Article(id=1321425814650990844, tenantId=1146029695717560320, journalId=1235980733773295621, issueId=1321425776323449136, articleNumber=null, orderNo=null, doi=10.20251/j.cnki.1003-3734.2026.14.004, pmid=null, cstr=null, oa=null, hot=null, price=null, onlineType=0, articleFormat=0, articleType=null, articleTypeStr=null, receivedDate=null, receivedDateStr=null, revisedDate=null, revisedDateStr=null, acceptedDate=1756137600000, acceptedDateStr=2025-08-26, onlineDate=1792982065117, onlineDateStr=2026-10-26, pubDate=1785340800000, pubDateStr=2026-07-30, doiRegisterDate=null, doiRegisterDateStr=null, onlineIssueDate=1792982065117, onlineIssueDateStr=2026-10-26, onlineJustAcceptDate=null, onlineJustAcceptDateStr=null, onlineFirstDate=null, onlineFirstDateStr=null, sourceXml=null, magXml=null, createTime=1792982065117, creator=13701087609, updateTime=1792982065117, updator=13701087609, issue=Issue{id=1321425776323449136, tenantId=1146029695717560320, journalId=1235980733773295621, year='2026', volume='35', issue='14', pageStart='1457', pageEnd='1568', issueExtLink='null', onlineDate='null', pubDate='1785340800000', pubDateStr='2026-07-30', beforeIssueId=null, nextIssueId=null, price=null, status=1, issueComplete=1, articleOrder=1, issueType=-1, specialIssue=null, createTime=1792982055979, creator='13701087609', updateTime=1792982930455, updator='13701087609', preIssue=null, nextIssue=null, articleTotal=null, ext={EN=IssueExt(id=1321429444191966074, tenantId=1146029695717560320, journalId=1235980733773295621, issueId=1321425776323449136, language=EN, specialIssueTitle=, coverIllustrator=null, specialIssueEditor=, specialIssueAbout=), CN=IssueExt(id=1321429444196160379, tenantId=1146029695717560320, journalId=1235980733773295621, issueId=1321425776323449136, language=CN, specialIssueTitle=, coverIllustrator=null, specialIssueEditor=, specialIssueAbout=)}, issueFiles=null, downloadFileDto=null}, startPage=1478, endPage=1487, ext={EN=ArticleExt(id=1321425814827151613, articleId=1321425814650990844, tenantId=1146029695717560320, journalId=1235980733773295621, language=EN, title=Comparison of research status of gene therapy drugs at home and abroad based on bibliometric analysis, columnId=null, journalTitle=Chinese Journal of New Drugs, columnName=null, runingTitle=null, highlight=null, articleAbstract=
Objective: To conduct a comprehensive investigation on the developmental trajectory, current status, and research hotspots in gene therapy drugs both domestically and internationally, thus to provide references for promoting technological innovation and facilitating the translation of research outcomes.
Methods: This study retrieved data from the CNKI, Web of Science Core Collection, and ClinicalTrials.gov databases, incorporating 1 890 Chinese articles, 7 598 English articles, and 1 806 clinical trial records. Visualization analysis of the current landscape and trends in the field of gene therapy drugs was conducted using VOSviewer and CiteSpace.
Results: Research in this field has demonstrated sustained growth. Domestic research hotspots are primarily focused on the application of CRISPR-Cas9 technology, the development of nanocarriers, and targeted cancer therapies. In recent years, international research has shown heightened interest in exploratory technologies such as gene editing, rare disease treatments, and artificial intelligence. These findings provide a theoretical basis for understanding the evolution of the gene therapy drug field.
Conclusion: Gene therapy drug research has entered a phase of rapid development. Emerging hotspots include AI-enabled vector design and the optimization of off-target effects. Future research holds great potential through the deep integration of molecular biology, materials science, and information technology. Nevertheless, challenges such as vector delivery efficiency, immunogenicity control, and scalable production processes remain the central to current research efforts and represent key areas for ongoing investigation.
, authors=Wan-ru JIA, Hui-ye WANG, Li DONG
*, authorsList=Wan-ru JIA, Hui-ye WANG, Li DONG, authorCompany=null, correspAuthors=Li DONG, authorNote=null, correspAuthorsNote=null, copyrightStatement=null, copyrightOwner=null, extLink=null, articleAbsUrl=null, sourceXml=null, magXml=null, pdfUrl=null, pdf=null, pdfFileSize=null, pdfExtLink=null, richHtmlUrl=null, mobilePdfUrl=null, reviewReport=null, pdfFirstPage=null, abstractGraph=null, abstractGraphContent=null, abstractVideo=null, citation=null, cebUrl=null, magXmlContent=null, mapNumber=null, fund=null), CN=ArticleExt(id=1321425815179473150, articleId=1321425814650990844, tenantId=1146029695717560320, journalId=1235980733773295621, language=CN, title=基于文献计量分析的国内外基因治疗药物研究现状对比, columnId=1236331162679955859, journalTitle=中国新药杂志, columnName=新药述评与论坛, runingTitle=null, highlight=null, articleAbstract=
目的： 基因治疗是通过修复或替换缺陷基因从而治疗疾病的方法，全面了解国内外基因治疗药物研究的发展脉络、现状及热点，对于促进该领域的技术创新和成果转化具有重要意义。
方法： 本研究检索了中国知网（CNKI）、Web of Science核心数据库、ClinicalTrial.gov临床实验数据，纳入1 890篇中文文献、7 598篇英文文献、1 806条临床试验数据，运用VOSviewer和CiteSpace等软件对基因治疗药物领域现状及发展进行可视化分析。
结果： 该领域研究呈持续增长态势，国内研究热点集中于CRISPR-Cas9基因编辑技术应用、纳米载体开发及肿瘤靶向治疗等方向；近年国际研究在基因编辑、罕见病治疗及人工智能等探索性技术方向呈较高关注，这些发现为理解基因治疗药物领域发展提供理论依据。
结论： 基因治疗药物研究已进入快速发展阶段，其中人工智能赋能的载体设计与脱靶效应优化正在成为新兴热点，未来在深度融合分子生物学、材料科学与信息技术等领域研究大有可为。同时，载体递送效率、免疫原性控制和规模化生产工艺仍是当前研究的核心挑战与重点方向。
, authors=贾婉茹, 王惠叶, 董丽
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贾婉茹，女，硕士研究生，研究方向：药事管理、药品知识产权。E-mail: j1750826446@163.com。
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贾婉茹，女，硕士研究生，研究方向：药事管理、药品知识产权。E-mail: j1750826446@163.com。
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王惠叶，女，硕士研究生，研究方向：工商管理、医药企业管理。E-mail: 18289803726@163.com。
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王惠叶，女，硕士研究生，研究方向：工商管理、医药企业管理。E-mail: 18289803726@163.com。
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