Margherita Piccardi, Valeria Butera, Ignazio Sardo, Stefano Landi, Federica Gemignani, Giampaolo Barone, Angelo Spinello, Sarah J Moore
{"title":"肿瘤生物标志物间皮素与具有治疗活性的工程靶向蛋白之间相互作用的计算建模和实验验证。","authors":"Margherita Piccardi, Valeria Butera, Ignazio Sardo, Stefano Landi, Federica Gemignani, Giampaolo Barone, Angelo Spinello, Sarah J Moore","doi":"10.1002/pro.70263","DOIUrl":null,"url":null,"abstract":"<p><p>Mesothelin (MSLN) is a cell surface glycoprotein overexpressed in many solid tumors, which is known to interact with cancer antigen CA125/MUC16, promoting cancer cell adhesion and metastasis. MSLN has been used as a target of multiple antibody-based therapeutic strategies, but their efficacy remains limited, potentially due to inherent pharmacokinetics conferred by the structure of antibodies (~150 kDa). To provide an alternative targeting molecule, we engineered a small scaffold protein derived from the tenth domain of human fibronectin type III (Fn3, 12.8 kDa) to bind MSLN with nanomolar affinity as a theranostic agent for MSLN-positive cancers. In this study, we explored the Fn3-MSLN interaction site through computational modeling and experimentally validated the model through domain-level and fine epitope mapping. Fn3-MSLN binding was predicted by a consensus approach, comparing multiple protein-protein docking software, the deep-learning-based algorithm AlphaFold3, and performing molecular dynamics (MD) simulations. To validate the prediction, full-length MSLN, single MSLN domains, or combinations of domains were expressed on the yeast surface, and Fn3 binding to displayed MSLN domains was measured by flow cytometry. The employed algorithms predicted two distinct binding modes for Fn3. Overall, experimental data agreed with our in silico prediction resulting from the AlphaFold3 model, confirming that MSLN domains B and C are predominantly involved in the interaction.</p>","PeriodicalId":20761,"journal":{"name":"Protein Science","volume":"34 9","pages":"e70263"},"PeriodicalIF":5.2000,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12355969/pdf/","citationCount":"0","resultStr":"{\"title\":\"Computational modeling and experimental validation of the interaction between tumor biomarker mesothelin and an engineered targeting protein with therapeutic activity.\",\"authors\":\"Margherita Piccardi, Valeria Butera, Ignazio Sardo, Stefano Landi, Federica Gemignani, Giampaolo Barone, Angelo Spinello, Sarah J Moore\",\"doi\":\"10.1002/pro.70263\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Mesothelin (MSLN) is a cell surface glycoprotein overexpressed in many solid tumors, which is known to interact with cancer antigen CA125/MUC16, promoting cancer cell adhesion and metastasis. MSLN has been used as a target of multiple antibody-based therapeutic strategies, but their efficacy remains limited, potentially due to inherent pharmacokinetics conferred by the structure of antibodies (~150 kDa). To provide an alternative targeting molecule, we engineered a small scaffold protein derived from the tenth domain of human fibronectin type III (Fn3, 12.8 kDa) to bind MSLN with nanomolar affinity as a theranostic agent for MSLN-positive cancers. In this study, we explored the Fn3-MSLN interaction site through computational modeling and experimentally validated the model through domain-level and fine epitope mapping. Fn3-MSLN binding was predicted by a consensus approach, comparing multiple protein-protein docking software, the deep-learning-based algorithm AlphaFold3, and performing molecular dynamics (MD) simulations. To validate the prediction, full-length MSLN, single MSLN domains, or combinations of domains were expressed on the yeast surface, and Fn3 binding to displayed MSLN domains was measured by flow cytometry. The employed algorithms predicted two distinct binding modes for Fn3. Overall, experimental data agreed with our in silico prediction resulting from the AlphaFold3 model, confirming that MSLN domains B and C are predominantly involved in the interaction.</p>\",\"PeriodicalId\":20761,\"journal\":{\"name\":\"Protein Science\",\"volume\":\"34 9\",\"pages\":\"e70263\"},\"PeriodicalIF\":5.2000,\"publicationDate\":\"2025-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12355969/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Protein Science\",\"FirstCategoryId\":\"99\",\"ListUrlMain\":\"https://doi.org/10.1002/pro.70263\",\"RegionNum\":3,\"RegionCategory\":\"生物学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"BIOCHEMISTRY & MOLECULAR BIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Protein Science","FirstCategoryId":"99","ListUrlMain":"https://doi.org/10.1002/pro.70263","RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"BIOCHEMISTRY & MOLECULAR BIOLOGY","Score":null,"Total":0}
Computational modeling and experimental validation of the interaction between tumor biomarker mesothelin and an engineered targeting protein with therapeutic activity.
Mesothelin (MSLN) is a cell surface glycoprotein overexpressed in many solid tumors, which is known to interact with cancer antigen CA125/MUC16, promoting cancer cell adhesion and metastasis. MSLN has been used as a target of multiple antibody-based therapeutic strategies, but their efficacy remains limited, potentially due to inherent pharmacokinetics conferred by the structure of antibodies (~150 kDa). To provide an alternative targeting molecule, we engineered a small scaffold protein derived from the tenth domain of human fibronectin type III (Fn3, 12.8 kDa) to bind MSLN with nanomolar affinity as a theranostic agent for MSLN-positive cancers. In this study, we explored the Fn3-MSLN interaction site through computational modeling and experimentally validated the model through domain-level and fine epitope mapping. Fn3-MSLN binding was predicted by a consensus approach, comparing multiple protein-protein docking software, the deep-learning-based algorithm AlphaFold3, and performing molecular dynamics (MD) simulations. To validate the prediction, full-length MSLN, single MSLN domains, or combinations of domains were expressed on the yeast surface, and Fn3 binding to displayed MSLN domains was measured by flow cytometry. The employed algorithms predicted two distinct binding modes for Fn3. Overall, experimental data agreed with our in silico prediction resulting from the AlphaFold3 model, confirming that MSLN domains B and C are predominantly involved in the interaction.
期刊介绍:
Protein Science, the flagship journal of The Protein Society, is a publication that focuses on advancing fundamental knowledge in the field of protein molecules. The journal welcomes original reports and review articles that contribute to our understanding of protein function, structure, folding, design, and evolution.
Additionally, Protein Science encourages papers that explore the applications of protein science in various areas such as therapeutics, protein-based biomaterials, bionanotechnology, synthetic biology, and bioelectronics.
The journal accepts manuscript submissions in any suitable format for review, with the requirement of converting the manuscript to journal-style format only upon acceptance for publication.
Protein Science is indexed and abstracted in numerous databases, including the Agricultural & Environmental Science Database (ProQuest), Biological Science Database (ProQuest), CAS: Chemical Abstracts Service (ACS), Embase (Elsevier), Health & Medical Collection (ProQuest), Health Research Premium Collection (ProQuest), Materials Science & Engineering Database (ProQuest), MEDLINE/PubMed (NLM), Natural Science Collection (ProQuest), and SciTech Premium Collection (ProQuest).