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Cystic fibrosis at a glance: from disease mechanism to therapy. 囊性纤维化概览：从发病机制到治疗。
IF 12.8 1区 医学
Trends in molecular medicine Pub Date : 2025-04-01 Epub Date: 2025-03-21 DOI: 10.1016/j.molmed.2025.02.001
Kasper Gryspeert, Laudonia L Dipalo, Ana L Da Silva Cunha, Mattijs Bulcaen, Marjolein M Ensinck, Marianne S Carlon
{"title":"Cystic fibrosis at a glance: from disease mechanism to therapy.","authors":"Kasper Gryspeert, Laudonia L Dipalo, Ana L Da Silva Cunha, Mattijs Bulcaen, Marjolein M Ensinck, Marianne S Carlon","doi":"10.1016/j.molmed.2025.02.001","DOIUrl":"10.1016/j.molmed.2025.02.001","url":null,"abstract":"","PeriodicalId":23263,"journal":{"name":"Trends in molecular medicine","volume":" ","pages":"399-400"},"PeriodicalIF":12.8,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143693370","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Here's to 30 Years of Trends in Molecular Medicine! 为分子医学30年的发展趋势干杯！
IF 12.8 1区 医学
Trends in molecular medicine Pub Date : 2025-04-01 DOI: 10.1016/j.molmed.2024.12.011
Aliki Perdikari
{"title":"Here's to 30 Years of Trends in Molecular Medicine!","authors":"Aliki Perdikari","doi":"10.1016/j.molmed.2024.12.011","DOIUrl":"https://doi.org/10.1016/j.molmed.2024.12.011","url":null,"abstract":"","PeriodicalId":23263,"journal":{"name":"Trends in molecular medicine","volume":"31 4","pages":"297"},"PeriodicalIF":12.8,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019133","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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 cytokines for tumor-targeting and selective T cell activation. 用于肿瘤靶向和选择性T细胞活化的工程细胞因子。
IF 12.8 1区 医学
Trends in molecular medicine Pub Date : 2025-04-01 Epub Date: 2025-02-14 DOI: 10.1016/j.molmed.2025.01.010
Kun Peng, Yang-Xin Fu, Yong Liang
{"title":"Engineering cytokines for tumor-targeting and selective T cell activation.","authors":"Kun Peng, Yang-Xin Fu, Yong Liang","doi":"10.1016/j.molmed.2025.01.010","DOIUrl":"10.1016/j.molmed.2025.01.010","url":null,"abstract":"<p><p>Cytokines are promising therapeutic agents for cancer due to their immune-stimulating properties. However, their clinical application is limited by a narrow therapeutic window and dose-limiting on-target, off-tumor toxicity. Advances in protein engineering enable the selective delivery of cytokines to the tumor microenvironment (TME) and antigen-specific T cells, enhancing antitumor efficacy while reducing systemic side effects. This review focuses on selected cytokines and outlines their developmental trajectory for treating solid tumors. We highlight strategies for constructing cis-signaling immunocytokines and procytokines for precise delivery to tumor sites and discuss the biological mechanisms through which these cytokines reactivate antitumor immunity. We also discuss the challenges and future directions for creating more effective cytokine-based therapeutics.</p>","PeriodicalId":23263,"journal":{"name":"Trends in molecular medicine","volume":" ","pages":"373-387"},"PeriodicalIF":12.8,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426266","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
The bleeding edge: broadening horizons for hematopoietic stem cell therapies. 前沿：拓宽造血干细胞治疗的视野。
IF 12.8 1区 医学
Trends in molecular medicine Pub Date : 2025-04-01 Epub Date: 2025-03-21 DOI: 10.1016/j.molmed.2025.03.003
Spencer D Shelton, Vijay G Sankaran
{"title":"The bleeding edge: broadening horizons for hematopoietic stem cell therapies.","authors":"Spencer D Shelton, Vijay G Sankaran","doi":"10.1016/j.molmed.2025.03.003","DOIUrl":"10.1016/j.molmed.2025.03.003","url":null,"abstract":"<p><p>Recent reports of gene therapy using autologous hematopoietic stem cell transplantation (HSCT) have addressed protein deficiencies of extra-hematopoietic origin. In a recent study, Srivastava et al. report that patients with hemophilia A receiving F8 lentiviral HSCT gene therapy achieved lasting factor VIII restoration and clinical improvement, marking an advance that could enable broader applications of HSCT.</p>","PeriodicalId":23263,"journal":{"name":"Trends in molecular medicine","volume":" ","pages":"301-303"},"PeriodicalIF":12.8,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11985263/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143693371","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Adeno-associated viral vector targeted evolution for neurofibromatosis gene delivery. 腺相关病毒载体靶向进化用于神经纤维瘤病基因传递。
IF 12.8 1区 医学
Trends in molecular medicine Pub Date : 2025-04-01 Epub Date: 2025-01-30 DOI: 10.1016/j.molmed.2025.01.004
Brett Henshey, Ana Carneiro, Kecheng Lei, David Schaffer, Nicholas M Boulis
{"title":"Adeno-associated viral vector targeted evolution for neurofibromatosis gene delivery.","authors":"Brett Henshey, Ana Carneiro, Kecheng Lei, David Schaffer, Nicholas M Boulis","doi":"10.1016/j.molmed.2025.01.004","DOIUrl":"10.1016/j.molmed.2025.01.004","url":null,"abstract":"<p><p>Neurofibromatosis type 1 (NF1) is an inherited genetic disease resulting from pathogenic mutations in NF1 that drive tumor formation along peripheral nerves, leading to many functional consequences. Tumor removal or treatment often results in regrowth and/or nerve damage. Addressing NF1 pathogenic variations at the cellular level through gene therapy holds great potential for long-term treatment of patients with NF1. Adeno-associated viruses (AAVs) are broadly used gene delivery vehicles for gene therapies because of their low pathogenicity, ability to transduce nondividing cells, and potential for long-term gene expression. This article explores the landscape of AAV-mediated gene delivery strategies for NF1, discusses the challenges of efficient delivery to relevant cell types, and highlights the progress in vector design strategies.</p>","PeriodicalId":23263,"journal":{"name":"Trends in molecular medicine","volume":" ","pages":"388-398"},"PeriodicalIF":12.8,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11985305/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143075425","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
A sex-biased target against demyelination in Alzheimer's disease. 针对阿尔茨海默病脱髓鞘的性别偏见靶点。
IF 12.8 1区 医学
Trends in molecular medicine Pub Date : 2025-04-01 Epub Date: 2025-03-24 DOI: 10.1016/j.molmed.2025.03.004
Kelsey R Babcock, Doudou Yu, Ashley E Webb
{"title":"A sex-biased target against demyelination in Alzheimer's disease.","authors":"Kelsey R Babcock, Doudou Yu, Ashley E Webb","doi":"10.1016/j.molmed.2025.03.004","DOIUrl":"10.1016/j.molmed.2025.03.004","url":null,"abstract":"<p><p>Aging and Alzheimer's disease (AD) exhibit sex differences in several biological processes, including demyelination. In a recent study, Lopez-Lee et al. uncover the contributions of sex chromosomes and gonadal hormones to sex differences in demyelination and identify Toll-like receptor 7 (TLR7) as a potential target to ameliorate tauopathy-induced demyelination in men.</p>","PeriodicalId":23263,"journal":{"name":"Trends in molecular medicine","volume":" ","pages":"304-306"},"PeriodicalIF":12.8,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11985262/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143711381","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Advances in Hodgkin lymphoma research. 霍奇金淋巴瘤研究进展。
IF 12.8 1区 医学
Trends in molecular medicine Pub Date : 2025-04-01 Epub Date: 2024-10-22 DOI: 10.1016/j.molmed.2024.10.004
Ralf Küppers
{"title":"Advances in Hodgkin lymphoma research.","authors":"Ralf Küppers","doi":"10.1016/j.molmed.2024.10.004","DOIUrl":"10.1016/j.molmed.2024.10.004","url":null,"abstract":"<p><p>Hodgkin lymphoma (HL) has been and still is the most enigmatic lymphoid malignancy in humans. Since the first molecular analysis of isolated Hodgkin and Reed-Sternberg (HRS) tumor cells of classic HL 30 years ago, substantial advances in our understanding of HL have been made. This review describes the cellular origin of HL, summarizes the current knowledge about the genetic lesions in HRS cells, and highlights the role of Epstein-Barr virus (EBV) in HL pathogenesis. Moreover, the pathobiological roles of altered gene expression and deregulated signaling pathways are discussed and key aspects of the HL microenvironment are presented.</p>","PeriodicalId":23263,"journal":{"name":"Trends in molecular medicine","volume":" ","pages":"326-343"},"PeriodicalIF":12.8,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142508721","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Gene therapy for β-thalassemia: current and future options. β-地中海贫血的基因治疗：当前和未来的选择。
IF 12.8 1区 医学
Trends in molecular medicine Pub Date : 2025-04-01 Epub Date: 2025-01-10 DOI: 10.1016/j.molmed.2024.12.001
Giulia Hardouin, Annarita Miccio, Megane Brusson
{"title":"Gene therapy for β-thalassemia: current and future options.","authors":"Giulia Hardouin, Annarita Miccio, Megane Brusson","doi":"10.1016/j.molmed.2024.12.001","DOIUrl":"10.1016/j.molmed.2024.12.001","url":null,"abstract":"<p><p>Beta-thalassemia is a severe, hereditary blood disorder characterized by anemia, transfusion dependence, reduced life expectancy, and poor quality of life. Allogeneic transplantation of hematopoietic stem cells (HSCs) is the only curative treatment for transfusion-dependent β-thalassemia, but a lack of compatible donors prevents the use of this approach for most patients. Over the past 20 years, the rise of gene therapy and the development of lentiviral vectors and genome-editing tools has extended curative options to a broader range of patients. Here, we review breakthroughs in gene addition- and genome-editing-based therapies for β-thalassemia, the clinical outcomes enabling approval by regulatory agencies, and perspectives for further development.</p>","PeriodicalId":23263,"journal":{"name":"Trends in molecular medicine","volume":" ","pages":"344-358"},"PeriodicalIF":12.8,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142967054","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Thirty years of molecular medicine: reflections from an academic physician-scientist, 1995-2025. 分子医学三十年：一位学术医师兼科学家的反思，1995-2025。
IF 12.8 1区 医学
Trends in molecular medicine Pub Date : 2025-04-01 Epub Date: 2025-02-05 DOI: 10.1016/j.molmed.2025.01.012
William A Petri
{"title":"Thirty years of molecular medicine: reflections from an academic physician-scientist, 1995-2025.","authors":"William A Petri","doi":"10.1016/j.molmed.2025.01.012","DOIUrl":"10.1016/j.molmed.2025.01.012","url":null,"abstract":"","PeriodicalId":23263,"journal":{"name":"Trends in molecular medicine","volume":" ","pages":"298-300"},"PeriodicalIF":12.8,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143366017","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Pulmonary arterial hypertension and cancer: exploring their resemblance as channelopathies. 肺动脉高压与癌症：探讨其作为通道病变的相似性。
IF 12.8 1区 医学
Trends in molecular medicine Pub Date : 2025-04-01 DOI: 10.1016/j.molmed.2025.03.002
Fabrice Antigny, David Crottès, Christophe Vandier, Véronique Capuano, Maxime Guéguinou
{"title":"Pulmonary arterial hypertension and cancer: exploring their resemblance as channelopathies.","authors":"Fabrice Antigny, David Crottès, Christophe Vandier, Véronique Capuano, Maxime Guéguinou","doi":"10.1016/j.molmed.2025.03.002","DOIUrl":"https://doi.org/10.1016/j.molmed.2025.03.002","url":null,"abstract":"<p><p>Pulmonary arterial hypertension (PAH) and cancer may appear to be unrelated at first, but there is increasing evidence that they share many characteristics and complexities. Pulmonary vascular cells in PAH resemble cancer cells in that they display abnormal growth patterns, resistance to cell death, metabolic changes, and channelopathies. These similarities open new possibilities for researchers and clinicians to apply cancer treatment strategies to PAH and possibly reverse the condition. This review explores the complex parallels between PAH and cancer, and emphasizes their similar channelopathy-like features at the molecular, cellular, and clinical levels. We also discuss the potential implications of these similarities for developing new treatments.</p>","PeriodicalId":23263,"journal":{"name":"Trends in molecular medicine","volume":" ","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143773458","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
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