Expert Reviews in Molecular Medicine最新文献

{"title":"Cell therapy and autoimmune diseases: application prospects and challenges.","authors":"Wenqin He, Xinying Fan, Baiyan Zhang, Haodong Su, Yangyang Lu, Lizhong Zhang, Dan Ma","doi":"10.1017/erm.2026.10073","DOIUrl":"https://doi.org/10.1017/erm.2026.10073","url":null,"abstract":"<p><strong>Background: </strong>Autoimmune diseases (AIDs) refer to a pathological condition in which the body's immune tolerance mechanism is disrupted or fails to function properly, leading to damage or dysfunction of its own tissues. Most AIDs are chronic and recurrent, severely affecting patients' quality of life and ability to work. Currently, there is no curative treatment for AIDs; conventional strategies mainly focus on suppressing immune responses and managing symptoms. However, long-term use of these approaches often leads to significant adverse reactions, and some patients respond poorly to therapy. Therefore, new, safe, and effective treatments are urgently needed. Cell therapy, which involves collecting cells via conventional or bioengineering methods, expanding or modifying them in vitro under specific conditions, and then reinfusing them into the body, has emerged as a promising treatment approach.</p><p><strong>Methods: </strong>This review summarizes current cell-based therapeutic strategies, their clinical applications, and the key challenges in the context of AIDs.</p><p><strong>Results: </strong>Recent studies have reported remarkable outcomes with cell therapy across various diseases, making it a major research focus. Notably, cell therapy offers distinct advantages for treating AIDs, including targeted immune regulation and the potential for durable remission. Nevertheless, it also faces challenges such as complex manufacturing processes, high costs, safety concerns, and variable efficacy.</p><p><strong>Conclusion: </strong>Cell therapy is a promising treatment approach for AIDs. However, further research is needed to overcome its current limitations and establish its safety and efficacy in clinical practice.</p>","PeriodicalId":50462,"journal":{"name":"Expert Reviews in Molecular Medicine","volume":"28 ","pages":"e54"},"PeriodicalIF":6.5,"publicationDate":"2026-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149327130","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Post-ARDS Pulmonary Fibrosis: From Molecular Mechanisms to Clinical Management.","authors":"Guiyang Jia, Zhikun Gan, Enhua Feng, Zhiyou Zheng, Erqin Song, Mingjiang Qian, Miao Chen, Guoyue Liu","doi":"10.1017/erm.2026.10077","DOIUrl":"https://doi.org/10.1017/erm.2026.10077","url":null,"abstract":"<p><strong>Background: </strong>Acute respiratory distress syndrome (ARDS) is a severe inflammatory lung injury caused by various factors, including infection, trauma and shock. The predominant pathological alteration in its late stages is pulmonary fibrosis (PF), characterised by progressive extracellular matrix deposition, architectural destruction and functional impairment. Particularly in COVID-19-induced ARDS, PF incidence has markedly increased, making it a major sequela impacting patients' long-term quality of life.</p><p><strong>Methods: </strong>This review systematically examines post-acute lung injury (ALI)/ARDS PF through three perspectives: pathogenesis, prediction and treatment.</p><p><strong>Results: </strong>Mechanistically, the fibrotic process involves epithelial injury and mesenchymal transition, fibroblast activation with aberrant extracellular matrix remodelling, dysregulated inflammation with immune cell involvement and clinical management-related factors. For prediction, various biomarkers (including growth factors, matrix proteins and inflammatory markers) and imaging features (particularly high-resolution computed tomography (HRCT) and magnetic resonance imaging (MRI) characteristics) demonstrate clinical value. Integrated multiparameter models incorporating artificial intelligence show promise for early identification and risk stratification of high-risk patients. Therapeutic strategies encompass anti-inflammatory approaches (e.g., corticosteroids and antioxidants), antifibrotic agents (pirfenidone and nintedanib), cell-based therapies (mesenchymal stem cells and exosomes) and emerging genetic/RNA interventions. However, specific preventive and therapeutic approaches tailored to the post-ALI/ARDS PF population remain inadequate in clinical practice.</p><p><strong>Conclusions: </strong>Future research should focus on elucidating underlying mechanisms, refining early-warning systems, exploring personalised treatments and facilitating clinical translation. The ultimate goal is to transition from traditional supportive care to proactive prevention through enhanced clinical surveillance, early prediction and rational intervention, thereby preventing fibrosis progression and improving outcomes in ARDS survivors.</p>","PeriodicalId":50462,"journal":{"name":"Expert Reviews in Molecular Medicine","volume":"28 ","pages":"e53"},"PeriodicalIF":6.5,"publicationDate":"2026-11-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149327131","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluating Alignment of Prader-Willi Syndrome with the Australian Newborn Bloodspot Screening Criteria.","authors":"Mohammed A Alshawsh, Oshadi Tennakoon, Sheena Arora, Ronda F Greaves, Jeanie Cheong, Katrina Williams, David E Godler","doi":"10.1017/erm.2026.10080","DOIUrl":"https://doi.org/10.1017/erm.2026.10080","url":null,"abstract":"<p><strong>Background: </strong>Prader-Willi syndrome (PWS) is a leading genetic cause of life-threatening obesity, presenting with hypotonia and feeding difficulties in infancy, progressing to hyperphagia, cognitive impairment and behavioural challenges from childhood. It is a rare imprinting disorder caused by gene silencing at the 15q11-q13 locus. Early detection through newborn bloodspot screening (NBS) could enable timely interventions such as growth hormone therapy, dietary management and behavioural support to improve health and developmental outcomes for the affected infants.</p><p><strong>Methods: </strong>This review applies the Australian Newborn Bloodspot Screening National Policy Framework as a reference to evaluate the suitability of incorporating PWS into NBS programs. The review outlines benefits and challenges in relation to key criteria used by this Framework, including condition severity, early detection benefits and screening test reliability.</p><p><strong>Results: </strong>Emerging screening technologies are recognised for their high sensitivity and specificity and could be integrated into existing NBS workflows using dried blood spots. However, several challenges outlined in this review need to be addressed, including the lack of cost-effectiveness data for population-wide PWS screening programs and the absence of a curative treatment for PWS.</p><p><strong>Conclusion: </strong>Expanding NBS to include PWS has the potential to improve health and developmental outcomes through earlier diagnosis and timely interventions. Successful implementation will depend on addressing the remaining challenges.</p>","PeriodicalId":50462,"journal":{"name":"Expert Reviews in Molecular Medicine","volume":"28 ","pages":"e52"},"PeriodicalIF":6.5,"publicationDate":"2026-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149289731","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"BMP4 as a Potential Molecular Link between Obesity and Female Fertility.","authors":"Yuwei Zhang, Jie Zhang, Gantao Chen, Fangfang Dai","doi":"10.1017/erm.2026.10068","DOIUrl":"https://doi.org/10.1017/erm.2026.10068","url":null,"abstract":"<p><p>Bone morphogenetic protein 4 (BMP4) is a member of the transforming growth factor β (TGF-β) superfamily. Accumulating evidence indicates that BMP4 is widely expressed in female reproductive tissues, including the ovary and endometrium. Therefore, this review summarizes the roles of BMP4 in regulating the physiological functions of the ovary and endometrium, including folliculogenesis, ovarian steroidogenesis, endometrial decidualization and regulation of maternal-foetal immunity. Notably, increasing studies revealed that BMP4 is also critically involved in obesity. Given the continuously rising prevalence of obesity worldwide, this review further summarizes emerging evidence on the role of BMP4 in adipogenesis, the shift between white adipose tissue (WAT) and brown adipose tissue (BAT) and metabolic dysregulation. To further elucidate the role of BMP4, we also summarize the expression patterns and potential mechanisms in reproductive disorders and obesity. We found that dysregulated BMP4 expression has been reported in polycystic ovary syndrome (PCOS), recurrent spontaneous abortion (RSA) and obesity, suggesting a potential molecular link between obesity and female fertility. Hence, the potential role of BMP4 between obesity and women's fertility is further discussed and may provide direction for further reproductive-related research. This hypothesis may guide further studies to explore the underlying mechanisms and their translational implications.</p>","PeriodicalId":50462,"journal":{"name":"Expert Reviews in Molecular Medicine","volume":"28 ","pages":"e49"},"PeriodicalIF":6.5,"publicationDate":"2026-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149289739","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Role of Intestinal Stem Cells in the Pathogenesis and Repair of Necrotizing Enterocolitis.","authors":"Fan Zhang, Xiaohua Zhang, Zhanhu Zhang","doi":"10.1017/erm.2026.10084","DOIUrl":"https://doi.org/10.1017/erm.2026.10084","url":null,"abstract":"<p><strong>Background: </strong>Necrotizing enterocolitis (NEC) is a leading gastrointestinal emergency in preterm infants, characterized by severe epithelial injury, dysregulated inflammation, and defective mucosal repair. Intestinal stem cells (ISCs) at the crypt base are essential for epithelial renewal and barrier restoration after injury.</p><p><strong>Methods: </strong>We summarized recent advances in the molecular and cellular mechanisms governing ISC injury and regeneration in NEC, emphasizing canonical repair pathways, niche-derived regulatory circuits, and emerging ISC-targeted therapies.</p><p><strong>Results: </strong>NEC disrupts ISC survival, proliferation, and differentiation through aberrant innate immune activation, microbial dysbiosis, and impairment of Wnt, Notch, and epidermal growth factor (EGF) signalling. ISC function is also shaped by interactions with Paneth cells, mesenchymal niche components, immune cells, and microbial metabolites. Disruption of this niche compromises crypt regeneration, perpetuates barrier dysfunction, and amplifies intestinal inflammation. Restoring ISC activity and niche integrity has therefore prompted interest in growth factor supplementation, microbiota modulation, immune-based interventions, and stem cell-oriented approaches.</p><p><strong>Conclusions: </strong>ISC-centred regenerative failure provides an integrated framework linking epithelial biology, immunology, and microbiome research in NEC. Restoring ISC function and niche integrity represents a promising therapeutic direction with experimental and translational relevance.</p>","PeriodicalId":50462,"journal":{"name":"Expert Reviews in Molecular Medicine","volume":"28 ","pages":"e51"},"PeriodicalIF":6.5,"publicationDate":"2026-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149289745","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adiponectin Therapeutics for Fibrotic Diseases: Current Knowledge and Future Perspectives.","authors":"Divya Shah, Jialing Tang, Eddie Tam, Henry H Hsu, Gary Sweeney","doi":"10.1017/erm.2026.10086","DOIUrl":"https://doi.org/10.1017/erm.2026.10086","url":null,"abstract":"<p><strong>Background: </strong>Fibrosis is characterised by the pathological accumulation of extracellular matrix components and represents both a driver and consequence of chronic tissue injury across multiple organs and is a major contributor to global morbidity and mortality. Despite this substantial clinical burden, approved antifibrotic therapies remain limited. Adiponectin, an adipokine secreted predominantly by adipocytes, is an endogenous antifibrotic mediator with potential therapeutic applications.</p><p><strong>Methods: </strong>This review examines the pathobiology of fibrosis, the molecular mechanisms underlying adiponectin-mediated antifibrotic effects, and current advances in adiponectin-based therapeutic development. Evidence from preclinical studies across multiple organ system is discussed, with a focus on adiponectin signaling pathways and receptor agonists.</p><p><strong>Results: </strong>Adiponectin actsprimarily through its cognate receptors AdipoR1 and AdipoR2, adiponectin suppresses key profibrotic signalling cascades, including TGF-β/Smad, NF-κB, Wnt/β-catenin, Notch and platelet-derived growth factor pathways, while simultaneously promoting anti-inflammatory, antioxidant and cytoprotective responses. Preclinical studies demonstrate that adiponectin deficiency exacerbates fibrotic remodelling in the liver, lungs, heart, kidneys, skeletal muscle and skin, whereas genetic or pharmacological augmentation of adiponectin signalling attenuates fibrosis progression. Two adiponectin receptor agonists, the small molecule AdipoRON and the peptide mimetic ALY688, have shown promising antifibrotic efficacy across multiple disease models.</p><p><strong>Conclusions: </strong>Adiponectin signaling represents a promising therapeutic strategy for the prevention and treatment across multiple organs. Further investigation is needed to address current challenges and establish the safety, efficacy, and translational potential of adiponectin-based therapies in clinical settings.</p>","PeriodicalId":50462,"journal":{"name":"Expert Reviews in Molecular Medicine","volume":"28 ","pages":"e50"},"PeriodicalIF":6.5,"publicationDate":"2026-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149276524","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Diagnostic Significance of microRNA-146 in Asthma: A Systematic Review.","authors":"Zahra Shojaei, Behzad Khansarinejad, Amir Almasi-Hashiani, Mahdieh Mondanizadeh, Javad Nazari","doi":"10.1017/erm.2026.10089","DOIUrl":"https://doi.org/10.1017/erm.2026.10089","url":null,"abstract":"","PeriodicalId":50462,"journal":{"name":"Expert Reviews in Molecular Medicine","volume":" ","pages":"1-27"},"PeriodicalIF":6.5,"publicationDate":"2026-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149276533","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"RNA-Binding Proteins Regulate CD8⁺ T Cell Biology: Mechanisms and Therapeutic Opportunities.","authors":"Ashwin B R Kumar","doi":"10.1017/erm.2026.10075","DOIUrl":"https://doi.org/10.1017/erm.2026.10075","url":null,"abstract":"<p><strong>Background: </strong>Immune-related diseases contribute substantially to global morbidity and mortality. CD8⁺ T cells play a critical role in adaptive immunity by eliminating virus-infected and malignant cells. The biology of CD8⁺ T cells is governed by tightly regulated gene expression programs, and disruption of these programs can lead to diverse immunopathologies and malignancies. This article evaluates our current understanding of post-transcriptional and translational regulation of gene expression in CD8⁺ T cells by RNA-binding proteins.</p><p><strong>Methods: </strong>Relevant and up-to-date literature was critically analysed to gain mechanistic insights into the roles of RNA-binding proteins in CD8⁺ T cell biology and to identify key knowledge gaps and future directions, with particular emphasis on therapeutic implications.</p><p><strong>Results: </strong>RNA-binding proteins regulate diverse aspects of CD8⁺ T cell biology, including differentiation, cytokine production, stress adaptation and proliferation. Recent studies further reveal how dysregulated expression and function of RNA-binding proteins contribute to hyperinflammation and haematological malignancies.</p><p><strong>Conclusions: </strong>RNA-binding proteins are emerging as key post-transcriptional and translational checkpoint regulators of CD8⁺ T cell biology and represent promising therapeutic targets for immune-related diseases; however, substantial mechanistic and disease-specific studies, as well as rigorous translational validation, are required before RNA-binding protein-targeted therapeutic strategies can be safely and effectively advanced into clinical practice.</p>","PeriodicalId":50462,"journal":{"name":"Expert Reviews in Molecular Medicine","volume":"28 ","pages":"e45"},"PeriodicalIF":6.5,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149197664","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Spinal muscular atrophy as a blueprint for precision therapy in neuromuscular disease.","authors":"Busra Cetin, Melike Aliciaslan, Ezgi Erbasan, Busra Cesur Ergun, Salih Sanlioglu","doi":"10.1017/erm.2026.10072","DOIUrl":"https://doi.org/10.1017/erm.2026.10072","url":null,"abstract":"<p><strong>Background: </strong>Spinal Muscular Atrophy (SMA) is caused by a deficiency of the survival motor neuron (SMN) protein due to loss of <i>SMN1</i> and inefficient compensation by <i>SMN2.</i> This genetic architecture has driven the development of precision therapeutics. Over the past decade, SMA management has progressed from supportive care to RNA-based splicing modulation and gene replacement therapy.</p><p><strong>Methods: </strong>This review analyzes the evolution of three therapeutic waves in SMA management: antisense oligonucleotides (nusinersen) for <i>SMN2</i> splicing correction; systemic small-molecule splicing modifiers (risdiplam) addressing SMA as a multisystem disorder; and gene replacement therapy (onasemnogene abeparvovec), including recent intrathecal formulations expanding patient eligibility.</p><p><strong>Results: </strong>Precision therapies have profoundly altered the disease trajectory, especially when administered presymptomatically. Nusinersen sustains splicing correction, risdiplam restores SMN across central and peripheral tissues, and gene replacement offers durable, one-time genetic correction. However, critical challenges persist regarding therapeutic durability, optimal treatment sequencing, systemic involvement, and long-term safety.</p><p><strong>Conclusions: </strong>The evolution of SMA therapies has transformed neurogenetics. Clinical benchmarks have successfully shifted from reactive, symptomatic management to proactive, molecularly targeted precision medicine.</p>","PeriodicalId":50462,"journal":{"name":"Expert Reviews in Molecular Medicine","volume":"28 ","pages":"e42"},"PeriodicalIF":6.5,"publicationDate":"2026-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149026607","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multi-Omics in Kidney Disease Research: Technological Advances, Biomarker Discovery and Clinical Translation.","authors":"Xuemei Gong, Xiaoxi Zeng, Ping Fu","doi":"10.1017/erm.2026.10067","DOIUrl":"10.1017/erm.2026.10067","url":null,"abstract":"<p><strong>Background: </strong>Multi-omics research is reshaping precision nephrology by linking genetic susceptibility, epigenetic regulation, transcriptional state, protein abundance, metabolic activity and spatial tissue context within integrated analytical frameworks.</p><p><strong>Methods: </strong>We critically reviewed recent technological advances and applications of multi-omics in kidney diseases, with particular attention to biomarker discovery and clinical translation, and discussed the challenges and future directions of multi-omics integration and its application in precision medicine.</p><p><strong>Results: </strong>Emerging approaches, including single-cell and spatial omics, artificial intelligence-assisted analytics and integrative computational frameworks, have substantially improved the resolution of renal pathophysiology and molecular heterogeneity. These advancements not only reveal novel molecular pathways underlying chronic kidney disease, diabetic kidney disease, acute kidney injury and immune-mediated nephropathies, but also significantly facilitate biomarker discovery and therapeutic target prioritization. However, major challenges remain, including limited cohort diversity, high analytical complexity, lack of standardized pipelines, insufficient external validation and barriers to clinical implementation, such as cost, scalability and regulatory integration.</p><p><strong>Conclusions: </strong>Collectively, multi-omics strategies are reshaping kidney disease research and hold considerable promise for advancing precision diagnosis, risk stratification and personalized therapeutics. Future efforts should prioritize harmonized multicentre datasets, longitudinal validation, explainable artificial intelligence models and clinically actionable frameworks to bridge the gap between omics discovery and routine nephrology practice.</p>","PeriodicalId":50462,"journal":{"name":"Expert Reviews in Molecular Medicine","volume":" ","pages":"e43"},"PeriodicalIF":6.5,"publicationDate":"2026-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148708383","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}