of 0.6851 nm, fluctuating with a difference of around 0.3 nm. Both complexes demonstrated minimal fluctuations and maintained stability throughout the simulation (Figure 9). As already known, arginine structure consists of a guanidine group. Even though that fact might suggest that arginine and Gdm+ ion should have the same effect on protein stability due to their structural similarity, the RMSD results suggest that both L and D arginine bindings are more stable than the Gdm+ ion binding to the protein. This could be due to other functional groups, such as the carboxyl group in the arginine structure.

Figure 7: RMSD and RMSF profiles of the free ClpB monomer obtained from the 100 ns MD simulation. RMSD stabilized around 45 ns to 100 ns with an average value of 0.5185 nm. RMSF analysis revealed notable flexibility at residues 270–290 (NBD-1), 415–500 (M-domain), and 620–650 (NBD-2), each showing values between 0.75 and 0.8 nm

Udari et al. 971

Figure 8: RMSD and RMSF profiles of the ClpB–ADP complex obtained from the 100 ns MD simulation. The complex maintained an average RMSD of 0.5826 nm and no significant fluctuations over time. RMSF analysis showed no major oscillations compared to the free protein The RMSF of the protein GuHCl and L-arginine-complexes shows similar deviations in the residues across the overall protein structure, and more prominent fluctuations can be observed towards NBD-1 and M-domain. In contrast, the D-arginine complex exhibits no significant changes in the RMSF plot compared to the free protein (Figure 9). Notably, the Gdm⁺ ion binds and remains in the NBD-2 domain, while both L-arginine and D-arginine remain in the NBD-1 domain.

According to the study conducted by Cathleen Zeymer and colleagues using the 4HSE ClpB structure, they have suggested that Gdm+ binding can affect ATP hydrolysis due to binding to the conserved GLU residue present in the NBD-1. This interaction affects ATP binding to NBD1, inducing a conformational change in the domain that leads to inhibitory effects. They have also suggested that this inhibitory change only occurs in NBD-1 and not in NBD-2 in the presence of GuHCl (Zeymer et al., 2013).

972 Ceylon Journal of Science 54 (4) 2025: 963-977

Figure 9: RMSD and RMSF profiles of ClpB in complex with GuHCl, L-arginine, and D-arginine from 100 ns MD simulations. The GuHCl complex was stable until ~60 ns, fluctuated between 60–90 ns, and regained stability in the final 10 ns (average RMSD: 0.7355 nm). L-arginine and D-arginine complexes stabilized approximately after 45 ns and 30 ns, respectively, and remained stable (average RMSDs: 0.7771 nm and 0.6851 nm). RMSF deviations were most pronounced in NBD-1 and the M-domain for GuHCl and L-arginine, while D-arginine showed minimal changes A study conducted by Eva Kummer and colleagues also suggests that Gdm+ ion binds to the NBD-1 and interferes with ClpB function by reducing ATP turnover from NBD1 via making conformational changes there. They also suggest that Gdm+ ion can cause conformational changes in the middle domain, thereby interfering with the DnaK activation of ClpB (Kummer et al., 2013). Thus, this information can be used in our study as a justification for the RMSF obtained for Gdm+ ion, which displayed significant oscillations between residues 160-460 (NBD-1 and middle

domain), even though in contrast to their findings, Gdm+ ion remained in the NBD-2 during whole 100 ns of MD simulation, which was the best binding site suggested by our docking results. Therefore, we suggest that although Gdm+ ion binds to the NBD-2 with more affinity, it may cause conformational changes in NBD-1 and middle domain. Hence, this binding can inhibit ClpB by interfering with ATP hydrolysis in the NBD-1 and disrupting the connection between the middle domain and DnaK, which is essential for activating ClpB, as the experimental data

Udari et al. suggested. Moreover, L-arginine portrays guanidinelike effects because the RMSF results showed similar fluctuations to those of the Gdm+ ion. We can also suggest that it might be a good candidate molecule to target ClpB as the RMSD curve displayed more favourable stability in the binding than the Gdm+ ion. H-bond analysis, Rg, and SASA were also conducted and compared with the free protein to understand more about the interactions of the test ligands. Hydrogen bonds are formed in the vicinity of an electronegative atom, and hydrogen atoms are covalently bound to another electronegative atom. The stability of a protein-ligand complex depends on the number of intermolecular hydrogen bonds (da Fonseca et al., 2023). Hydrogen bond formation within the protein (intraprotein hydrogen bonds) in the presence and

973 absence of the primary ligands and between the ligand and the protein was assessed to evaluate the protein stability and protein-ligand complex stability throughout the 100 ns period of the simulation (Figure 10). After the ligand binding, intraprotein hydrogen bond formation has been favoured with all three test ligands and especially with GuHCl in the first half of the simulation. The region of a protein surface that comes into contact with its surrounding solvent molecules is known as the SASA (Mazola et al., 2015). According to Figure 11, the computed SASA of the entire protein illustrated increasing trends for GuHCl, L-arginine and D-arginine complexes. Furthermore, the calculation of the SASA of the entire protein for GuHCl displayed a relatively similar but higher SASA to that of the free protein. Steeper peeks in SASA of

Figure 10: Protein–ligand hydrogen bond formation of the ClpB monomer during 100 ns MD simulations in the presence of primary ligands (GuHCl, L-arginine, and D-arginine), and intraprotein hydrogen bond formation in the free protein. Ligand binding increased intraprotein hydrogen bond formation for all three ligands, with GuHCl showing the greatest effect

974 Ceylon Journal of Science 54 (4) 2025: 963-977 the entire protein within the first 20 ns were indicated for L-arginine and D-arginine, demonstrating the hydrophobic core of ClpB becoming more accessible to the surrounding aqueous environment. Overall, these observations point toward the fact that there might be a possibility of conformational changes, which leads to an increase in the SASA of the protein with all the primary ligands.

the gap between the terminal of each protein atom and its centre of mass during a certain period. The fewer fluctuations there are, the better the stability of the folded protein structure (Choudhary et al., 2023). Figure 11 represents variations in Rg value over the simulation period and indicates that all the protein-ligand complexes were more extended than the free protein except for GuHCl.

The Rg showed the protein structure’s overall compactness throughout the simulation. It is determined by measuring

Considering the results of both SASA and Rg, decreasing protein compactness (higher Rg) and increased SASA were

Figure 11: SASA profiles of the ClpB monomer during 100 ns molecular dynamics simulations in the presence of primary ligands (GuHCl, L-arginine, and D-arginine) and in the free protein. The protein–ligand complexes showed increasing SASA trends, with GuHCl exhibiting a consistently higher SASA compared to the free protein. Early pronounced peaks in SASA for L-arginine and D-arginine occurred within the first 20 ns

975

Udari et al. observed. In GuHCl, it is noticeable that Rg has decreased even though the SASA remains high compared to the free protein. This could suggest a conformational change causing lower Rg, which may lead to concealed residues of the protein being more accessible, resulting in higher SASA. Total Binding free energy calculations The 100 ns trajectory obtained during MD simulations was used for computing the total binding free energy of the ligand in the complex. The g_mmpbsa tool, which was used with GROMACS, implies that the MM/PBSA method was used to compute the binding energy. For each complex of the ClpB protein with the control ligand ADP and test ligands GuHCl, L-arginine and D-arginine, Evdw, Eelec, Gpolar, Gnonpolar, and △Gbind were calculated using the g_mmpbsa module and are shown in Table 3. Gpolar contributes positively to the total binding free energy, while Evdw, Eelec, Gpolar, Gpolar contribute negatively to the calculation. According to the results depicted in Table 3 and Figure 12, the total binding free energy of all four compounds is below zero, which indicates a good affinity towards ClpB, and it shows that the three test molecules show more affinity towards the protein structure than the control ligand ADP itself.

CONCLUSIONS ClpB, a bacterial molecular chaperone critical for protein homeostasis under stress, represents a compelling target for novel antimicrobial development. This study computationally evaluated GuHCl, L-arginine, and D-arginine as potential inhibitors of ClpB. Docking analyses identified favourable binding sites, and ADMET predictions suggested oral bioavailability, high GI absorption, and safe toxicity profiles. MD simulations studies revealed ligand-induced conformational changes in the NBD-1 and M-domain of ClpB. These changes may impair ATP turnover and disrupt co-chaperone DnaK interactions necessary for aggregate reactivation. Hydrogen bond analysis indicated that ligand binding, favored intraprotein hydrogen bond formation supporting enhanced protein stability and stable complex formation. Post MD binding free energy calculations further confirmed all three test ligands’ stability and favourable interaction with ClpB, exhibiting higher affinity than the control ligand ADP itself. These findings suggest that GuHCl, L-arginine, and D-arginine could serve as potential ClpB inhibitors, offering a novel approach to antimicrobial therapy. The observed conformational changes, particularly in NBD-1 and M-domain, align with inhibitory mechanisms suggested by prior experimental studies, though discrepancies in

Table 3: Binding energy calculations of ADP, GuHCl, L-arginine and D-arginine Ligand Name Evdw (kcal/mol) Eelec (kcal/mol) Gpolar (kcal/mol) Gnon-polar (γSASA + b) (kcal/mol) ADP GuHCl L-arginine D-arginine

-29.95 -3.52 -14.88 -13.12 -49.54 -293.97 -245.04 -279.22 65.73 278.14 237.90 256.38 -3.44 -1.30 -2.36 -2.56 Figure 12: The binding energy of ADP, GuHCl, L-arginine and D-arginine △Gbind (kcal/mol) -17.20 -20.65 -24.39 -38.52

976 binding site preferences warrant further investigation. Future in vitro and in vivo studies are essential to validate these findings and explore their therapeutic potential. DECLARATION OF CONFLICT OF INTEREST The authors declare no conflicts of interest

📖 中文全文 Chinese Full Text

中文

# 胍和精氨酸异构体作为酪蛋白水解肽酶B(ClpB)抑制剂的计算评估:靶向细菌分子伴侣的新型抗菌策略

R. S. J. Udari, H. M. S. Shamodhi, N. R. M. Nelumdeniya, R. J. K. U. Ranatunga, S. P. N. N. Senadeera 和 C. B. Ranaweera

## 亮点

• 利用计算方法评估了盐酸胍(GuHCl)、L-精氨酸和D-精氨酸对细菌ClpB蛋白的抑制潜力。

• 报告了由氢键和分子对接及分子动力学模拟所支持的稳定结合。

• ADMET分析显示测试配体具有良好的口服生物利用度,且未违反利平斯基五规则。

• 低毒性和可接受的LD₅₀值支持了测试配体作为安全ClpB抑制剂的潜力。

• 研究结果表明,靶向ClpB可为抗菌治疗提供有前景的策略,值得进一步的体外和体内验证。

## 摘要

细菌感染因其对广谱抗菌药物的耐药性而构成重大全球健康威胁,亟需新型治疗策略。酪蛋白水解肽酶B(ClpB)是一种不存在于人体细胞中的细菌分子伴侣,通过解聚蛋白质聚集体在细菌应激存活中发挥关键作用,使其成为有前景的抗菌靶点。本研究利用计算方法评估了盐酸胍(GuHCl)、D-精氨酸和L-精氨酸作为潜在ClpB抑制剂的效果。使用AutoDock Vina对ClpB(PDB ID: 1QVR)进行分子对接研究,确定了配体的可能结合构象。D-精氨酸和L-精氨酸对核苷酸结合结构域-1(NBD-1)的结合亲和力均为-5.9 kcal/mol,而GuHCl对核苷酸结合结构域-2(NBD-2)的结合亲和力为-3.5 kcal/mol。蛋白质与配体之间形成的有利常规氢键是对接结果中观察到的结合亲和力的主要贡献因素。SwissADME预测了药物相似性和药代动力学特性,ProTox-II评估了毒性。所有配体均未违反利平斯基规则,表明其适合口服给药。计算机毒性预测将GuHCl归类为轻度毒性(IV类;LD₅₀:350 mg/kg),L-精氨酸和D-精氨酸归类为可能有害(V类;LD₅₀:245,050 mg/kg),但总体反映出良好的安全性特征。100 ns的分子动力学(MD)模拟轨迹显示了稳定的均方根偏差(RMSD)和持续的氢键形成,表明配体结合稳定。然而,溶剂可及表面积(SASA)、回转半径(Rg)和均方根波动(RMSF)分析揭示了NBD-1结构域的细微结构变化。这些结构变化与GuHCl的实验发现一致,提示ClpB活性受损。使用MM/PBSA进行的结合自由能计算证实了有利的相互作用,所有测试分子均显示负自由能值。这些发现表明,GuHCl、L-精氨酸和D-精氨酸具有作为ClpB抑制剂的潜力,值得进一步进行体外和体内验证以用于抗菌治疗。

**关键词:** ClpB;GuHCl;L-精氨酸和D-精氨酸;分子对接;分子动力学

## 引言

分子伴侣是一类蛋白质,能够促进其他蛋白质稳定并正确折叠为其功能性天然构象,而不会成为最终结构的一部分(Doyle等,2012)。从核糖体新合成的多肽在细胞环境中偶尔会发生错误折叠,尽管大多数能够正确折叠为有活性的天然状态。同样,某些蛋白质在应激条件下也可能发生错误折叠并丧失其天然构象。分子伴侣通过解聚蛋白质聚集体并将其转化为非结构化多肽,在逆转蛋白质聚集过程中发挥关键作用。这些释放的多肽随后可在其他分子伴侣的辅助下重新折叠为有活性的天然结构,或被细胞蛋白酶机制靶向降解(Doyle等,2012;Ranaweera,2021)。

细菌中的酪蛋白水解肽酶B(ClpB)、植物中的Hsp101和酵母中的Hsp104均属于Hsp100家族分子伴侣,因其单体分子量约为100 kDa(千道尔顿)而被归入此类(Doyle等,2012;Ranaweera,2021)。ClpB的生理活性形式为六聚体,分子量约为575 kDa,由六个约95 kDa的ClpB单体组成。这些单体在三磷酸腺苷(ATP)或二磷酸腺苷(ADP)等核苷酸存在下组装。在组装过程中,六聚体内部形成一个狭窄的中央通道,在ClpB水解ATP时促进从聚集体中提取的多肽移动(Zolkiewski等,2012)。

大肠杆菌ClpB单体含有857个氨基酸。前146个氨基酸(AA)组成N端结构域,通过从147 AA到163 AA的保守连接区与核苷酸结合结构域-1(NBD-1)相连。NBD-1从164 AA开始,到413 AA结束,连接至中间(M)结构域(414 AA至525 AA),M结构域位于NBD-1和核苷酸结合结构域-2(NBD-2)之间。NBD-2从526 AA开始,到769 AA结束,连接至最后87个氨基酸,构成C端(图1)(Ranaweera,2021)。

抗菌药物耐药性产生于细菌、病毒、真菌和寄生虫发生基因变化,使其对抗菌药物不再敏感。这一现象使感染更难治疗,增加了疾病传播风险,导致更严重的疾病,并可能造成死亡(Antimicrobial Resistance,2021;Capozzi等,2019)。抗菌药物耐药性的快速升级,加上感染性细菌的激增,强调了开发新型抗菌药物和识别新型抗菌靶点的迫切需求。在实验室环境中,一个靶点要被视为抗菌靶点,必须满足以下标准:它必须对微生物的生存至关重要,在目标生物中广泛分布,理想情况下在人类或其他真核生物中没有同源物,并且具有可药性,即能够与体内药物相互作用并被小分子或生物治疗剂抑制(Alksne & Dunman,2008)。

有趣的是,后生动物蛋白质组缺乏Hsp100分子伴侣,该伴侣仅存在于细菌、原生动物、真菌和植物中(Ranaweera等,2018)。ClpB在高等真核生物(包括哺乳动物和人类)中没有直系同源物。因此,ClpB代表了开发新型抗生素的有吸引力的靶点,因为其抑制可选择性地破坏微生物功能,而不会对哺乳动物细胞产生不利影响(Glaza等,2021;Kędzierska-Mieszkowska & Zolkiewski,2021)。

拥有ClpB的生物包括临床重要的病原体,如金黄色葡萄球菌(Staphylococcus aureus)、肺炎克雷伯菌(Klebsiella pneumoniae)、铜绿假单胞菌(Pseudomonas aeruginosa)、屎肠球菌(Enterococcus faecium)、鲍曼不动杆菌(Acinetobacter baumannii)和肠杆菌属(Enterobacter spp.)。这些统称为ESKAPE病原体。在这些细菌中,ClpB对生存、复制和致病性至关重要,突出了其在应激反应和毒力机制中的关键作用(Udari等,2025)。ClpB也存在于其他专性细胞内细菌和原生动物病原体中,如嗜吞噬细胞无形体(Anaplasma phagocytophilum)和引起疟疾的恶性疟原虫(Plasmodium falciparum)(Ranaweera等,2024)。

体内和体外数据证明,ClpB可被盐酸胍(GuHCl)靶向(Ranaweera,2021)。对ClpB 4HSE结构的研究,包括与ADP共结晶的Gdm⁺离子,揭示了GuHCl可通过两种机制结合并抑制Hsp104/ClpB。首先,GuHCl破坏DnaK辅助伴侣与M结构域之间的相互作用,M结构域是ClpB活性的关键组分,从而通过加强M结构域/NBD-1连接将Hsp104/ClpB稳定在抑制构象中。其次,GuHCl抑制NBD-1的持续ATP周转(Kummer等,2013)。在酵母中,GuHCl通过抑制Hsp100分子伴侣治愈朊病毒。这种抑制发生的原因是GuHCl的胍离子(Gdm⁺)与结合的核苷酸和保守的GLU相互作用,影响核苷酸结合亲和力,并通过特异性结合N端核苷酸结合区域干扰ATP水解所需的必需GLU(Zeymer等,2013)。

多年来,药物发现工作严重依赖于计算机辅助药物设计(CADD),显著减少了在实验室中规划、执行和测试实验所需的时间和成本(Garg等,2020)。通过利用计算机方法,如分子对接和分子动力学,研究人员可以预测潜在药物候选物与靶蛋白的结合亲和力和稳定性。此外,计算机吸收、分布、代谢、排泄和毒性(ADMET)分析有助于识别最有前景的化合物用于进一步研究(Tabani,2023)。

本研究旨在利用计算机方法评估靶向细菌ClpB的抑制剂分子。GuHCl的选择基于先前体内和体外研究结果,证明其能够结合并抑制ClpB,如前所述。由于L-精氨酸和D-精氨酸中的胍基在结构上与GuHCl中的胍离子(Gdm⁺)相似,两种对映体也被视为候选配体。对所选分子进行了结合潜力和药代动力学特性评估。进行了包括分子对接和分子动力学模拟在内的计算分析,以深入了解蛋白质-配体相互作用的强度和稳定性。

## 材料与方法

### 蛋白质结构的制备

本研究使用了从嗜热菌(Thermus thermophilus)获得的ClpB的X射线衍射晶体结构(PDB ID: 1QVR;分辨率3.00 Å),该结构可从RCSB蛋白质数据库(PDB)(http://rscb.org)获取(Lee等,2003)。在对接前,通过去除捕获的水分子和共结晶配体对靶蛋白进行清洁和精制。随后,筛选缺失的氨基酸。该结构的FASTA序列(氨基酸序列)显示每个单体具有854个氨基酸残基的序列长度,但PDB格式结构由803个氨基酸残基组成,每个单体缺失51个氨基酸。

使用Modeller 10.4工具填补蛋白质结构中缺失的氨基酸残基(Šali & Blundell,1993)。填补缺失氨基酸残基后,使用ModRefiner算法(Xu & Zhang,2011)精制结构,该算法可在https://zhanggroup.org/ModRefiner/获取。该算法通过两步过程最小化蛋白质的原子级能量。首先,它利用Cα骨架并在低分辨率步骤中最小化主链能量。随后,通过在更高分辨率和更快速度下加入初始侧链原子来最小化整个原子结构的能量。在该过程结束时,算法计算精制结构的均方根偏差(RMSD)和模板建模评分(TM-score),以比较和验证所得精制模型。使用BIOVIA Discovery Studio v21.1.0.20298软件包(Systèmes,2020)为1QVR的精制完整单体生成拉氏图(Ramachandran plot),以评估蛋白质主链构象。为进行结构验证,使用可视化分子动力学(VMD)软件包叠加1QVR的天然和建模单体结构,允许对结构偏差进行比较分析。

### 配体结构的获取

使用PubChem(https://pubchem.ncbi.nlm.nih.gov/)数据库获取配体的三维结构,格式为结构数据文件(.sdf)。使用GuHCl的Gdm⁺离子、L-精氨酸和D-精氨酸作为测试分子。ADP已知与ClpB在4HSE PDB结构中共结晶,用作参比配体(Zeymer等,2013)。测试配体的规范简化分子输入线性输入系统(SMILES)用于计算机ADMET和毒性分析。

### 分子对接

使用AutoDockTools 1.5.7(Goodsell & Olson,1990)生成ClpB PDB结构的蛋白质数据库、部分电荷(Q)和原子类型(T)/PDBQT格式文件,同时引入氢原子和部分原子电荷(Kollman电荷)。配体的能量最小化使用PyRx 0.8(Dallakyan & Olson,2015)上的可用选项,采用MMFF94力场进行。每个配体能量最小化后,分别以pdb、pdbqt和mol2格式保存,用于对接和MD研究。使用PyRx开源平台结合AutoDock Vina(Trott & Olson,2010)继续进行对接。网格盒被定义为覆盖整个蛋白质。X×Y×Z尺寸设置为111.6939 Å × 124.4924 Å × 74.0728 Å,中心网格坐标X、Y、Z分别设置为45.8962、23.4164、33.1118。

对接过程产生的结合构象被进一步分析,使用BIOVIA Discovery Studio v21.1.0.20298软件包(Systèmes,2020)观察其对接分数和与蛋白质关键氨基酸的相互作用。

### 理化性质、ADME分析以及药物相似性和毒性预测

与对接研究并行,使用SwissADME在线网络服务器(Daina等,2017)筛选和预测配体的理化性质、药代动力学和药物相似性,同时使用ProTox-3.0网络服务器(Banerjee等,2018)分析毒性。

### 分子动力学模拟研究

对接结果分析完成后,使用GROMACS-2024软件包(Berendsen等,1995)结合CHARMM36力场(Huang & MacKerell,2013)对游离蛋白质和所有配体-蛋白质复合物进行MD模拟,以探究配体对ClpB蛋白稳定性和结构变化的影响。清洁蛋白质的拓扑文件使用内置GROMACS命令结合CHARMM36全原子力场生成,而配体拓扑文件由SwissParam(http://www.swissparam.ch/)在线服务器创建(Zoete等,2011)。随后,通过合并蛋白质和配体文件制作蛋白质-配体复合物文件。

复合物在截角八面体周期性盒子(111.01 × 124.01 × 74.91 Å)中溶剂化,蛋白质与每侧边界之间距离为12 Å,使用TIP3P水模型,将复合物保持在中间。然后,通过添加Na⁺离子中和系统,并进行能量最小化步骤。系统在300 K和1 bar压力下分别使用NVT和NPT条件平衡100 ps。最后,生产运行进行100 ns,时间步长保持为2 fs(Bhattarai & Emerson,2021)。

最后,通过计算RMSD、均方根波动(RMSF)、氢键、回转半径(Rg)和溶剂可及表面积(SASA),使用GROMACS内置工具gmx rms、gmx rmsf、gmx hbond、gmx gyrate和gmx sasa分别分析游离蛋白质和蛋白质-配体复合物的轨迹文件(Kushwaha等,2021)。使用VMD软件包(Humphrey等,1996)分析轨迹,并使用GraphPad Prism 8.4.2软件生成图表。

### 总结合自由能计算

为计算蛋白质-配体复合物的总结合自由能(ΔGbind),将g_mmpbsa软件包与GROMACS结合使用(Valdés-Tresanco等,2021)。g_mmpbsa使用分子力学/泊松-玻尔兹曼表面积(MM/PBSA)方法计算复合物的ΔGbind(Kushwaha等,2021)。使用100 ns期间的全部MD模拟轨迹计算所有蛋白质-配体复合物的ΔGbind。以下方程提供了水溶液中蛋白质-配体复合物的ΔGbind。

ΔGbind = Gcomplex - (Gprotein + Gligand)

其中,Gcomplex是蛋白质-配体复合物的能量,Gprotein和Gligand分别表示水溶液中蛋白质和配体的能量。方程中各项的自由能使用以下方程计算。

Gx = Ebonded + (Evdw + Eelec) + Gpolar + Gnon-polar

在此方程中,Gx可以是Gcomplex、Gprotein或Gligand,Ebonded表示分子内键合接触提供的能量,始终视为零(Kollman等,2000)。Evdw表示范德华能量,Eelec表示静电能量。Gpolar是与分子(蛋白质或配体)与周围溶剂之间静电相互作用相关的能量。Gnon-polar表示非极性或疏水性溶剂化能量,即非静电相互作用(如疏水效应)引起的自由能贡献。

Gnon-polar推导如下:

Gnon-polar = γ·SASA + b

在此方程中,SASA表示溶剂可及表面积,b是拟合参数,γ是表面张力系数。因此,g_mmpbsa工具通过计算水溶液环境中蛋白质、配体和总复合物的该方程的每个能量组分来确定总结合自由能(Sundar等,2019)。

## 结果与讨论

本研究利用计算机方法研究了ClpB蛋白,重点关注其与GuHCl的胍离子(Gdm⁺)以及L-精氨酸和D-精氨酸的相互作用,以ADP作为对照。研究分为三个主要步骤:分子对接、ADMET分析和分子动力学(MD)模拟。

分子对接作为虚拟筛选步骤,用于识别配体结合位点及其亲和力。ADMET分析评估了测试配体的药代动力学和毒性特征。最后,通过MD模拟在预定条件下评估游离蛋白质和蛋白质-配体复合物的稳定性。

### 蛋白质精制

在使用Modeller工具填补1QVR单体结构中缺失的氨基酸残基后,所得模型需要精制以消除非键合原子之间不自然重叠引起的空间冲突等伪影。这可以通过短时分子动力学(MD)模拟实现,使结构弛豫为更有利的能量构象(Adiyaman & McGuffin,2019)。或者,可以使用经过验证的计算工具如ModRefiner进行精制,这是一种快速高效的蛋白质结构优化算法。

ModRefiner分两个阶段运行:第一阶段使用预定义的Cα骨架构象重建真实的主链,第二阶段通过使用基于物理和基于知识的力场的组合加入侧链原子并最小化结构能量来提高结构准确性。最终精制模型的质量通过RMSD更低和TM-score更高等指标评估,这些指标表明折叠准确性和结构真实性得到改善(Xu & Zhang,2011)。图3显示了1QVR的精制结构。RMSD值在0-2 Å范围内,表明与初始建模结构具有相当高的结构相似性。TM-score接近1表明折叠模式的相似性和准确性更高。

### 拉氏图

使用拉氏图验证1QVR单体结构的精制模型,并与原始1QVR结构进行比较(图4)。拉氏图帮助我们理解氨基酸可用的构象空间,并基于主链二面角(φ和ψ)显示其理论上有利的区域。蛋白质主链Cα-N和Cα-C键之间的旋转角分别用φ(phi)和ψ(psi)表示(Ramachandran & Sasisekharan,1968)。ψ通常绘制在y轴上,φ绘制在x轴上。在两个图中,三角形代表甘氨酸,方形代表脯氨酸,圆形代表所有其他氨基酸。

在这些图中可以识别三个主要区域。浅蓝色边缘区域代表α-螺旋和β-折叠构象,这些是不会发生空间冲突的允许构象。粉色边缘区域内的区域是部分允许区域,代表罕见的左手α-螺旋构象(Ramachandran & Sasisekharan,1968)。这些区域中的氨基酸以绿色显示,精制结构(图4-a)中的大多数残基位于空间有利区域内。

没有彩色线条边缘的区域表示空间禁阻构象,其中多肽中的原子比其范德华半径之和更接近,除了没有侧链的甘氨酸(红色三角形)(Ho & Brasseur,2005)。精制结构中这些禁阻区域中的非甘氨酸残基(红色圆形)极少。因此,总体而言,拉氏图表明精制的1QVR单体结构以最小的空间冲突折叠,并采用能量有利的主链构象。

### 分子对接

将GuHCl的Gdm⁺离子、L-精氨酸和D-精氨酸作为测试配体,ADP作为对照配体进行分子对接。所有配体使用盲对接方法与1QVR结构对接。如图5所示,4HSE晶体结构包含NBD-1至M区域(残基141至534),带有结晶的Gdm⁺离子和ADP。在该结构中,Gdm⁺离子与GLU 209、ASP 170和PRO 171形成氢键,而ADP与THR 205、GLY 201、ALA 206、GLY 203和ILE 73结合(Zeymer等,2013)。

有意采用盲对接方法,通过评估ADP是否仍会结合到4HSE结构中观察到的相同功能相关残基,来评估对接方案的可靠性和准确性。此外,该方法确保了对测试配体的无偏评估,允许它们在1QVR结构中探索并结合到能量最有利的位点,而不受预定义约束的限制。

在1QVR的对接结果中(图6),ADP结合到NBD-1,与GLN 8、ARG 11、GLU 12、GLU 144、ILE 215、VAL 216、LYS 225、GLY 226、LYS 227形成常规氢键。相比之下,Gdm⁺离子通过形成常规氢键与SER 539、LYS 540、GLU 543和GLU 545结合到NBD-2,并与GLU 545、ASP 614形成吸引电荷相互作用。如前所述,该Gdm⁺离子与带负电荷的氨基酸GLU相互作用,与4HSE晶体结构相同,尽管在对接结果显示其对NBD-2具有高亲和力。L-精氨酸和D-精氨酸中带正电荷的胍基与1QVR结构NBD-1中带负电荷的GLU相互作用,如图6所示。表1显示了所有配体与蛋白质的结合亲和力以及它们与最有利构象相邻残基的相互作用。

### ADMET结果

SwissADME服务器在三个主要类别中提供分析结果:理化性质、药代动力学和药物相似性。在分析配体的口服生物利用度药物相似性时,考虑了利平斯基五规则(RO5)。为使化合物具有口服生物利用度,配体不应违反RO5超过一次。为预测吸收和渗透是理想的并被接受为口服活性配体,RO5如下:氢键供体不超过五个,氢键受体不超过十个,分子量(MWT)小于500道尔顿,计算Log P(ClogP)小于五(Lipinski等,2001)。GuHCl、L-精氨酸和D-精氨酸显示零违规,因此被认为具有口服生物利用度。

在评估药代动力学性质时,考虑了血脑屏障(BBB)、胃肠道(GI)吸收、皮肤渗透系数(Log Kp)和细胞色素(CYPs)同工酶的底物。所有三种配体均显示有利性质,同时具有高GI吸收能力。

使用ProTox-II服务器评估毒性。GuHCl被预测为轻度毒性(IV类),LD₅₀为350 mg/kg,而L-精氨酸和D-精氨酸被归类为可能有害(V类),LD₅₀为245,050 mg/kg。尽管GuHCl的LD₅₀较低,但它在肝毒性、细胞毒性、热休克因子反应元件(HSE)、线粒体膜电位(MMP)影响、磷蛋白肿瘤抑制因子(p53)活性和ATP酶家族AAA结构域含蛋白5(ATAD5)方面与L-精氨酸和D-精氨酸一样显示无活性结果。因此,从总体ADMET结果来看,测试配体具有被相应修饰为药物候选物的潜力。

### MD结果

完成对接研究后,对游离蛋白质和蛋白质-配体复合物(即ADP、GuHCl、L-精氨酸和D-精氨酸)进行MD模拟。如前所述,使用RMSD、RMSF、氢键、Rg和SASA计算来分析游离蛋白质的动力学行为,并检查蛋白质-配体复合物在100 ns期间的稳定性。

均方根偏差(RMSD)测量系统在指定时间段内原子相对于参考结构的平均位移。RMSD是复合物稳定性的指标;较高波动表明较低稳定性。最初,RMSD可能在模拟过程中逐渐增加,但一旦复合物达到构象平衡,通常会稳定下来。均方根波动(RMSF)通过评估模拟过程中特定残基的可变性来评估蛋白质的结构变化。较低的RMSF值表示更稳定的残基,提供了配体如何影响蛋白质结构的见解。这有助于理解配体对蛋白质整体稳定性的影响(Opo等,2021)。

根据图7,游离蛋白质从约45 ns到100 ns时间线显示稳定的RMSD,平均RMSD值为0.5185 nm,波动在1 Å以内。在游离蛋白质的几个位置观察到RMSF的变化,它们分布在NBD-1、M结构域和NBD-2之间。在残基编号270-290(NBD-1)、415-500(M结构域)和620-650(NBD-2)之间观察到显著变化,给出0.75 nm至0.8 nm的相似RMSF值。

根据图8中的图表,蛋白质-ADP复合物显示稳定的RMSD,平均为0.5826 nm。随时间推移,复合物未显示显著波动,表明稳定性。考虑到蛋白质-ADP复合物的RMSF,与游离蛋白质相比未显示重大振荡。

GuHCl的RMSD(图9)显示稳定性直至60 ns。从60-90 ns,观察到严重波动。还注意到,在生产运行的最后10 ns未观察到显著波动,表明如果运行持续超过100 ns,蛋白质-GuHCl复合物可能会稳定。总体而言,蛋白质-GuHCl复合物的平均RMSD为0.7355 nm。

蛋白质-L-精氨酸复合物的RMSD在45 ns后稳定,平均RMSD为0.7771 nm,波动差异约0.2 nm。类似地,D-精氨酸复合物在30 ns后稳定并保持稳定至模拟结束,平均RMSD为0.6851 nm,波动差异约0.3 nm。两个复合物均显示最小波动并在整个模拟过程中保持稳定(图9)。

众所周知,精氨酸结构由胍基组成。尽管这一事实可能表明精氨酸和Gdm⁺离子由于结构相似性应对蛋白质稳定性具有相同影响,但RMSD结果表明L-精氨酸和D-精氨酸结合比Gdm⁺离子与蛋白质的结合更稳定。这可能是由于其他官能团,如精氨酸结构中的羧基。

蛋白质-GuHCl和L-精氨酸复合物的RMSF显示整个蛋白质结构中残基的类似偏差,在NBD-1和M结构域方向可观察到更显著的波动。相比之下,D-精氨酸复合物与游离蛋白质相比在RMSF图中未显示显著变化(图9)。值得注意的是,Gdm⁺离子结合并保持在NBD-2结构域中,而L-精氨酸和D-精氨酸保持在NBD-1结构域中。

根据Cathleen Zeymer及其同事使用4HSE ClpB结构进行的研究,他们提出Gdm⁺结合可影响ATP水解,这是由于结合到NBD-1中存在的保守GLU残基。这种相互作用影响ATP与NBD-1的结合,诱导结构域中的构象变化,导致抑制效应。他们还提出,这种抑制变化仅在NBD-1中发生,而在GuHCl存在下NBD-2中不发生(Zeymer等,2013)。

图9:ClpB与GuHCl、L-精氨酸和D-精氨酸复合物在100 ns分子动力学模拟中的RMSD和RMSF分布图。GuHCl复合物在约60 ns前保持稳定,60–90 ns期间出现波动,并在最后10 ns恢复稳定(平均RMSD:0.7355 nm)。L-精氨酸和D-精氨酸复合物分别在约45 ns和30 ns后趋于稳定,并持续保持稳定状态(平均RMSD分别为0.7771 nm和0.6851 nm)。RMSF偏差在GuHCl和L-精氨酸作用下于NBD-1和M结构域最为显著,而D-精氨酸则表现出最小的变化。

Eva Kummer及其同事的研究也表明,Gdm⁺离子可与NBD-1结合,并通过在该区域引起构象变化从而降低NBD-1的ATP水解速率,进而干扰ClpB功能。他们还提出,Gdm⁺离子可在中间结构域引发构象变化,从而干扰DnaK对ClpB的激活(Kummer et al., 2013)。因此,本研究可利用上述信息解释Gdm⁺离子所得RMSF结果:尽管其结合位点与前述研究不同——在整个100 ns模拟过程中,Gdm⁺离子始终结合于NBD-2(这是我们对接结果中预测的最佳结合位点),但其在残基160–460区域(涵盖NBD-1和中间结构域)仍表现出显著波动。由此我们推测,尽管Gdm⁺离子对NBD-2具有更高亲和力,但它仍可能引发NBD-1和中间结构域的构象变化。这种结合可能通过干扰NBD-1中的ATP水解以及破坏中间结构域与DnaK之间的连接(该连接对ClpB的激活至关重要,如Udari等人实验数据所示)来抑制ClpB活性。此外,L-精氨酸表现出类似胍基效应,因其RMSF结果与Gdm⁺离子呈现相似的波动模式。同时,其RMSD曲线显示结合稳定性优于Gdm⁺离子,提示L-精氨酸可能是靶向ClpB的潜在候选分子。

为进一步理解测试配体的相互作用,还进行了氢键分析、回转半径(Rg)和可及表面积(SASA)计算,并与游离蛋白进行比较。氢键形成于电负性原子附近,且氢原子与另一电负性原子共价结合。蛋白-配体复合物的稳定性取决于分子间氢键的数量(da Fonseca et al., 2023)。通过评估在存在或不存在主要配体条件下蛋白内氢键(intraprotein hydrogen bonds)以及配体与蛋白之间氢键的形成情况,评价了模拟100 ns期间蛋白稳定性及蛋白-配体复合物稳定性(图10)。配体结合后,三种测试配体均促进了蛋白内氢键的形成,尤其在模拟前半段,GuHCl的作用最为显著。

蛋白表面与周围溶剂分子接触的区域称为可及表面积(SASA)(Mazola et al., 2015)。根据图11,整个蛋白的SASA计算结果显示,GuHCl、L-精氨酸和D-精氨酸复合物均呈上升趋势。此外,GuHCl复合物的整体蛋白SASA与游离蛋白相比略高但趋势相似。L-精氨酸和D-精氨酸在模拟前20 ns内出现更陡峭的SASA峰值,表明ClpB疏水核心更易暴露于周围水环境中。总体而言,这些观察结果表明,所有主要配体均可能引起蛋白构象变化,从而导致SASA增加。

回转半径(Rg)反映模拟期间蛋白结构的整体致密性,通过测量各蛋白原子末端与其质心之间的距离变化来计算。波动越小,折叠蛋白结构的稳定性越好(Choudhary et al., 2023)。图11展示了模拟过程中Rg值的变化,显示除GuHCl外,所有蛋白-配体复合物均比游离蛋白更为伸展。

综合SASA和Rg结果,观察到蛋白致密性降低(Rg升高)与SASA增加并存。值得注意的是,在GuHCl体系中,尽管SASA仍高于游离蛋白,Rg却有所下降。这可能表明发生了构象变化,导致Rg降低,同时使原本隐蔽的残基更易暴露,从而引起SASA升高。

总结合自由能计算 利用100 ns分子动力学模拟轨迹计算配体在复合物中的总结合自由能。采用与GROMACS配套使用的g_mmpbsa工具,基于MM/PBSA方法进行结合能计算。 使用g_mmpbsa模块分别计算了ClpB蛋白与对照配体ADP及测试配体GuHCl、L-精氨酸和D-精氨酸复合物的Evdw、Eelec、Gpolar、Gnonpolar和△Gbind,结果见表3。其中,Gpolar对总结合自由能有正贡献,而Evdw、Eelec、Gnonpolar为负贡献。如表3和图12所示,四种化合物的总结合自由能均低于零,表明其对ClpB具有良好的亲和力,且三种测试分子对蛋白结构的亲和力均高于对照配体ADP本身。

结论 ClpB是一种在应激条件下维持蛋白质稳态的关键细菌分子伴侣,是开发新型抗菌药物的重要靶点。本研究通过计算方法评估了GuHCl、L-精氨酸和D-精氨酸作为ClpB潜在抑制剂的可行性。对接分析确定了有利的结合位点,ADMET预测显示其具有良好的口服生物利用度、高胃肠道吸收率和安全的毒性特征。分子动力学模拟揭示了配体诱导的ClpB在NBD-1和M结构域的构象变化,这些变化可能损害ATP水解并破坏与共伴侣DnaK的相互作用,从而影响聚集体的再激活。氢键分析表明,配体结合促进了蛋白内氢键的形成,有助于增强蛋白稳定性并形成稳定的复合物。后MD结合自由能计算进一步证实,三种测试配体均与ClpB表现出良好的稳定性和相互作用,其亲和力高于对照配体ADP。上述结果表明,GuHCl、L-精氨酸和D-精氨酸有望成为ClpB的潜在抑制剂,为抗菌治疗提供新策略。所观察到的构象变化(尤其是NBD-1和M结构域)与先前实验研究提出的抑制机制一致,但在结合位点偏好方面存在差异,需进一步研究。未来需开展体内外实验以验证这些发现并探索其治疗潜力。

利益冲突声明 作者声明无利益冲突。