Regenerative medicine最新文献

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 2.6 4区医学

Regenerative medicine Pub 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":"https://doi.org/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with clinicaltrials.gov, with many in various stages of completion, and more than 40 having been terminated or withdrawn.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":"1-12"},"PeriodicalIF":2.6,"publicationDate":"2025-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","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 2.6 4区医学

Regenerative medicine Pub 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":"https://doi.org/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":"1-18"},"PeriodicalIF":2.6,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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 2.6 4区医学

Regenerative medicine Pub 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":"https://doi.org/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":"1-27"},"PeriodicalIF":2.6,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","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 2.6 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":2.6,"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 2.6 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":2.6,"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

The effects of polynucleotides-based biomimetic hydrogels in tissue repair: a 2D and 3D in vitro study. 基于多核苷酸的仿生水凝胶在组织修复中的作用：2D和3D体外研究。

IF 2.6 4区医学

Regenerative medicine Pub Date : 2025-09-01 Epub Date: 2025-09-29 DOI: 10.1080/17460751.2025.2567177

Maria Teresa Colangelo, Stefano Guizzardi, Luana Laschera, Marco Meleti, Carlo Galli

{"title":"The effects of polynucleotides-based biomimetic hydrogels in tissue repair: a 2D and 3D in vitro study.","authors":"Maria Teresa Colangelo, Stefano Guizzardi, Luana Laschera, Marco Meleti, Carlo Galli","doi":"10.1080/17460751.2025.2567177","DOIUrl":"10.1080/17460751.2025.2567177","url":null,"abstract":"Introduction: Biomimetics offers promising tools to improve wound healing in difficult clinical conditions. Polynucleotides (PN) show high potential for tissue repair in oral and periodontal surgery, by relying on the body's inherent self-healing capabilities. The aim of the present study was to elucidate in vitro the effects of Odonto-PN (O-PN) and Regenfast (REG), two PN-based compounds, on oral tissue repair.Methods: We employed 3D spheroid cultures and cell scratch assays to simulate wound healing in vitro, assessing cell migration and morphology under normal conditions and following mitomycin-induced inhibition of cell growth.Results: Both O-PN and REG supported early cell viability and spheroid disassembly. O-PN supported the initial outgrowth of fibroblasts, whereas REG enhanced sustained cell migration at later time points. In scratch assays, REG effectively facilitated defect closure - even under mitomycin treatment - and induced a more elongated, migratory cell phenotype.Conclusions: These findings suggest that both O-PN and REG can favorably modulate fibroblast function to support wound repair. While O-PN fosters early activation and cell viability, REG exerts potent pro-migratory effects that may be particularly useful for complex periodontal regeneration. Their selective use could provide valuable adjuncts in clinical protocols aimed at restoring delicate oral structures, such as the interdental papillae.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"365-373"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12502828/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145192505","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

Industry updates from the field of stem cell research and regenerative medicine in May 2025. 2025年5月来自干细胞研究和再生医学领域的行业更新。

IF 2.6 4区医学

Regenerative medicine Pub Date : 2025-09-01 Epub Date: 2025-06-29 DOI: 10.1080/17460751.2025.2525719

Dusko Ilic, Mirjana Liovic

引用次数: 0

Building the framework for bioprinted human heart tissue: recent developments and future prospects. 构建生物打印人类心脏组织的框架：最近的发展和未来前景。

IF 2.6 4区医学

Regenerative medicine Pub Date : 2025-09-01 Epub Date: 2025-09-11 DOI: 10.1080/17460751.2025.2558269

Victor A da Silva, Man Chi Leung, McGregor Clayton, Leya Oommen, Hannia Madrigal, Zachary Laksman, Bosco Yu, Stephanie M Willerth

引用次数: 0

LGBTQ+ persons, queer bioethics, and inclusivity in stem cell research and regenerative medicine. LGBTQ+人群，酷儿生物伦理，以及干细胞研究和再生医学的包容性。

IF 2.6 4区医学

Regenerative medicine Pub Date : 2025-09-01 Epub Date: 2025-09-22 DOI: 10.1080/17460751.2025.2561461

Sydney E Prange, Leigh Turner

{"title":"LGBTQ+ persons, queer bioethics, and inclusivity in stem cell research and regenerative medicine.","authors":"Sydney E Prange, Leigh Turner","doi":"10.1080/17460751.2025.2561461","DOIUrl":"10.1080/17460751.2025.2561461","url":null,"abstract":"Despite the increasing visibility of the LGBTQ+ (Lesbian, Gay, Bisexual, Transgender, Queer, and other expansive genders or sexual orientations) community, LGBTQ+ persons continue to face significant barriers, discrimination, and stigmatization in the health care space. The unique challenges faced by LGBTQ+ persons in healthcare led bioethicists to develop queer bioethics. This approach to ethical reflection suggests that we should develop understanding of biomedical and biological topics related to LGBTQ+ persons and that we should examine topics not traditionally associated with LGBTQ+ persons through a queer lens. Here, we argue that queer bioethics provides valuable new perspectives for improving stem cell science. We examine specific issues that should be examined using a queer bioethical framework for the benefit of all persons and the overall advancement of stem cell science. We specifically address strategies for stem cell donation recruitment, creation of more comprehensive stem cell models for precision medicine, and the promotion of more inclusive practices for LGBTQ+ persons in the stem cell science workforce. We contend that these important and timely topics provide suitable starting points for applying a queer bioethical perspective to stem cell research and regenerative medicine. Furthermore, we underscore the importance of addressing these topics in the current political climate.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"387-397"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12502810/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145125917","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

Stem cell-derived extracellular vesicles as a therapeutic for avascular necrosis: current status and future prospects. 干细胞来源的细胞外囊泡治疗缺血性坏死：现状和未来前景。

IF 2.6 4区医学

Regenerative medicine Pub Date : 2025-09-01 Epub Date: 2025-09-23 DOI: 10.1080/17460751.2025.2561454

Vaijayanti Kale

{"title":"Stem cell-derived extracellular vesicles as a therapeutic for avascular necrosis: current status and future prospects.","authors":"Vaijayanti Kale","doi":"10.1080/17460751.2025.2561454","DOIUrl":"10.1080/17460751.2025.2561454","url":null,"abstract":"Avascular necrosis (AVN), also referred to as osteonecrosis (ON), is a major clinical challenge in orthopedic practice. Current treatment strategies include surgical options such as core decompression, as well as non-surgical approaches including statin therapy, weight reduction, and physiotherapy. Regenerative therapies - such as platelet-rich plasma injections, autologous bone marrow cell concentrates, and mesenchymal stem/stromal cells (MSCs), among others have shown some success. Although induced pluripotent stem cells (iPSCs) represent a promising source for cell therapy, their clinical application is restricted due to the risk of teratoma formation. In this context, the therapeutic potential of extracellular vesicles (EVs) secreted by stem cells has emerged as a relatively new area of investigation. This review summarizes findings from preclinical studies in animal models that have explored the use of MSC- and iPSC-derived EVs in the regenerative treatment of AVN/ON. Compared with MSC-EVs, the therapeutic use of iPSC-EVs has progressed more slowly, partly due to the high cost of expanding iPSCs to obtain a sufficient quantity of their EVs. Therefore, instead of using iPSC-derived EVs, the use of a cocktail of EVs secreted by iPSC-derived cellular derivatives may represent a safer, more cost-effective, and potentially more efficacious strategy for treating AVN.","PeriodicalId":21043,"journal":{"name":"Regenerative medicine","volume":" ","pages":"399-408"},"PeriodicalIF":2.6,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145125929","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0