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Patient-Derived-Xenografts in Mice: A Preclinical Platform for Cancer Research. 小鼠中的患者衍生的X基因移植物：癌症研究的临床前平台。
IF 7.8 2区 医学
Cold Spring Harbor perspectives in medicine Pub Date : 2024-07-01 DOI: 10.1101/cshperspect.a041381
Emiliano Cocco, Elisa de Stanchina
{"title":"Patient-Derived-Xenografts in Mice: A Preclinical Platform for Cancer Research.","authors":"Emiliano Cocco, Elisa de Stanchina","doi":"10.1101/cshperspect.a041381","DOIUrl":"10.1101/cshperspect.a041381","url":null,"abstract":"<p><p>The use of patient-derived xenografts (PDXs) has dramatically improved drug development programs. PDXs (1) reproduce the pathological features and the genomic profile of the parental tumors more precisely than other preclinical models, and (2) more faithfully predict therapy response. However, PDXs have limitations. These include the inability to completely capture tumor heterogeneity and the role of the immune system, the low engraftment efficiency of certain tumor types, and the consequences of the human-host interactions. Recently, the use of novel mouse strains and specialized engraftment techniques has enabled the generation of \"humanized\" PDXs, partially overcoming such limitations. Importantly, establishing, characterizing, and maintaining PDXs is costly and requires a significant regulatory, administrative, clinical, and laboratory infrastructure. In this review, we will retrace the historical milestones that led to the implementation of PDXs for cancer research, review the most recent innovations in the field, and discuss future avenues to tackle deficiencies that still exist.</p>","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11216185/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10213390","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
Toxin-Induced Animal Models of Parkinson's Disease. 毒素诱导的帕金森病动物模型。
IF 7.8 2区 医学
Cold Spring Harbor perspectives in medicine Pub Date : 2024-07-01 DOI: 10.1101/cshperspect.a041643
Kim Tieu, Said S Salehe, Harry J Brown
{"title":"Toxin-Induced Animal Models of Parkinson's Disease.","authors":"Kim Tieu, Said S Salehe, Harry J Brown","doi":"10.1101/cshperspect.a041643","DOIUrl":"https://doi.org/10.1101/cshperspect.a041643","url":null,"abstract":"<p><p>The debilitating motor symptoms of Parkinson's disease (PD) result primarily from the degenerative nigrostriatal dopaminergic pathway. To elucidate pathogenic mechanisms and evaluate therapeutic strategies for PD, numerous animal models have been developed. Understanding the strengths and limitations of these models can significantly impact the choice of model, experimental design, and data interpretation. Herein, we systematically review the literature over the past decade. Some models no longer serve the purpose of PD models. The primary objectives of this review are: First, to assist new investigators in navigating through available animal models and making appropriate selections based on the objective of the study. Emphasis will be placed on common toxin-induced murine models. And second, to provide an overview of basic technical requirements for assessing the nigrostriatal pathway's pathology, structure, and function.</p>","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141476108","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
Current Status of Clinical Trials Design and Outcomes in Retinal Gene Therapy. 视网膜基因治疗的临床试验设计和结果现状。
IF 7.8 2区 医学
Cold Spring Harbor perspectives in medicine Pub Date : 2024-07-01 DOI: 10.1101/cshperspect.a041301
Boris Rosin, Eyal Banin, Jose-Alain Sahel
{"title":"Current Status of Clinical Trials Design and Outcomes in Retinal Gene Therapy.","authors":"Boris Rosin, Eyal Banin, Jose-Alain Sahel","doi":"10.1101/cshperspect.a041301","DOIUrl":"10.1101/cshperspect.a041301","url":null,"abstract":"<p><p>With the rapid expansion of methods encompassed by the term gene therapy, new trials exploring the safety and efficacy of these methods are initiated more frequently. As a result, important questions arise pertaining the design of these trials and patient participation. One of the most important aspects of any clinical trial is the ability to measure the trial's outcome in a manner that will reflect the effect of the treatment and allow its quantification, whether the trial is aimed at preservation or restoration of retinal cells (photoreceptors and others), vision, or both. Here we will review the existing methods for quantification of trial outcomes, stressing the importance of assessing the participant's visual function and not just visual acuity. We will also describe the key considerations in trial design. Finally, as patient safety remains the primary concern in any trial participation, we will outline the key principles in that regard.</p>","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11216172/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10215521","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
Interactions of Fatty Acid and Cholesterol Metabolism with Cellular Stress Response Pathways in Cancer. 癌症中脂肪酸和胆固醇代谢与细胞应激反应途径的相互作用
IF 7.8 2区 医学
Cold Spring Harbor perspectives in medicine Pub Date : 2024-07-01 DOI: 10.1101/cshperspect.a041548
Alina M Winkelkotte, Kamal Al-Shami, Adriano B Chaves-Filho, Felix C E Vogel, Almut Schulze
{"title":"Interactions of Fatty Acid and Cholesterol Metabolism with Cellular Stress Response Pathways in Cancer.","authors":"Alina M Winkelkotte, Kamal Al-Shami, Adriano B Chaves-Filho, Felix C E Vogel, Almut Schulze","doi":"10.1101/cshperspect.a041548","DOIUrl":"https://doi.org/10.1101/cshperspect.a041548","url":null,"abstract":"<p><p>Lipids have essential functions as structural components of cellular membranes, as efficient energy storage molecules, and as precursors of signaling mediators. While deregulated glucose and amino acid metabolism in cancer have received substantial attention, the roles of lipids in the metabolic reprogramming of cancer cells are less well understood. However, since the first description of de novo fatty acid biosynthesis in cancer tissues almost 70 years ago, numerous studies have investigated the complex functions of altered lipid metabolism in cancer. Here, we will summarize the mechanisms by which oncogenic signaling pathways regulate fatty acid and cholesterol metabolism to drive rapid proliferation and protect cancer cells from environmental stress. The review also discusses the role of fatty acid metabolism in metabolic plasticity required for the adaptation to changing microenvironments during cancer progression and the connections between fatty acid and cholesterol metabolism and ferroptosis.</p>","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141476106","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 Chicken or the Egg Dilemma: Understanding the Interplay between the Immune System and the β Cell in Type 1 Diabetes. 鸡还是蛋的两难选择：了解 1 型糖尿病中免疫系统与 β 细胞之间的相互作用。
IF 7.8 2区 医学
Cold Spring Harbor perspectives in medicine Pub Date : 2024-07-01 DOI: 10.1101/cshperspect.a041591
Maria Skjøtt Hansen, Pravil Pokharel, Jon Piganelli, Lori Sussel
{"title":"The Chicken or the Egg Dilemma: Understanding the Interplay between the Immune System and the β Cell in Type 1 Diabetes.","authors":"Maria Skjøtt Hansen, Pravil Pokharel, Jon Piganelli, Lori Sussel","doi":"10.1101/cshperspect.a041591","DOIUrl":"https://doi.org/10.1101/cshperspect.a041591","url":null,"abstract":"<p><p>In this review, we explore the complex interplay between the immune system and pancreatic β cells in the context of type 1 diabetes (T1D). While T1D is predominantly considered a T-cell-mediated autoimmune disease, the inability of human leukocyte antigen (HLA)-risk alleles alone to explain disease development suggests a role for β cells in initiating and/or propagating disease. This review delves into the vulnerability of β cells, emphasizing their susceptibility to endoplasmic reticulum (ER) stress and protein modifications, which may give rise to neoantigens. Additionally, we discuss the role of viral infections as contributors to T1D onset, and of genetic factors with dual impacts on the immune system and β cells. A greater understanding of the interplay between environmental triggers, autoimmunity, and the β cell will not only lead to insight as to why the islet β cells are specifically targeted by the immune system in T1D but may also reveal potential novel therapeutic interventions.</p>","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141476107","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
Mouse Models to Evaluate the Functional Role of the Tumor Microenvironment in Cancer Progression and Therapy Responses. 评估肿瘤微环境在癌症进展和治疗反应中的功能作用的小鼠模型
IF 7.8 2区 医学
Cold Spring Harbor perspectives in medicine Pub Date : 2024-07-01 DOI: 10.1101/cshperspect.a041411
Kathleen M McAndrews, Krishnan K Mahadevan, Raghu Kalluri
{"title":"Mouse Models to Evaluate the Functional Role of the Tumor Microenvironment in Cancer Progression and Therapy Responses.","authors":"Kathleen M McAndrews, Krishnan K Mahadevan, Raghu Kalluri","doi":"10.1101/cshperspect.a041411","DOIUrl":"10.1101/cshperspect.a041411","url":null,"abstract":"<p><p>The tumor microenvironment (TME) is a complex ecosystem of both cellular and noncellular components that functions to impact the evolution of cancer. Various aspects of the TME have been targeted for the control of cancer; however, TME composition is dynamic, with the overall abundance of immune cells, endothelial cells (ECs), fibroblasts, and extracellular matrix (ECM) as well as subsets of TME components changing at different stages of progression and in response to therapy. To effectively treat cancer, an understanding of the functional role of the TME is needed. Genetically engineered mouse models have enabled comprehensive insight into the complex interactions within the TME ecosystem that regulate disease progression. Here, we review recent advances in mouse models that have been employed to understand how the TME regulates cancer initiation, progression, metastasis, and response to therapy.</p>","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11216184/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139402161","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
Role of Tumor Cell Intrinsic and Host Autophagy in Cancer. 肿瘤细胞内在自噬和宿主自噬在癌症中的作用
IF 7.8 2区 医学
Cold Spring Harbor perspectives in medicine Pub Date : 2024-07-01 DOI: 10.1101/cshperspect.a041539
Jessie Yanxiang Guo, Eileen White
{"title":"Role of Tumor Cell Intrinsic and Host Autophagy in Cancer.","authors":"Jessie Yanxiang Guo, Eileen White","doi":"10.1101/cshperspect.a041539","DOIUrl":"10.1101/cshperspect.a041539","url":null,"abstract":"<p><p>Macroautophagy (autophagy hereafter) is an intracellular nutrient scavenging pathway induced by starvation and other stressors whereby cellular components such as organelles are captured in double-membrane vesicles (autophagosomes), whereupon their contents are degraded through fusion with lysosomes. Two main purposes of autophagy are to recycle the intracellular breakdown products to sustain metabolism and survival during starvation and to eliminate damaged or excess cellular components to suppress inflammation and maintain homeostasis. In contrast to most normal cells and tissues in the fed state, tumor cells up-regulate autophagy to promote their growth, survival, and malignancy. This tumor-cell-autonomous autophagy supports elevated metabolic demand and suppresses tumoricidal activation of the innate and adaptive immune responses. Tumor-cell-nonautonomous (e.g., host) autophagy also supports tumor growth by maintaining essential tumor nutrients in the circulation and tumor microenvironment and by suppressing an antitumor immune response. In the setting of cancer therapy, autophagy is a resistance mechanism to chemotherapy, targeted therapy, and immunotherapy. Thus, tumor and host autophagy are protumorigenic and autophagy inhibition is being examined as a novel therapeutic approach to treat cancer.</p>","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11216174/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139520131","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
Integrating Omics into Functional Biomarkers of Type 1 Diabetes. 将 Omics 整合到 1 型糖尿病的功能生物标记中。
IF 7.8 2区 医学
Cold Spring Harbor perspectives in medicine Pub Date : 2024-07-01 DOI: 10.1101/cshperspect.a041602
S Alice Long, Peter S Linsley
{"title":"Integrating Omics into Functional Biomarkers of Type 1 Diabetes.","authors":"S Alice Long, Peter S Linsley","doi":"10.1101/cshperspect.a041602","DOIUrl":"10.1101/cshperspect.a041602","url":null,"abstract":"<p><p>Biomarkers are critical to the staging and diagnosis of type 1 diabetes (T1D). Functional biomarkers offer insights into T1D immunopathogenesis and are often revealed using \"omics\" approaches that integrate multiple measures to identify involved pathways and functions. Application of the omics biomarker discovery may enable personalized medicine approaches to circumvent the more recently appreciated heterogeneity of T1D progression and treatment. Use of omics to define functional biomarkers is still in its early years, yet findings to date emphasize the role of cytokine signaling and adaptive immunity in biomarkers of progression and response to therapy. Here, we share examples of the use of omics to define functional biomarkers focusing on two signatures, T-cell exhaustion and T-cell help, which have been associated with outcomes in both the natural history and treatment contexts.</p>","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":7.8,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11216170/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141075805","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
Historical Perspectives of Parkinson's Disease: Early Clinical Descriptions and Neurological Therapies. 帕金森病的历史展望：早期临床描述和神经疗法。
IF 5.4 2区 医学
Cold Spring Harbor perspectives in medicine Pub Date : 2024-06-10 DOI: 10.1101/cshperspect.a041642
Christopher G Goetz
{"title":"Historical Perspectives of Parkinson's Disease: Early Clinical Descriptions and Neurological Therapies.","authors":"Christopher G Goetz","doi":"10.1101/cshperspect.a041642","DOIUrl":"https://doi.org/10.1101/cshperspect.a041642","url":null,"abstract":"<p><p>Although components of possible Parkinson's disease can be found in earlier documents, the first clear medical description was written in 1817 by James Parkinson. In the mid-1800s, Jean-Martin Charcot was particularly influential in refining and expanding this early description and in disseminating information internationally about Parkinson's disease. He separated the clinical spectrum of Parkinson's disease from multiple sclerosis and other disorders characterized by tremor, and he recognized cases that later would likely be classified among the parkinsonism-plus syndromes. Early treatments of Parkinson's disease were based on empirical observation, and anticholinergic drugs were used as early as the nineteenth century. The discovery of dopaminergic deficits in Parkinson's disease and the synthetic pathway of dopamine led to the first human trials of levodopa. Further historically important anatomical, biochemical, and physiological studies identified additional pharmacological and neurosurgical targets for Parkinson's disease and allow modern clinicians to offer an array of therapies aimed at improving function in this still incurable disease.</p>","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2024-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141300205","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
Lessons Learned from Cancer Metabolism for Physiology and Disease. 从癌症代谢中汲取生理和疾病方面的经验教训。
IF 5.4 2区 医学
Cold Spring Harbor perspectives in medicine Pub Date : 2024-06-10 DOI: 10.1101/cshperspect.a041554
Sydney L Campbell, Heather R Christofk
{"title":"Lessons Learned from Cancer Metabolism for Physiology and Disease.","authors":"Sydney L Campbell, Heather R Christofk","doi":"10.1101/cshperspect.a041554","DOIUrl":"https://doi.org/10.1101/cshperspect.a041554","url":null,"abstract":"<p><p>Tumor cells divide rapidly and dramatically alter their metabolism to meet biosynthetic and bioenergetic needs. Through studying the aberrant metabolism of cancer cells, other contexts in which metabolism drives cell state transitions become apparent. In this work, we will discuss how principles established by the field of cancer metabolism have led to discoveries in the contexts of physiology and tissue injury, mammalian embryonic development, and virus infection. We present specific examples of findings from each of these fields that have been shaped by the study of cancer metabolism. We also discuss the next important scientific questions facing these subject areas collectively. Altogether, these examples demonstrate that the study of \"cancer metabolism\" is indeed the study of cell metabolism in the context of a tumor, and undoubtedly discoveries from each of the fields discussed here will continue to build on each other in the future.</p>","PeriodicalId":10452,"journal":{"name":"Cold Spring Harbor perspectives in medicine","volume":" ","pages":""},"PeriodicalIF":5.4,"publicationDate":"2024-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141300206","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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