Article(id=1321501055590970316, tenantId=1146029695717560320, journalId=1246415837536497731, issueId=1321500868936052946, articleNumber=PA20260310_NEkYkoJV, orderNo=null, doi=10.12307/2026.508, 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=1741104000000, acceptedDateStr=2025-03-05, onlineDate=1748534400000, onlineDateStr=2025-05-30, pubDate=1767801600000, pubDateStr=2026-01-08, doiRegisterDate=null, doiRegisterDateStr=null, onlineIssueDate=1793000003954, onlineIssueDateStr=2026-10-26, onlineJustAcceptDate=null, onlineJustAcceptDateStr=null, onlineFirstDate=null, onlineFirstDateStr=null, sourceXml=null, magXml=null, createTime=1793000003954, creator=admin, updateTime=1793000003954, updator=admin, issue=Issue{id=1321500868936052946, tenantId=1146029695717560320, journalId=1246415837536497731, year='2026', volume='30', issue='1', pageStart='null', pageEnd='null', issueExtLink='null', onlineDate='null', pubDate='1767801600000', pubDateStr='2026-01-08', beforeIssueId=null, nextIssueId=null, price=null, status=1, issueComplete=0, articleOrder=1, issueType=-1, specialIssue=null, createTime=1792999959453, creator='ztmeta', updateTime=1792999959453, updator='ztmeta', preIssue=null, nextIssue=null, articleTotal=null, ext=null, issueFiles=null, downloadFileDto=null}, startPage=145, endPage=152, ext={EN=ArticleExt(id=1321501058166272974, articleId=1321501055590970316, tenantId=1146029695717560320, journalId=1246415837536497731, language=EN, title=Regulation of lysosome function by stem cells in treatment of lysosomal storage diseases, columnId=null, journalTitle=Chinese Journal of Tissue Engineering Research, columnName=null, runingTitle=null, highlight=null, articleAbstract=BACKGROUND: Lysosomal storage diseases, as a group of rare genetic metabolic disorders, exhibit complex pathogenesis often leading to dysfunction of cells, tissues, and organs. Current therapeutic approaches have certain limitations. Stem cell transplantation, as an emerging treatment method, offers new options for patients with lysosomal storage diseases. OBJECTIVE: To review the mechanisms of action of stem cells in regulating lysosomes for the treatment of lysosomal storage diseases and explore the feasibility of traditional Chinese medicine in treating such diseases, providing new insights for the treatment of lysosomal storage diseases with stem cells. METHODS: Relevant literature from 2010 to 2024 was searched in CNKI and PubMed databases using keywords “stem cells, lysosomal storage disease, lysosome” in English and Chinese. Ultimately, 78 articles were included for review and analysis. RESULTS AND CONCLUSION: (1) Stem cells treat lysosomal storage diseases by regulating lysosomes primarily through three aspects: regulating stem cell differentiation and replacement, improving intercellular communication and the microenvironment, and enhancing lysosomal enzyme expression through gene editing. (2) Stem cells have achieved significant effects in the treatment of some lysosomal storage diseases, such as Niemann-Pick disease, mucopolysaccharidoses, Gaucher disease, and metachromatic leukodystrophy. (3) The procedure for stem cell transplantation needs further optimization. Adverse reactions post-transplantation urgently need to be addressed, and the efficiency and safety of gene-modified stem cells also need to be further improved. In the future, more research on the treatment of lysosomal storage diseases with traditional Chinese medicine is required to reveal the relevant mechanisms for the treatment of lysosomal storage diseases with traditional Chinese medicine., authors=null, authorsList=null, authorCompany=null, correspAuthors=null, 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=1321501058078192589, articleId=1321501055590970316, tenantId=1146029695717560320, journalId=1246415837536497731, language=CN, title=干细胞调控溶酶体功能治疗溶酶体贮积症, columnId=null, journalTitle=中国组织工程研究, columnName=null, runingTitle=null, highlight=null, articleAbstract=背景:溶酶体贮积症是一组罕见的遗传代谢疾病,发病机制复杂,常导致细胞、组织及器官功能障碍,目前的治疗方法具有一定局限性。干细胞移植作为一种新兴的治疗方法,为溶酶体贮积症患者提供了新的选择。 目的:综述干细胞调控溶酶体治疗溶酶体贮积症的作用机制,探讨中医药治疗此类疾病的可行性,为干细胞及中医药治疗溶酶体贮积症提供新的思路。 方法:以“stem cells,lysosomal storage disease,lysosome” 为英文检索词,以“干细胞,溶酶体贮积症,溶酶体”为中文检索词,检索中国知网、PubMed数据库2010-2024年的相关文献,最终纳入78篇文献进行综述分析。 结果与结论:①干细胞调控溶酶体治疗溶酶体贮积症,主要是通过调控干细胞分化与替代、改善细胞间通讯与微环境、基因编辑增强溶酶体酶表达3个方面发挥作用;②干细胞在部分溶酶体贮积症如尼曼匹克病、黏多糖贮积症、戈谢病及异染性脑白质营养不良的治疗中取得了明显效果;③干细胞移植的程序需要进一步优化,移植后的不良反应亟待解决,基因修饰干细胞的效率和安全性也需要进一步提高,未来还需要更多中医药治疗溶酶体贮积症的相关研究,揭示中医药治疗溶酶体贮积症的相关机制。, authors=null, authorsList=null, authorCompany=null, correspAuthors=null, authorNote=null, correspAuthorsNote=null, copyrightStatement=null, copyrightOwner=null, extLink=null, articleAbsUrl=null, sourceXml=null, magXml=null, 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干细胞调控溶酶体功能治疗溶酶体贮积症
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李怡文 1, , 刘飞祥 2, , 张运克 2,,1,
中国组织工程研究 | 2026,30(1): 145-152
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中国组织工程研究 2026 , 30 (1) : 145 -152
干细胞调控溶酶体功能治疗溶酶体贮积症
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李怡文1, , 刘飞祥2,, 张运克2,,1,
作者信息
  • 1.南中医药大学第一临床医学院,河南省郑州市 450046
  • 2.南中医药大学第一附属医院脑病科,河南省郑州市 450003
  • 3.南中医药大学第一附属医院脑病科,河南省郑州市 450003
  • 4.南中医药大学第一临床医学院,河南省郑州市 450046
Regulation of lysosome function by stem cells in treatment of lysosomal storage diseases
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出版时间: 2026-01-08 doi: 10.12307/2026.508
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背景:溶酶体贮积症是一组罕见的遗传代谢疾病,发病机制复杂,常导致细胞、组织及器官功能障碍,目前的治疗方法具有一定局限性。干细胞移植作为一种新兴的治疗方法,为溶酶体贮积症患者提供了新的选择。 目的:综述干细胞调控溶酶体治疗溶酶体贮积症的作用机制,探讨中医药治疗此类疾病的可行性,为干细胞及中医药治疗溶酶体贮积症提供新的思路。 方法:以“stem cells,lysosomal storage disease,lysosome” 为英文检索词,以“干细胞,溶酶体贮积症,溶酶体”为中文检索词,检索中国知网、PubMed数据库2010-2024年的相关文献,最终纳入78篇文献进行综述分析。 结果与结论:①干细胞调控溶酶体治疗溶酶体贮积症,主要是通过调控干细胞分化与替代、改善细胞间通讯与微环境、基因编辑增强溶酶体酶表达3个方面发挥作用;②干细胞在部分溶酶体贮积症如尼曼匹克病、黏多糖贮积症、戈谢病及异染性脑白质营养不良的治疗中取得了明显效果;③干细胞移植的程序需要进一步优化,移植后的不良反应亟待解决,基因修饰干细胞的效率和安全性也需要进一步提高,未来还需要更多中医药治疗溶酶体贮积症的相关研究,揭示中医药治疗溶酶体贮积症的相关机制。
溶酶体贮积症  /  溶酶体  /  干细胞  /  造血干细胞移植  /  中医药  /  中药  /  复方  /  研究进展
BACKGROUND: Lysosomal storage diseases, as a group of rare genetic metabolic disorders, exhibit complex pathogenesis often leading to dysfunction of cells, tissues, and organs. Current therapeutic approaches have certain limitations. Stem cell transplantation, as an emerging treatment method, offers new options for patients with lysosomal storage diseases. OBJECTIVE: To review the mechanisms of action of stem cells in regulating lysosomes for the treatment of lysosomal storage diseases and explore the feasibility of traditional Chinese medicine in treating such diseases, providing new insights for the treatment of lysosomal storage diseases with stem cells. METHODS: Relevant literature from 2010 to 2024 was searched in CNKI and PubMed databases using keywords “stem cells, lysosomal storage disease, lysosome” in English and Chinese. Ultimately, 78 articles were included for review and analysis. RESULTS AND CONCLUSION: (1) Stem cells treat lysosomal storage diseases by regulating lysosomes primarily through three aspects: regulating stem cell differentiation and replacement, improving intercellular communication and the microenvironment, and enhancing lysosomal enzyme expression through gene editing. (2) Stem cells have achieved significant effects in the treatment of some lysosomal storage diseases, such as Niemann-Pick disease, mucopolysaccharidoses, Gaucher disease, and metachromatic leukodystrophy. (3) The procedure for stem cell transplantation needs further optimization. Adverse reactions post-transplantation urgently need to be addressed, and the efficiency and safety of gene-modified stem cells also need to be further improved. In the future, more research on the treatment of lysosomal storage diseases with traditional Chinese medicine is required to reveal the relevant mechanisms for the treatment of lysosomal storage diseases with traditional Chinese medicine.
lysosomal storage diseases  /  lysosome  /  stem cells  /  hematopoietic stem cell transplantation  /  traditional Chinese medicine  /  Chinese herbal medicine  /  compound prescription  /  research progress
李怡文, 刘飞祥, 张运克. 干细胞调控溶酶体功能治疗溶酶体贮积症. 中国组织工程研究, 2026 , 30 (1) : 145 -152 . DOI: 10.12307/2026.508
. Regulation of lysosome function by stem cells in treatment of lysosomal storage diseases[J]. Chinese Journal of Tissue Engineering Research, 2026 , 30 (1) : 145 -152 . DOI: 10.12307/2026.508
  • 国家自然科学基金项目
  • 国家自然科学基金项目
  • 中原英才计划——科技创新领军人才项目——河南省科技厅
  • 河南省“双一流”创建学科中医学科学研究专项
  • 河南省中医药科学研究专项课题
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2026年第30卷第1期
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doi: 10.12307/2026.508
  • 首发时间:2025-05-30
  • 出版时间:2026-01-08
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  • 录用日期:2025-03-05
基金
国家自然科学基金项目
国家自然科学基金项目
中原英才计划——科技创新领军人才项目——河南省科技厅
河南省“双一流”创建学科中医学科学研究专项
河南省中医药科学研究专项课题
作者信息
    1.南中医药大学第一临床医学院,河南省郑州市 450046
    2.南中医药大学第一附属医院脑病科,河南省郑州市 450003
    3.南中医药大学第一附属医院脑病科,河南省郑州市 450003
    4.南中医药大学第一临床医学院,河南省郑州市 450046

通讯作者:

张运克: 河南中医药大学第一附属医院,河南省郑州市 450046; 河南中医药大学,河南省郑州市 450046 Corresponding author: Zhang Yunke, PhD, Professor, Chief physician, Doctoral supervisor, Post-doctoral co-supervisor, First Affiliated Hospital of Henan University of Chinese Medicine, Zhengzhou 450046, Henan Province, China; Henan University of Chinese Medicine, Zhengzhou 450046, Henan Province, China
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鹅膏菌科Amanitaceae 2 11 5.26 鹅膏菌属 Amanita 10 4.78
小菇科 Mycenaceae 2 12 5.74 丝盖伞属 Inocybe 5 2.39
多孔菌科 Polyporaceae 8 14 6.70 蜡蘑属 Laccaria 5 2.39
红菇科 Russulaceae 3 23 11.00 小皮伞属 Marasmius 6 2.87
小菇属 Mycena 11 5.26
光柄菇属 Pluteus 5 2.39
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