Regenerative medicine最新文献_第7页

Industry updates from the field of stem cell research and regenerative medicine in June 2025. 2025年6月来自干细胞研究和再生医学领域的行业更新。

IF 3 4区医学

Regenerative medicine Pub Date : 2025-10-01 Epub Date: 2025-07-31 DOI: 10.1080/17460751.2025.2542038

Dusko Ilic, Mirjana Liovic

引用次数: 0

Stem cell-derived exosomes in wound healing: mechanistic insights and delivery strategies. 干细胞来源的外泌体在伤口愈合：机制的见解和递送策略。

IF 3 4区医学

Regenerative medicine Pub Date : 2025-10-01 Epub Date: 2025-09-29 DOI: 10.1080/17460751.2025.2561449

Divya Kakade, Shantanu Date, Vinita Patole, Ganesh Ingavle, Surekha K Satpute, Avinash Sanap, Ajinkya Aher, Pawan Karwa, Prabhanjan Giram

{"title":"Stem cell-derived exosomes in wound healing: mechanistic insights and delivery strategies.","authors":"Divya Kakade, Shantanu Date, Vinita Patole, Ganesh Ingavle, Surekha K Satpute, Avinash Sanap, Ajinkya Aher, Pawan Karwa, Prabhanjan Giram","doi":"10.1080/17460751.2025.2561449","DOIUrl":"10.1080/17460751.2025.2561449","url":null,"abstract":"Mesenchymal stem cells (MSCs) contribute significantly to wound healing due to their ability to self-renew, modulate immune responses, and differentiate into various cell types. However, challenges such as unpredictable growth, limited vascular transport efficiency, stringent storage and maintenance requirements that limit the widespread clinical use of MSC-based therapy, highlighting the need for developing effective cell-free alternatives. The regenerative effects of MSCs are mediated through paracrine signaling, primarily via their secretome, which includes extracellular vesicles and soluble factors, especially exosomes. Compared to MSC therapy, exosomes provide superior benefits in terms of storage, safety, and efficiency in targeting the wound sites due to their enhanced tissue penetration capabilities. However, a specific aspect that remains underexplored in exosome-based therapy for wound healing is the development of optimized delivery systems, to ensure controlled, sustained release and precise localization of the exosomes at the wound sites. This review uniquely focuses on this critical and emerging area, providing a detailed overview of the current advancements and limitations in exosomes-based wound healing therapies, with a focus on their delivery strategies. The insights presented in this review are expected to accelerate the development of innovative, effective treatments, revolutionizing wound care management and advancing regenerative medicine in clinical practice.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"527-553"},"PeriodicalIF":3.0,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12562669/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145186538","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Allogeneic mesenchymal stromal cell injections for knee osteoarthritis: a review of clinical outcomes. 同种异体间充质细胞注射治疗膝关节骨关节炎：临床结果综述。

IF 3 4区医学

Regenerative medicine Pub Date : 2025-10-01 Epub Date: 2025-10-18 DOI: 10.1080/17460751.2025.2572202

Shelby Johnson, Brennan J Boettcher, Jasmijn V Korpershoek, Mario Hevesi, Daniel B F Saris, Christopher V Nagelli

{"title":"Allogeneic mesenchymal stromal cell injections for knee osteoarthritis: a review of clinical outcomes.","authors":"Shelby Johnson, Brennan J Boettcher, Jasmijn V Korpershoek, Mario Hevesi, Daniel B F Saris, Christopher V Nagelli","doi":"10.1080/17460751.2025.2572202","DOIUrl":"10.1080/17460751.2025.2572202","url":null,"abstract":"Mesenchymal stromal cell (MSC) therapy is an injectable, orthobiologic treatment for patients with knee osteoarthritis (OA). The purpose of this narrative review is to evaluate the literature on allogeneic MSCs used in knee OA patients and to report on clinical outcomes. A comprehensive literature search was performed using the following keywords: osteoarthritis, knee, stem cell transplantation, allogeneic or allogenic, human mesenchymal stem cells, and human mesenchymal stromal cells. There was no restriction on time, and subjective and objective clinical outcomes were reported. A total of 11 (n = 11) clinical trial studies were included. The most common allogeneic source was adipose tissue (AD-MSCs). Most studies included a control group and included participants with Kellgren-Lawrence grades of II-III or II-IV. The MSC dose used was highly variable. There were no significant adverse safety events. All studies reported an improvement in patient reported outcomes from baseline, with the most common observation being a durable reduction in pain for 6 to 24 months. There were positive effects of AD-MSCs on cartilage imaging in most studies, although outcomes were variable. Allogeneic MSC injections for knee OA resulted in improved OA related symptom scores, are safe, and provided patients with a reduction in pain at long-term follow-up.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"501-507"},"PeriodicalIF":3.0,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12562666/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145313584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Translational progress in the development of pharmacotherapies for Duchenne muscular dystrophy. 杜氏肌营养不良症药物治疗的研究进展。

IF 3 4区医学

Regenerative medicine Pub Date : 2025-10-01 Epub Date: 2025-10-08 DOI: 10.1080/17460751.2025.2571355

Kristy Swiderski, Gordon S Lynch

{"title":"Translational progress in the development of pharmacotherapies for Duchenne muscular dystrophy.","authors":"Kristy Swiderski, Gordon S Lynch","doi":"10.1080/17460751.2025.2571355","DOIUrl":"10.1080/17460751.2025.2571355","url":null,"abstract":"Despite the discovery, nearly 40 years ago, that mutations in the dystrophin gene were responsible for Duchenne muscular dystrophy (DMD), a cure for this devastating disease remains elusive. Considerable effort worldwide is focused on understanding DMD and devising treatments, including gene-, cell-, and pharmacologic-based therapies. More than 400 clinical trials for DMD and/or the related Becker muscular dystrophy (BMD) have been registered with clinicaltrials.gov, with many in various stages of completion, and more than 40 having been terminated or withdrawn. The failure of interventions in clinical trials represents a significant emotional burden for the entire DMD community. While some gene-based therapies are being approved, these can be expensive, and currently tend to target specific mutations. Several cell-based therapies and tissue engineering strategies are also currently in development. Of the many pharmacotherapies to address aspects of the pathophysiology of DMD, like preserving muscle fibers, enhancing regeneration, and increasing strength, glucocorticoids remain the most efficacious for attenuating the disease progression. Successful pharmacotherapies may enable patients to take advantage of perfected gene therapies when they eventually become available. Here, we explore the therapeutic merit of different pharmacotherapies currently under consideration and provide an update on recent advances in gene therapies for DMD.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"489-500"},"PeriodicalIF":3.0,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12562787/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145252552","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Effect of mesenchymal stem cell conditioned medium on hepatocyte matrix implant to alleviate liver cirrhosis in rats. 间充质干细胞条件培养基对肝细胞基质移植缓解大鼠肝硬化的影响。

IF 3 4区医学

Regenerative medicine Pub Date : 2025-10-01 Epub Date: 2025-10-14 DOI: 10.1080/17460751.2025.2574178

Siufui Hendrawan, Jennifer Lheman, Fitriya Nur Annisa Dewi, Nuraeni, Permanawati, Hans Ulrich Baer

{"title":"Effect of mesenchymal stem cell conditioned medium on hepatocyte matrix implant to alleviate liver cirrhosis in rats.","authors":"Siufui Hendrawan, Jennifer Lheman, Fitriya Nur Annisa Dewi, Nuraeni, Permanawati, Hans Ulrich Baer","doi":"10.1080/17460751.2025.2574178","DOIUrl":"10.1080/17460751.2025.2574178","url":null,"abstract":"Background: Hepatocyte transplantation has gained importance as an alternative treatment to orthotopic liver transplantation for end-stage liver disease. This study explores the modification of the hepatocyte matrix implant (HMI) procedure by replacing islet cells with conditioned medium (CM) derived from human umbilical cord-MSC (hUC-MSC) supplementation to alleviate liver cirrhosis in a rat model.Methods: The male Sprague Dawley rats were induced liver cirrhosis using thioacetamide for 11 weeks, and implanted with matrices on the small bowel mesentery, according to the groups. Four groups were assessed: blank matrix (cell-free), hepatocytes seeded-matrix (Hep), hepatocytes+islets co-seeded-matrix (Hep:Islet), and hepatocytes seeded-matrix supplemented with CM (Hep+CM).Results: In vitro, the Hep+CM group showed significantly higher hepatocyte proliferation than the Hep:Islet group, though albumin production was similar. The in vivo study further confirmed that the implanted hepatocytes remained viable and were able to produce albumin for at least 4 months post-implantation. Liver function parameters were shown to be improved in Hep:Islet and Hep+CM groups. Notably, collagen deposition in the liver was lower in Hep:Islet and Hep+CM groups compared to other groups.Conclusion: These findings suggest that CM can effectively replace islet in supporting hepatocyte proliferation and function, enhancing the therapeutic potential of HMI procedure. No writing assistance was utilized in the production of this manuscript or liver cirrhosis treatment.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"475-487"},"PeriodicalIF":3.0,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12562743/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145286770","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

One down but many more to go: the state of gene therapy for inherited retinal disease. 一项研究已经结束，但还有很多有待研究：遗传性视网膜疾病的基因治疗现状。

IF 3 4区医学

Regenerative medicine Pub Date : 2025-10-01 Epub Date: 2025-10-06 DOI: 10.1080/17460751.2025.2571360

Tien-En Tan, Christopher Z Y Sun, Stanley S J Poh, Joshua Lim, Jasmin X J Teo, Sonali Dey, Zachary W X Chua, Jing Guo, Zhenxun Wang, Hwee Goon Tay, Beau J Fenner

{"title":"One down but many more to go: the state of gene therapy for inherited retinal disease.","authors":"Tien-En Tan, Christopher Z Y Sun, Stanley S J Poh, Joshua Lim, Jasmin X J Teo, Sonali Dey, Zachary W X Chua, Jing Guo, Zhenxun Wang, Hwee Goon Tay, Beau J Fenner","doi":"10.1080/17460751.2025.2571360","DOIUrl":"10.1080/17460751.2025.2571360","url":null,"abstract":"Gene therapy has ushered in a new era for the treatment of inherited retinal diseases (IRDs). The approval of voretigene neparvovec-rzyl (Luxturna) for RPE65-associated retinal dystrophy marked a pivotal milestone, establishing proof-of-concept that gene addition can restore visual function in IRDs. However, the success of Luxturna is tempered by the reality that it applies to a narrow subset of IRDs, and that no other IRD gene therapy has thus far received regulatory approval. This review outlines the current landscape of IRD gene therapy, including trials for several forms of IRD including achromatopsia, choroideremia, Leber congenital amaurosis, X-linked retinitis pigmentosa, and X-linked retinoschisis. We highlight the central challenges facing the field: narrow gene- or variant-specific indications, vector limitations, and reliance on suboptimal clinical trial endpoints. The review also discusses emerging strategies - including dual AAV and split-intein vectors, non-viral delivery platforms, and precision gene editing technologies such as CRISPR, base editing, and prime editing. These innovations promise to expand therapeutic reach. Finally, we emphasize the need for improved regulatory frameworks and ethical considerations for gene-based therapies for IRD. The field now stands at a critical juncture, where the lessons of Luxturna can inform a more scalable, inclusive, and transformative future.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"509-526"},"PeriodicalIF":3.0,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12562747/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145239429","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Comparison of 3D culture systems on mesenchymal stem cell longevity, regenerative potential, and secretome production. 3D培养系统对间充质干细胞寿命、再生潜能和分泌组产生的影响。

IF 3 4区医学

Regenerative medicine Pub Date : 2025-10-01 Epub Date: 2025-10-12 DOI: 10.1080/17460751.2025.2572177

Jacob Hodge, Aidyn Medina-Lopez, Christopher Neal, Heather Decker, Jennifer Robinson, Adam J Mellott

{"title":"Comparison of 3D culture systems on mesenchymal stem cell longevity, regenerative potential, and secretome production.","authors":"Jacob Hodge, Aidyn Medina-Lopez, Christopher Neal, Heather Decker, Jennifer Robinson, Adam J Mellott","doi":"10.1080/17460751.2025.2572177","DOIUrl":"10.1080/17460751.2025.2572177","url":null,"abstract":"Background/aims: Mesenchymal stem/stromal cells (MSCs) are widely investigated for regenerative therapies, yet current expansion methods often compromise their stem-like properties, limiting large-scale clinical translation. We aimed to evaluate whether a novel hydrogel-based Bio-Block® platform preserved intrinsic adipose-derived MSC (ASCs) phenotype and secretome compared with conventional systems.Materials/methods: ASCs were cultured for four weeks in 2D, spheroids, Matrigel, or Bio-Blocks. Cultures were assessed for proliferation, senescence, apoptosis, trilineage differentiation, stem-like gene expression, secretome protein and extracellular vesicle (EV) production, and EV potency on endothelial cells (ECs).Results: Bio-Block ASCs exhibited ~2-fold higher proliferation than spheroid and Matrigel groups, with senescence reduced 30-37% and apoptosis decreased 2-3-fold. Trilineage differentiation and stem-like markers (e.g. LIF, OCT4, IGF1) were significantly higher in Bio-Block ASCs. Secretome protein declined 35%, 47%, and 10% in 2D, spheroid, and Matrigel, respectively, but was preserved in Bio-Blocks. Similarly, EV production increased ~44% in Bio-Blocks, while other systems declined 30-70%. Bio-Block EVs enhanced EC proliferation, migration, and VE-cadherin expression, whereas spheroid EVs induced senescence and apoptosis.Conclusion: This study highlights the critical influence of culture systems on scaling robust MSC-based therapies and introduces a biomimetic platform that represents a potential scalable strategy for producing high-potency and robust regenerative therapies.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"455-474"},"PeriodicalIF":3.0,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12562725/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145281025","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Controlled Codelivery of Hydrogen Sulfide and Nitric Oxide for the Treatment of Full-Thickness Skin Wound 控制硫化氢和一氧化氮共递送治疗全层皮肤创面

4区医学

Regenerative medicine Pub Date : 2025-09-19 DOI: 10.53941/rmd.2025.100013

Qiang Zhao, Jiansong Cheng, Huan Wang, Zhixin Xu, Weiliang Deng, Yating Zhang, Pei Liu, Guangbo Ji, Meng Qian

{"title":"Controlled Codelivery of Hydrogen Sulfide and Nitric Oxide for the Treatment of Full-Thickness Skin Wound","authors":"Qiang Zhao, Jiansong Cheng, Huan Wang, Zhixin Xu, Weiliang Deng, Yating Zhang, Pei Liu, Guangbo Ji, Meng Qian","doi":"10.53941/rmd.2025.100013","DOIUrl":"https://doi.org/10.53941/rmd.2025.100013","url":null,"abstract":"Chronic wounds present significant clinical challenges stemming from persistent inflammation, impaired angiogenesis, and disrupted tissue homeostasis. Nitric oxide (NO) and hydrogen sulfide (H2S) are important gaseous signalling molecules that play critical regulatory roles in the pathophysiology of numerous diseases. In the present study, we designed a novel prodrug, TSNO, which releases H2S and NO in a controlled manner under the catalysis of GSH and the engineered enzyme A4-β-GalH363A. Furthermore, a functional wound dressing was prepared by incorporating TSNO into an electrospun poly(ε-caprolactone) (PCL) mat, and the therapeutic efficacy was evaluated in a full-thickness skin wound model established in mice. Results showed that the functional wound dressing effectively accelerated the wound healing process by enhancing collagen synthesis, promoting angiogenesis, and regulating inflammation via controlled codelivery of H2S and NO. Taken together, the functional wound dressing developed in this study represents a promising candidate for treating large-scale skin wounds in the clinic.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":"2 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-09-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147334222","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Engineering a cell-free bone regeneration platform using osteogenically primed MSC-EVs and nHAp-enriched IPN hydrogels. 利用成骨引物msc - ev和富含nhap的IPN水凝胶构建无细胞骨再生平台。

IF 3 4区医学

Regenerative medicine Pub Date : 2025-09-01 Epub Date: 2025-09-08 DOI: 10.1080/17460751.2025.2557770

Ketki Holkar, Prasad Pethe, Vaijayanti Kale, Ganesh Ingavle

{"title":"Engineering a cell-free bone regeneration platform using osteogenically primed MSC-EVs and nHAp-enriched IPN hydrogels.","authors":"Ketki Holkar, Prasad Pethe, Vaijayanti Kale, Ganesh Ingavle","doi":"10.1080/17460751.2025.2557770","DOIUrl":"10.1080/17460751.2025.2557770","url":null,"abstract":"Aims: This study aimed to enhance the osteoinductive potential of mesenchymal stem cell-derived extracellular vesicles (MSC-EVs) by integrating them into a nano-hydroxyapatite (nHAp)-enriched hydrogel scaffold for bone regeneration applications.Materials & methods: EVs were isolated from naïve and osteogenically primed MSCs and characterized for morphology, cargo content, and cytocompatibility. Their uptake and osteoinductive activity were assessed in vitro using MC3T3 cells within a 3D interpenetrating network (IPN) hydrogel. The most effective EV formulation was incorporated into an nHAp - IPN hydrogel scaffold and evaluated both in vitro and in a murine subcutaneous implantation model.Results: Primed MSC-EVs showed elevated calcium, ALP activity, and osteogenic/angiogenic mRNAs (Runx2, Vegf-a) compared to naïve EVs, with comparable size and morphology. Both EV types were internalized efficiently without cytotoxicity. In combination with nHAp, primed EVs enhanced ALP activity, calcium deposition, and in vivo mineralization. Histological analysis confirmed scaffold biocompatibility and mineralized tissue formation.Conclusions: Osteogenically primed MSC-EVs significantly improved the osteoinductive performance of nHAp-based hydrogels, supporting their potential as a cell-free therapeutic strategy for bone tissue engineering.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"375-386"},"PeriodicalIF":3.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12502811/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145016193","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

CRISPR and Myelin regeneration: a systematic review of applications in demyelinating CNS Disorders, with a focus on MS. CRISPR和髓鞘再生：脱髓鞘性中枢神经系统疾病应用的系统综述，重点是MS。

IF 3 4区医学

Regenerative medicine Pub Date : 2025-09-01 Epub Date: 2025-09-19 DOI: 10.1080/17460751.2025.2561451

Aliakbar Mariki, Kristi Anne Kohlmeier, Seyed Mohammad Mousavi, Mohammad Shabani

{"title":"CRISPR and Myelin regeneration: a systematic review of applications in demyelinating CNS Disorders, with a focus on MS.","authors":"Aliakbar Mariki, Kristi Anne Kohlmeier, Seyed Mohammad Mousavi, Mohammad Shabani","doi":"10.1080/17460751.2025.2561451","DOIUrl":"10.1080/17460751.2025.2561451","url":null,"abstract":"Aims: Current treatments for demyelinating disorders focus on slowing progression but fail to repair damaged myelin. Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) -based technology has the potential to address key challenges in myelin repair by targeting genetic dysfunctions, modulating immune responses, and promoting oligodendrocyte differentiation. This systematic review aimed to evaluate CRISPR applications for myelin regeneration.Methods: A comprehensive search of PubMed, Scopus, and other databases identified 48 studies. The included studies employed CRISPR in diverse experimental models, targeting genes associated with immune regulation and astrocyte activity, as well as correcting RNA splicing dysfunctions linked to neurodegeneration.Results: CRISPR-edited stem cells showed significant potential in promoting myelin regeneration, with enhanced functional recovery in animal models of multiple sclerosis (MS). While most research focused on MS, promising applications were also observed in neuromyelitis optica spectrum disorder (NMOSD), such as reducing astrocytic damage via AQP4 targeting, and in progressive multifocal leukoencephalopathy (PML), where CRISPR disrupted JC polyomavirus replication.Conclusions: Despite its promise, challenges remain. Future research should prioritize optimizing CRISPR delivery systems, expanding applications to underexplored disorders, and conducting long-term safety assessments. Early results are encouraging, but further studies are essential to translate preclinical success into clinical therapies.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"431-443"},"PeriodicalIF":3.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12502815/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145092329","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0