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Expert Opinion on Therapeutic Targets最新文献

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Therapeutic targeting of the polyglutamine androgen receptor in Spinal and Bulbar Muscular Atrophy. 多谷氨酰胺雄激素受体治疗脊髓和球性肌萎缩的靶向性研究。
IF 4.6 2区 医学
Expert Opinion on Therapeutic Targets Pub Date : 2025-01-01 Epub Date: 2025-02-10 DOI: 10.1080/14728222.2025.2464173
Agamjot Sangotra, Andrew P Lieberman
{"title":"Therapeutic targeting of the polyglutamine androgen receptor in Spinal and Bulbar Muscular Atrophy.","authors":"Agamjot Sangotra, Andrew P Lieberman","doi":"10.1080/14728222.2025.2464173","DOIUrl":"10.1080/14728222.2025.2464173","url":null,"abstract":"<p><strong>Introduction: </strong>Spinal and Bulbar Muscular Atrophy (SBMA) is a slowly progressive, X-linked, and sex-limited degenerative disorder affecting lower motor neurons and skeletal muscle which lacks disease-modifying therapies. This disease is caused by a CAG/polyglutamine (polyQ) tract expansion in the androgen receptor (AR) gene, and its pathogenesis is driven by toxic gain-of-function mechanisms. Affected men develop proximal limb and bulbar muscle weakness along with signs of partial androgen insensitivity.</p><p><strong>Areas covered: </strong>Toxicity of the polyQ AR is mediated by protein misfolding and nuclear translocation that follow ligand binding, resulting in the disruption of downstream homeostatic mechanisms. This review highlights what is known about disease pathogenesis and how this has been leveraged to test potential therapeutic approaches. The focus is on strategies that alleviate polyQ AR toxicity in SBMA, including those that alter AR function, diminish the expression of the encoding gene, or promote clearance of the misfolded, mutant protein.</p><p><strong>Expert opinion: </strong>We discuss emerging strategies to mitigate polyQ AR toxicity, including gene editing, RNA targeted therapies, and efforts to harness proteostatic mechanisms. These promising approaches are discussed in the context of challenges for drug discovery efforts that are faced when attempting to treat a rare and slowly progressive neurodegenerative disorder.</p>","PeriodicalId":12185,"journal":{"name":"Expert Opinion on Therapeutic Targets","volume":" ","pages":"29-41"},"PeriodicalIF":4.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11888889/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143364174","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Targeting acid-sensing ion channels in glioblastoma: is there any therapeutic potential? 以胶质母细胞瘤中的酸感应离子通道为靶点：是否有治疗潜力？
IF 4.6 2区 医学
Expert Opinion on Therapeutic Targets Pub Date : 2025-01-01 Epub Date: 2025-02-06 DOI: 10.1080/14728222.2025.2463357
Andrea Menegon
{"title":"Targeting acid-sensing ion channels in glioblastoma: is there any therapeutic potential?","authors":"Andrea Menegon","doi":"10.1080/14728222.2025.2463357","DOIUrl":"10.1080/14728222.2025.2463357","url":null,"abstract":"","PeriodicalId":12185,"journal":{"name":"Expert Opinion on Therapeutic Targets","volume":" ","pages":"5-8"},"PeriodicalIF":4.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143188754","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}
引用次数: 0
Assessing platelet-derived extracellular vesicles for potential as therapeutic targets in cardiovascular diseases. 评估血小板来源的细胞外囊泡作为心血管疾病治疗靶点的潜力。
IF 4.6 2区 医学
Expert Opinion on Therapeutic Targets Pub Date : 2025-01-01 Epub Date: 2025-01-18 DOI: 10.1080/14728222.2025.2454617
Xin Xin, Rory R Koenen
{"title":"Assessing platelet-derived extracellular vesicles for potential as therapeutic targets in cardiovascular diseases.","authors":"Xin Xin, Rory R Koenen","doi":"10.1080/14728222.2025.2454617","DOIUrl":"10.1080/14728222.2025.2454617","url":null,"abstract":"<p><strong>Introduction: </strong>Cardiovascular disease (CVD) is the leading cause of death worldwide. Platelet-derived extracellular vesicles (PEV) have attracted extensive attention in cardiovascular disease research in recent years because their cargo is involved in a variety of pathophysiological processes, such as thrombosis, immune response, promotion or inhibition of inflammatory response, promotion of angiogenesis as well as cell proliferation and migration.</p><p><strong>Areas covered: </strong>This review explores the role of PEV in various cardiovascular diseases (such as atherosclerosis, myocardial infarction, ischemia-reperfusion injury, and heart failure), with relation to its molecular cargo (nucleic acids, bioactive lipids, proteins) and aims to provide new insights in the pathophysiologic role of PEV, and methods for preventing and treating cardiovascular diseases based on PEV.</p><p><strong>Expert opinion: </strong>Studies have shown that the cargo of PEV may be dysregulated during cardiovascular disease and delivery to tissues can result in detrimental pathophysiologic effects. Counteracting this process might have the potential to inhibit inflammation, promote angiogenesis, and inhibit cardiomyocyte death. In addition, PEV have potential as biocompatible and autologous drug carriers. Therefore, better research on the mechanisms how PEV act during cardiovascular disease and could be implemented as a therapeutic will provide new perspectives for the treatment of cardiovascular disease.</p>","PeriodicalId":12185,"journal":{"name":"Expert Opinion on Therapeutic Targets","volume":" ","pages":"17-28"},"PeriodicalIF":4.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143002694","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}
引用次数: 0
Targeting glypican 3 by immunotoxins: the promise of immunotherapy in hepatocellular carcinoma. 免疫毒素靶向glypican 3的研究肝细胞癌免疫治疗的前景。
IF 4.6 2区 医学
Expert Opinion on Therapeutic Targets Pub Date : 2025-01-01 Epub Date: 2025-02-26 DOI: 10.1080/14728222.2025.2471581
Elham Rismani, Nikoo Hossein-Khannazer, Moustapha Hassan, Elahe Shams, Mustapha Najimi, Massoud Vosough
{"title":"Targeting glypican 3 by immunotoxins: the promise of immunotherapy in hepatocellular carcinoma.","authors":"Elham Rismani, Nikoo Hossein-Khannazer, Moustapha Hassan, Elahe Shams, Mustapha Najimi, Massoud Vosough","doi":"10.1080/14728222.2025.2471581","DOIUrl":"10.1080/14728222.2025.2471581","url":null,"abstract":"<p><strong>Introduction: </strong>Tumor cell's resistance, high recurrence rate, and low overall survival rate have made hepatocellular carcinoma (HCC) a major health concern. The combination of advanced targeted therapies such as immunotherapy, with conventional treatments has gained traction for application on HCC. Immunotoxins (ITs) represent a category of biomolecules that combine the targeted affinity of antibodies with the cytotoxic properties of toxins.</p><p><strong>Areas covered: </strong>This study highlights Glypican3 (GPC3) as a potential candidate for targeted therapeutic interventions using ITs. It presents a comprehensive overview of the advantages and challenges associated with these modalities, and their promising outcomes in HCC treatment. A systematic literature review was conducted using PubMed, Web of Science and Scopus from 2015 to 2024.</p><p><strong>Expert opinion: </strong>Despite potential applicability, many concerns should be addressed before the employment of GPC3-based ITs. These include improving efficient penetration of ITs into the solid tumors, considering neutralizing antibodies against the drugs, and enhancing serum half-life of ITs. Furthermore, the ITs potential in eliminating cancer stem cells (CSCs) and residual tumor cells is discussed. The ability to target CSCs can significantly reduce the likelihood of recurrence and improve overall survival rate. This could make ITs a pivotal component in the future of HCC treatment.</p>","PeriodicalId":12185,"journal":{"name":"Expert Opinion on Therapeutic Targets","volume":" ","pages":"59-73"},"PeriodicalIF":4.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476478","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}
引用次数: 0
NF-κB-inducing kinase (NIK): an emerging therapeutic target in human disease. nf - kb诱导激酶（NIK）：人类疾病新出现的治疗靶点。
IF 4.6 2区 医学
Expert Opinion on Therapeutic Targets Pub Date : 2025-01-01 Epub Date: 2025-02-09 DOI: 10.1080/14728222.2025.2464175
Cassandra S Poole, Irving Coy Allen
{"title":"NF-κB-inducing kinase (NIK): an emerging therapeutic target in human disease.","authors":"Cassandra S Poole, Irving Coy Allen","doi":"10.1080/14728222.2025.2464175","DOIUrl":"10.1080/14728222.2025.2464175","url":null,"abstract":"","PeriodicalId":12185,"journal":{"name":"Expert Opinion on Therapeutic Targets","volume":" ","pages":"13-16"},"PeriodicalIF":4.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143364171","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}
引用次数: 0
Mast cell chymase-1 and tryptases: therapeutic targets for COPD? 肥大细胞乳糜酶-1和胰蛋白酶：COPD的治疗靶点？
IF 4.6 2区 医学
Expert Opinion on Therapeutic Targets Pub Date : 2025-01-01 Epub Date: 2025-02-23 DOI: 10.1080/14728222.2025.2464176
Gang Liu, Angelica Tiotiu, Philip M Hansbro
{"title":"Mast cell chymase-1 and tryptases: therapeutic targets for COPD?","authors":"Gang Liu, Angelica Tiotiu, Philip M Hansbro","doi":"10.1080/14728222.2025.2464176","DOIUrl":"10.1080/14728222.2025.2464176","url":null,"abstract":"","PeriodicalId":12185,"journal":{"name":"Expert Opinion on Therapeutic Targets","volume":" ","pages":"9-12"},"PeriodicalIF":4.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143467481","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}
引用次数: 0
Pharmacological targeting of ECM homeostasis, fibroblast activation and invasion for the treatment of pulmonary fibrosis. ECM稳态、成纤维细胞激活和侵袭的药物靶向治疗肺纤维化。
IF 4.6 2区 医学
Expert Opinion on Therapeutic Targets Pub Date : 2025-01-01 Epub Date: 2025-02-27 DOI: 10.1080/14728222.2025.2471579
Ioannis Tomos, Paraskevi Kanellopoulou, Dimitris Nastos, Vassilis Aidinis
{"title":"Pharmacological targeting of ECM homeostasis, fibroblast activation and invasion for the treatment of pulmonary fibrosis.","authors":"Ioannis Tomos, Paraskevi Kanellopoulou, Dimitris Nastos, Vassilis Aidinis","doi":"10.1080/14728222.2025.2471579","DOIUrl":"10.1080/14728222.2025.2471579","url":null,"abstract":"<p><strong>Introduction: </strong>Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive interstitial lung disease with a dismal prognosis. While the standard-of-care (SOC) drugs approved for IPF represent a significant advancement in antifibrotic therapies, they primarily slow disease progression and have limited overall efficacy and many side effects. Consequently, IPF remains a condition with high unmet medical and pharmacological needs.</p><p><strong>Areas covered: </strong>A wide variety of molecules and mechanisms have been implicated in the pathogenesis of IPF, many of which have been targeted in clinical trials. In this review, we discuss the latest therapeutic targets that affect extracellular matrix (ECM) homeostasis and the activation of lung fibroblasts, with a specific focus on ECM invasion.</p><p><strong>Expert opinion: </strong>A promising new approach involves targeting ECM invasion by fibroblasts, a process that parallels cancer cell behavior. Several cancer drugs are now being tested in IPF for their ability to inhibit ECM invasion, offering significant potential for future treatments. The delivery of these therapies by inhalation is a promising development, as it may enhance local effectiveness and minimize systemic side effects, thereby improving patient safety and treatment efficacy.</p>","PeriodicalId":12185,"journal":{"name":"Expert Opinion on Therapeutic Targets","volume":" ","pages":"43-57"},"PeriodicalIF":4.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476476","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}
引用次数: 0
The potential of multimolecular G-quadruplex structures for targeted treatment of Amyotrophic Lateral Sclerosis. 多分子g -四重结构靶向治疗肌萎缩性侧索硬化症的潜力。
IF 4.6 2区 医学
Expert Opinion on Therapeutic Targets Pub Date : 2025-01-01 Epub Date: 2025-02-07 DOI: 10.1080/14728222.2025.2463361
Nereida Abad-Yang, Federica Raguseo, Lorenzo Di Michele, Rickie Patani, Marco Di Antonio
{"title":"The potential of multimolecular G-quadruplex structures for targeted treatment of Amyotrophic Lateral Sclerosis.","authors":"Nereida Abad-Yang, Federica Raguseo, Lorenzo Di Michele, Rickie Patani, Marco Di Antonio","doi":"10.1080/14728222.2025.2463361","DOIUrl":"10.1080/14728222.2025.2463361","url":null,"abstract":"","PeriodicalId":12185,"journal":{"name":"Expert Opinion on Therapeutic Targets","volume":" ","pages":"1-4"},"PeriodicalIF":4.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143188756","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}
引用次数: 0
Druggable genes for therapeutic targeting in PTH signaling for osteoporosis. 骨质疏松症PTH信号治疗靶点的药物基因。
IF 4.6 2区 医学
Expert Opinion on Therapeutic Targets Pub Date : 2024-12-20 DOI: 10.1080/14728222.2024.2443091
Chisato Sampei, Tadayoshi Hayata
{"title":"Druggable genes for therapeutic targeting in PTH signaling for osteoporosis.","authors":"Chisato Sampei, Tadayoshi Hayata","doi":"10.1080/14728222.2024.2443091","DOIUrl":"10.1080/14728222.2024.2443091","url":null,"abstract":"","PeriodicalId":12185,"journal":{"name":"Expert Opinion on Therapeutic Targets","volume":" ","pages":"1-3"},"PeriodicalIF":4.6,"publicationDate":"2024-12-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142824054","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}
引用次数: 0
Is Cav1.3 a feasible therapeutic target for a rare neurodevelopmental disorder? Cav1.3是一种罕见神经发育障碍的可行治疗靶点吗？
IF 4.6 2区 医学
Expert Opinion on Therapeutic Targets Pub Date : 2024-12-19 DOI: 10.1080/14728222.2024.2442428
Nadine J Ortner
{"title":"Is Cav1.3 a feasible therapeutic target for a rare neurodevelopmental disorder?","authors":"Nadine J Ortner","doi":"10.1080/14728222.2024.2442428","DOIUrl":"10.1080/14728222.2024.2442428","url":null,"abstract":"","PeriodicalId":12185,"journal":{"name":"Expert Opinion on Therapeutic Targets","volume":" ","pages":"1-5"},"PeriodicalIF":4.6,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142817651","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}
引用次数: 0
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