Yufei Pan , Mingyue Pan , Yuanzheng Huang , Baoping Lian , Yutian Feng , Juan Liu , Xiaoxuan Liu
{"title":"核酸治疗肝病:十年的技术融合和临床挑战","authors":"Yufei Pan , Mingyue Pan , Yuanzheng Huang , Baoping Lian , Yutian Feng , Juan Liu , Xiaoxuan Liu","doi":"10.1016/j.iliver.2025.100178","DOIUrl":null,"url":null,"abstract":"<div><div>This review synthesizes a decade of advancements in nucleic acid therapeutics for liver diseases, incorporating bibliometric analysis and translational evaluation. The field has evolved from foundational viral vector engineering to precision genome editing and RNA-based modulation, with advancements in CRISPR-Cas9 and innovations in non-viral delivery systems. Our analysis highlights the concentrated research efforts made in metabolic disorders and hepatocellular carcinoma, and reveals an emerging emphasis on multifactorial pathologies. Although clinical milestones highlight important advancements in targeting strategies, significant challenges remain in immune compatibility and preclinical translation. The integration of computational modeling, human-relevant disease models, and combinatorial strategies places nucleic acid therapies in a unique position to tackle the evolving global liver disease burden through mechanism-driven interventions.</div></div>","PeriodicalId":100657,"journal":{"name":"iLIVER","volume":"4 3","pages":"Article 100178"},"PeriodicalIF":0.0000,"publicationDate":"2025-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Nucleic acid therapeutics for liver diseases: A decade of technological convergence and clinical challenges\",\"authors\":\"Yufei Pan , Mingyue Pan , Yuanzheng Huang , Baoping Lian , Yutian Feng , Juan Liu , Xiaoxuan Liu\",\"doi\":\"10.1016/j.iliver.2025.100178\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><div>This review synthesizes a decade of advancements in nucleic acid therapeutics for liver diseases, incorporating bibliometric analysis and translational evaluation. The field has evolved from foundational viral vector engineering to precision genome editing and RNA-based modulation, with advancements in CRISPR-Cas9 and innovations in non-viral delivery systems. Our analysis highlights the concentrated research efforts made in metabolic disorders and hepatocellular carcinoma, and reveals an emerging emphasis on multifactorial pathologies. Although clinical milestones highlight important advancements in targeting strategies, significant challenges remain in immune compatibility and preclinical translation. The integration of computational modeling, human-relevant disease models, and combinatorial strategies places nucleic acid therapies in a unique position to tackle the evolving global liver disease burden through mechanism-driven interventions.</div></div>\",\"PeriodicalId\":100657,\"journal\":{\"name\":\"iLIVER\",\"volume\":\"4 3\",\"pages\":\"Article 100178\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-06-18\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"iLIVER\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2772947825000362\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"iLIVER","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2772947825000362","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Nucleic acid therapeutics for liver diseases: A decade of technological convergence and clinical challenges
This review synthesizes a decade of advancements in nucleic acid therapeutics for liver diseases, incorporating bibliometric analysis and translational evaluation. The field has evolved from foundational viral vector engineering to precision genome editing and RNA-based modulation, with advancements in CRISPR-Cas9 and innovations in non-viral delivery systems. Our analysis highlights the concentrated research efforts made in metabolic disorders and hepatocellular carcinoma, and reveals an emerging emphasis on multifactorial pathologies. Although clinical milestones highlight important advancements in targeting strategies, significant challenges remain in immune compatibility and preclinical translation. The integration of computational modeling, human-relevant disease models, and combinatorial strategies places nucleic acid therapies in a unique position to tackle the evolving global liver disease burden through mechanism-driven interventions.