[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"detail-sidebar-cat-0-en-105":3,"doc-seo-385162-105":59,"doc-detail-385162-en":130},{"code":4,"msg":5,"data":6},0,"success",[7,13,18,23,28,33,38,43,48,51,55],{"id":8,"doc_module":4,"doc_module_name":9,"category_name":10,"show_sort_weight":11,"slug":12},1,"Document","Story & Novel",90,"story-novel",{"id":14,"doc_module":4,"doc_module_name":9,"category_name":15,"show_sort_weight":16,"slug":17},2,"Literature",80,"literature",{"id":19,"doc_module":4,"doc_module_name":9,"category_name":20,"show_sort_weight":21,"slug":22},4,"Exam",70,"exam",{"id":24,"doc_module":4,"doc_module_name":9,"category_name":25,"show_sort_weight":26,"slug":27},5,"Comic",60,"comic",{"id":29,"doc_module":4,"doc_module_name":9,"category_name":30,"show_sort_weight":31,"slug":32},6,"Technology",50,"technology",{"id":34,"doc_module":4,"doc_module_name":9,"category_name":35,"show_sort_weight":36,"slug":37},7,"Healthcare",40,"healthcare",{"id":39,"doc_module":4,"doc_module_name":9,"category_name":40,"show_sort_weight":41,"slug":42},8,"Research & Report",30,"research-report",{"id":44,"doc_module":4,"doc_module_name":9,"category_name":45,"show_sort_weight":46,"slug":47},9,"Religion & Spirituality",20,"religion-spirituality",{"id":46,"doc_module":4,"doc_module_name":9,"category_name":49,"show_sort_weight":46,"slug":50},"World Cup","world-cup",{"id":52,"doc_module":4,"doc_module_name":9,"category_name":53,"show_sort_weight":52,"slug":54},10,"Lifestyle","lifestyle",{"id":56,"doc_module":4,"doc_module_name":9,"category_name":57,"show_sort_weight":24,"slug":58},19,"General","general",{"code":4,"msg":60,"data":61},"ok",{"site_id":62,"language":63,"slug":64,"title":65,"keywords":66,"description":67,"schema_data":68,"social_meta":123,"head_meta":125,"extra_data":127,"updated_unix":129},105,"en","the-potential-for-targeting-avil-and-other-actin-binding-proteins-in-rhabdomyosarcoma","The Potential for Targeting AVIL and Other Actin-Binding Proteins in Rhabdomyosarcoma","","Rhabdomyosarcoma (RMS) is the most common pediatric soft-tissue cancer, yet high-risk children show survival rates below 27% despite aggressive multi-modal therapy. Targeted treatments remain unavailable, and drugging actin-binding proteins has historically been difficult. Recent advances highlight AVIL (advillin), a novel oncogenic actin-binding protein implicated in cytoskeletal functions. AVIL is overexpressed in RMS cell lines, patient-derived xenografts, and clinical samples across alveolar and embryonal subtypes, and its dysregulation drives transformation. This perspective evaluates therapeutic targeting feasibility and cancer-specific disruption of cellular infrastructure.",{"@graph":69,"@context":122},[70,84,105],{"@type":71,"itemListElement":72},"BreadcrumbList",[73,77,79,82],{"item":74,"name":75,"@type":76,"position":8},"https://docshare.wps.com","Home","ListItem",{"item":78,"name":9,"@type":76,"position":14},"https://docshare.wps.com/document/",{"item":80,"name":35,"@type":76,"position":81},"https://docshare.wps.com/document/healthcare/",3,{"item":83,"name":65,"@type":76,"position":19},"https://docshare.wps.com/document/the-potential-for-targeting-avil-and-other-actin-binding-proteins-in-rhabdomyosarcoma/385162/",{"url":83,"name":65,"@type":85,"image":86,"author":91,"headline":65,"publisher":94,"fileFormat":97,"inLanguage":63,"description":67,"dateModified":98,"datePublished":99,"encodingFormat":97,"isAccessibleForFree":100,"interactionStatistic":101},"DigitalDocument",{"url":87,"@type":88,"width":89,"height":90},"https://docshare.wps.com/thumbnails/the-potential-for-targeting-avil-and-other-actin-binding-proteins-in-rhabdomyosarcoma/385162.png","ImageObject",300,407,{"name":92,"@type":93},"McQueen","Person",{"url":74,"name":95,"@type":96},"DocShare","Organization","application/pdf","2026-10-22","2026-09-24",true,{"@type":102,"interactionType":103,"userInteractionCount":29},"InteractionCounter",{"@type":104},"ViewAction",{"@type":106,"mainEntity":107},"FAQPage",[108,114,118],{"name":109,"@type":110,"acceptedAnswer":111},"Why is targeted therapy for RMS still a major challenge?","Question",{"text":112,"@type":113},"High-risk RMS has poor survival despite intensive multi-modal treatments, and targeted therapies are not currently available. Targeting actin-binding proteins has historically been difficult, contributing to the gap in durable options.","Answer",{"name":115,"@type":110,"acceptedAnswer":116},"What is AVIL (advillin) and why is it relevant to RMS?",{"text":117,"@type":113},"AVIL is described as a novel oncogenic actin-binding protein involved in many cytoskeletal functions. It is overexpressed in RMS models and clinical samples, suggesting an important role in RMS biology.",{"name":119,"@type":110,"acceptedAnswer":120},"How does altering AVIL expression affect RMS-related transformation?",{"text":121,"@type":113},"The document states that overexpression of AVIL in mesenchymal stem cells induces neoplastic transformation in vitro and in vivo. Reversing overexpression through genetic modulation reverses the transformation, supporting a causal role in tumorigenesis and maintenance.","https://schema.org",{"og:url":83,"og:type":124,"og:title":65,"og:site_name":95,"og:description":67},"article",{"robots":126,"canonical":83},"index,follow",{"doc_id":128,"site_id":62},385162,1791226233,{"code":4,"msg":5,"data":131},{"doc_id":128,"user_id":132,"nickname":92,"user_avatar":133,"doc_module":4,"category_id":34,"category_name":35,"doc_title":65,"doc_description":67,"doc_content":134,"file_id":135,"file_url":136,"file_type":137,"file_size":138,"view_count":29,"is_deleted":4,"is_public":8,"is_downloadable":8,"audit_status":8,"page_count":139,"language":140,"language_code":63,"site_id":62,"html_lang":63,"table_of_contents":141,"faqs":142,"seo_title":143,"seo_description":67,"update_tm":144,"read_time":41},5909890329169,"https://ap-avatar.wpscdn.com/davatar_9964176cb1d06d4a9deccf72a44ae3dc","Perspective  \nThe Potential for Targeting AVIL and Other Actin-Binding Proteins in Rhabdomyosarcoma  \nRobert Cornelison†, Laine Marrah †, Adelaide Fierti , Claire Piczak, Martyna Glowczyk, Anam Tajammal , Sarah Lynch and Hui Li *  \nCitation: Cornelison, R.; Marrah, L.; Fierti, A.; Piczak, C.; Glowczyk, M.; Tajammal, A.; Lynch, S.; Li, H. The Potential for Targeting AVIL and Other Actin-Binding Proteins in Rhabdomyosarcoma. Int. J. Mol. Sci. 2023, 24, 14196. [https://doi.org/](https://doi.org/)[ ](https://doi.org/)[10.3390/ijms241814196](10.3390/ijms241814196)  \nAcademic Editor: Wan Lee  \nReceived: 1 August 2023  \nRevised: 12 September 2023  \nAccepted: 13 September 2023  \nPublished: 17 September 2023  \nCopyright: © 2023 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license ([https://](https://)[ ](https://)[creativecommons.org/licenses/by/](creativecommons.org/licenses/by/)[ ](creativecommons.org/licenses/by/)[4.0/](4.0/)) .  \nDepartment of Pathology, School of Medicine, University of Virginia, Charlottesville, VA 22908, USA  \n* [Correspondence: hl9r@virginia.edu](Correspondence: hl9r@virginia.edu)  \n† These authors contributed equally to this work.  \nAbstract: Rhabdomyosarcoma (RMS) is the most common pediatric soft-tissue cancer with a survival rate below 27% for high-risk children despite aggressive multi-modal therapeutic interventions. After decades of research, no targeted therapies are currently available. Therapeutically targeting actinbinding proteins, although promising, has historically been challenging. Recent advances have made this possibility more salient, including our lab's identification of advillin (AVIL), a novel oncogenic actin-binding protein that plays a role in many cytoskeletal functions. AVIL is overexpressed in many RMS cell lines, patient-derived xenograft models, and a cohort of 30 clinical samples of both the alveolar (ARMS) and embryonal (ERMS) subtypes. Overexpression of AVIL in mesenchymal stem cells induces neoplastic transformation both in vitro and in vivo, and reversing overexpression through genetic modulation reverses the transformation. This suggests a critical role of AVIL in RMS tumorigenesis and maintenance. As an actin-binding protein, AVIL would not traditionally be considered a druggable target. This perspective will address the feasibility of targeting differentially expressed actin-binding proteins such as AVIL therapeutically, and how critical cell infrastructure can be damaged in a cancer-specific manner.  \nKeywords: rhabdomyosarcoma; pediatric cancer; actin-binding protein; advillin; AVIL  \n1. Introduction  \nRhabdomyosarcoma (RMS) is the most common cancer of soft tissue in children, accounting for at least half of all pediatric sarcomas [1] . Approximately 90% of all RMS cases occur in patients under 25 years of age, with the majority occurring in children under 10 [2] . RMS has significant variability in outcomes. Low-risk patients with localized disease face a survival rate of 90%, while high risk patients with metastatic or recurrent disease face dismal survival rates of 21% and 30%, respectively [3,4] . These high-risk groups have seen no improvement in outcomes in the last 30 years, making the lack of novel targeted treatments a major roadblock to durable cures.  \nThe major anatomic sites of RMS include the head, neck, and genitourinary tract. Previously, the classification of RMS was primarily based upon histology to delineate alveolar RMS (ARMS) and embryonal RMS (ERMS) . The most recent World Health Organization (WHO) classification of RMS consists of four groups: alveolar, embryonal, pleiomorphic, and spindle cell/sclerosing, with new subdivisions of spindle cell/sclerosing tumors based upon molecular alterations (Table 1) [5–7] . These alterations differentially regulate the pathways involved in promoting tumorigenesis. For instance, PAX3/7 an","cbCaic7JqdzXvPhF","https://ap.wps.com/l/cbCaic7JqdzXvPhF","pdf",1103326,12,"English","# Introduction\n## Disease burden and survival variability\n## Classification and molecular alterations of RMS\n# Abstract\n# Keywords","[{\"question\":\"Why is targeted therapy for RMS still a major challenge?\",\"answer\":\"High-risk RMS has poor survival despite intensive multi-modal treatments, and targeted therapies are not currently available. Targeting actin-binding proteins has historically been difficult, contributing to the gap in durable options.\"},{\"question\":\"What is AVIL (advillin) and why is it relevant to RMS?\",\"answer\":\"AVIL is described as a novel oncogenic actin-binding protein involved in many cytoskeletal functions. It is overexpressed in RMS models and clinical samples, suggesting an important role in RMS biology.\"},{\"question\":\"How does altering AVIL expression affect RMS-related transformation?\",\"answer\":\"The document states that overexpression of AVIL in mesenchymal stem cells induces neoplastic transformation in vitro and in vivo. Reversing overexpression through genetic modulation reverses the transformation, supporting a causal role in tumorigenesis and maintenance.\"}]","The Potential for Targeting AVIL and Other Actin-Binding Proteins in Rhabdomyosarcoma | PDF",1790267057]