Cold Spring Harbor perspectives in medicine最新文献

Innate Immunity and Parkinson's Disease. 先天免疫和帕金森病。

IF 7.8 2区医学

Cold Spring Harbor perspectives in medicine Pub Date : 2025-06-16 DOI: 10.1101/cshperspect.a041640

Davina B Oludipe, Xiaoqing Du, Samia Akter, Chen Zhang, R Lee Mosley, Howard E Gendelman, Susmita Sil

引用次数: 0

Adaptive Immunity and Parkinson's Disease. 适应性免疫和帕金森病

IF 7.8 2区医学

Cold Spring Harbor perspectives in medicine Pub Date : 2025-06-16 DOI: 10.1101/cshperspect.a041639

Wassim Elyaman

引用次数: 0

Lipid Alterations and Pathogenic Roles in Synucleinopathies. 脂质改变及其在突触核蛋白病中的致病作用。

IF 7.8 2区医学

Cold Spring Harbor perspectives in medicine Pub Date : 2025-06-09 DOI: 10.1101/cshperspect.a041646

Estela Area-Gómez, Saranna Fanning, Ulf Dettmer

{"title":"Lipid Alterations and Pathogenic Roles in Synucleinopathies.","authors":"Estela Area-Gómez, Saranna Fanning, Ulf Dettmer","doi":"10.1101/cshperspect.a041646","DOIUrl":"https://doi.org/10.1101/cshperspect.a041646","url":null,"abstract":"Mounting evidence highlights a role for lipid alterations and defects in lipid signaling in age-related neurodegenerative diseases such as Parkinson's disease (PD) and related conditions (collectively referred to as synucleinopathies). This growing interest is driven by several key findings: (1) lipid membranes are components of Lewy bodies and Lewy neurites, which are prototypical proteinaceous intraneuronal inclusions of PD and other synucleinopathies, primarily composed of α-synuclein (αS); (2) αS shares structural similarities with lipid-binding proteins and has been reported to bind to lipids; (3) glucocerebrosidase, a key enzyme in sphingolipid metabolism, is a major PD risk factor; (4) other enzymes involved in glycolipid and phospholipid regulation, such as diacylglycerol kinase-θ and fatty acid elongase-7, also contribute to PD risk; (5) αS alterations impact lipid homeostasis; (6) αS transiently binds lipid membranes, affecting its conformation. Given these findings, we review what is known about the role of lipids in normal αS biology as well as in the pathogenesis of PD and related conditions. We also highlight areas where further research is warranted.","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2025-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144257468","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

Clinical Management of Parkinson's Disease: Features, Diagnosis, and Principles of Treatment. 帕金森病的临床管理：特征、诊断和治疗原则。

IF 7.8 2区医学

Cold Spring Harbor perspectives in medicine Pub Date : 2025-06-09 DOI: 10.1101/cshperspect.a041638

Bhavana Patel, Ashley Rawls, Tracy Tholanikunnel, Michael S Okun

引用次数: 0

Immunotherapies for Childhood Cancer. 儿童癌症免疫疗法。

IF 7.8 2区医学

Cold Spring Harbor perspectives in medicine Pub Date : 2025-06-02 DOI: 10.1101/cshperspect.a041574

Jeong A Park, Nai-Kong V Cheung

{"title":"Immunotherapies for Childhood Cancer.","authors":"Jeong A Park, Nai-Kong V Cheung","doi":"10.1101/cshperspect.a041574","DOIUrl":"10.1101/cshperspect.a041574","url":null,"abstract":"Children are surviving cancer in greater numbers than ever. Over the last 50 years, substantial advancements in pediatric cancer treatment have resulted in an 85% 5-year survival rate. Nonetheless, a notable 10%-15% of patients encounter relapse or develop refractory disease, leading to significantly lower survival. Recent attempts to further intensify cytotoxic chemotherapy have failed due to either severe toxicities or ineffectiveness, highlighting the need for new treatment strategies. Immunotherapies are emerging and expanding their clinical application to a wide array of cancers, including those affecting children. In pediatric cancers, monoclonal antibodies targeting GD2 have demonstrated durable radiographic and histologic responses in neuroblastoma (NB), and CD19-targeted bispecific antibodies (BsAbs) and chimeric antigen receptor (CAR) T cells have likewise changed the outlook for refractory acute lymphoblastic leukemia (ALL) in children. This review discusses the clinical development of immunotherapies for pediatric cancers, focusing on pediatric ALL and NB, two major pediatric cancers transformed by immunotherapy, updates on the recent advancements in immunotherapies, and further discusses the future directions of immunotherapy for pediatric cancers.","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12128874/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141619555","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

Corrigendum: The Gut-Brain Axis in Parkinson's Disease. 勘误：帕金森病的肠脑轴。

IF 7.8 2区医学

Cold Spring Harbor perspectives in medicine Pub Date : 2025-06-02 DOI: 10.1101/cshperspect.a041885

Virginia Gao, Carl V Crawford, Jacqueline Burré

引用次数: 0

Autoantigen-Specific Immunotherapies for the Prevention and Treatment of Type 1 Diabetes. 预防和治疗 1 型糖尿病的自身抗原特异性免疫疗法。

IF 7.8 2区医学

Cold Spring Harbor perspectives in medicine Pub Date : 2025-06-02 DOI: 10.1101/cshperspect.a041598

Mark Peakman, Pere Santamaria

引用次数: 0

Imaging Tumor Metabolism. 肿瘤代谢成像。

IF 7.8 2区医学

Cold Spring Harbor perspectives in medicine Pub Date : 2025-06-02 DOI: 10.1101/cshperspect.a041551

Thomas Ruan, Kayvan R Keshari

引用次数: 0

Ovarian Cancer Therapy. 卵巢癌治疗。

IF 7.8 2区医学

Cold Spring Harbor perspectives in medicine Pub Date : 2025-05-19 DOI: 10.1101/cshperspect.a038232

Diana Miao, Ursula A Matulonis, Rebecca L Porter

{"title":"Ovarian Cancer Therapy.","authors":"Diana Miao, Ursula A Matulonis, Rebecca L Porter","doi":"10.1101/cshperspect.a038232","DOIUrl":"https://doi.org/10.1101/cshperspect.a038232","url":null,"abstract":"Significant advances in basic and translational research have improved our understanding of the molecular alterations and biological vulnerabilities of the different histologic subsets of epithelial ovarian cancer (EOC). This has led to clinical trials that have incorporated novel agents based on molecular aspects into the treatment paradigm for both newly diagnosed and recurrent disease. The past decade has witnessed several regulatory approvals in the United States and Europe for the treatment of EOC, including the antiangiogenic agent, bevacizumab, poly(ADP-ribose) polymerase inhibitors in various therapeutic settings, and the antibody-drug conjugate (ADC), mirvetuximab soravtansine. Immune checkpoint inhibitors do not demonstrate substantial activity as single agents in ovarian cancer, except for the rare entity of microsatellite instability (MSI) high ovarian cancer. Current research is focused on new treatment paradigms such as ADCs, genetically specific therapies, and other novel immunotherapies such as bispecific antibodies, radioligand therapies, cellular therapies, and vaccines. In addition, combination efforts are focused on incorporating conventional chemotherapy, targeted therapies, immune-oncology drugs, and/or novel agents to improve outcomes for patients with newly diagnosed as well as recurrent EOC. This review will focus on the management of high-grade serous ovarian cancer, the most common type of EOC, accounting for ∼75% of cases. Recent advances in the management of rarer histologic subtypes with distinct molecular and clinical characteristics, including clear cell, mucinous, endometrioid, and low-grade serous, will be briefly discussed. Non-EOCs, including germ cell and sex cord stromal tumors and their treatment, have been reviewed elsewhere [see Ray-Coquard et al. (2019). N Engl J Med 381: 2416-2428. doi:10.1056/NEJMoa1911361].","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144101487","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

Metabolic Reprogramming in Human Cancer Patients and Patient-Derived Models. 人类癌症患者和患者衍生模型的代谢重编程。

IF 7.8 2区医学

Cold Spring Harbor perspectives in medicine Pub Date : 2025-05-05 DOI: 10.1101/cshperspect.a041552

Teresa W-M Fan, Richard M Higashi, Andrew N Lane

{"title":"Metabolic Reprogramming in Human Cancer Patients and Patient-Derived Models.","authors":"Teresa W-M Fan, Richard M Higashi, Andrew N Lane","doi":"10.1101/cshperspect.a041552","DOIUrl":"10.1101/cshperspect.a041552","url":null,"abstract":"Stable isotope-resolved metabolomics delineates reprogrammed intersecting metabolic networks in human cancers. Knowledge gained from in vivo patient studies provides the \"benchmark\" for cancer models to recapitulate. It is particularly difficult to model patients' tumor microenvironment (TME) with its complex cell-cell/cell-matrix interactions, which shapes metabolic reprogramming crucial to cancer development/drug resistance. Patient-derived organotypic tissue cultures (PD-OTCs) represent a unique model that retains an individual patient's TME. PD-OTCs of non-small-cell lung cancer better recapitulated the in vivo metabolic reprogramming of patient tumors than the patient-derived tumor xenograft (PDTX), while enabling interrogation of immunometabolic response to modulators and TME-dependent resistance development. Patient-derived organoids (PDOs) are also good models for reconstituting TME-dependent metabolic reprogramming and for evaluating therapeutic responses. Single-cell based 'omics on combinations of PD-OTC and PDO models will afford an unprecedented understanding on TME dependence of human cancer metabolic reprogramming, which should translate into the identification of novel metabolic targets for regulating TME interactions and drug resistance.","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12047743/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141619556","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