Advances in experimental medicine and biology最新文献_第9页

Uncovering Novel Drugs that Restore Vision Using Orthogonal Pooling in Zebrafish. 利用正交池法在斑马鱼中发现恢复视力的新药。

4区医学

Advances in experimental medicine and biology Pub Date : 2025-01-01 DOI: 10.1007/978-3-031-76550-6_80

Justine O'Brien, Patrizia Colucci, Yolanda Alvarez, Breandán N Kennedy

引用次数: 0

Frequency and Pattern of Gene Therapy Clinical Trials for Inherited Retinal Diseases. 遗传性视网膜疾病基因治疗临床试验的频率和模式。

4区医学

Advances in experimental medicine and biology Pub Date : 2025-01-01 DOI: 10.1007/978-3-031-76550-6_15

Hossein Ameri, Niranjana Kesavamoorthy, Dara N Bruce

{"title":"Frequency and Pattern of Gene Therapy Clinical Trials for Inherited Retinal Diseases.","authors":"Hossein Ameri, Niranjana Kesavamoorthy, Dara N Bruce","doi":"10.1007/978-3-031-76550-6_15","DOIUrl":"10.1007/978-3-031-76550-6_15","url":null,"abstract":"This study describes worldwide gene therapy clinical trials aimed at treating inherited retinal diseases (IRD). The information was collected through 15 different international registries including clinicaltrials.gov . There have been 101 gene therapy clinical trials targeting IRD up until the end of 2022. Seventy-seven trials employed gene augmentation using viral vectors; other approaches included inhibitory RNA (9), encapsulated cell technology (6), systemic approach (1), and observational trials (8). The most common clinical trial phase was phase 1/2 (46), followed by phase 3 (12). One trial led to an FDA-approved treatment. Sixty-nine trials were conducted in a single country, and 32 trials were multinational; The USA had the highest share in both categories. Retinitis pigmentosa was the most common disease targeted (39), followed by RPE65-mediated retinal dystrophy (13), Leber hereditary optic neuropathy (13), choroideremia (10 and achromatopsia (8), Leber congenital amaurosis (4), X-linked retinoschisis (4), Stargardt disease (4), Bietti's crystalline dystrophy (2), autosomal dominant optic atrophy (1), and Gyrate atrophy (1). For gene augmentation trials, adeno-associated virus was the most commonly used viral vector (70 trials-90%).","PeriodicalId":7270,"journal":{"name":"Advances in experimental medicine and biology","volume":"1468 ","pages":"89-93"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143389717","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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 Caveolin-1 Required for Retinal Neuroprotection? 视网膜神经保护需要小窝蛋白-1吗？

4区医学

Advances in experimental medicine and biology Pub Date : 2025-01-01 DOI: 10.1007/978-3-031-76550-6_47

Olawale O Bankole, Michael H Elliott

引用次数: 0

Extracellular Matrix Gene Expression Patterns in Retinal Wound Healing: A Comparative Study Between Mouse and Zebrafish Laser Injury Models. 视网膜创伤愈合中的细胞外基质基因表达模式：小鼠和斑马鱼激光损伤模型的比较研究。

4区医学

Advances in experimental medicine and biology Pub Date : 2025-01-01 DOI: 10.1007/978-3-031-76550-6_35

Laura Jahnke, Volker Enzmann

引用次数: 0

Unveiling the Research Void: Exploring the Reproductive Effects of PFAS Compounds on Male Health. 揭示研究空白：探索PFAS化合物对男性健康的生殖影响。

4区医学

Advances in experimental medicine and biology Pub Date : 2025-01-01 DOI: 10.1007/978-3-031-82990-1_7

Haoyang Qu, Yating Han, Chenglu Wang, Dongwang Zheng, Ya Ni, Xiang Xiao

{"title":"Unveiling the Research Void: Exploring the Reproductive Effects of PFAS Compounds on Male Health.","authors":"Haoyang Qu, Yating Han, Chenglu Wang, Dongwang Zheng, Ya Ni, Xiang Xiao","doi":"10.1007/978-3-031-82990-1_7","DOIUrl":"https://doi.org/10.1007/978-3-031-82990-1_7","url":null,"abstract":"Per- and polyfluoroalkyl substances (PFAS) represent an emerging concern for male reproductive health. Epidemiological studies have reported associations between increased PFAS exposure and reduced semen quality parameters, lower sperm counts, and potential alterations in reproductive hormone levels. Toxicology research has revealed possible mechanisms including blood-testis barrier disruption, oxidative stress, interference with testicular cell function, and epigenetic changes. However, significant uncertainties remain regarding definitive exposure-response relationships, developmental windows of heightened vulnerability, combined mixture effects, and causality interpretation, given limitations inherent to observational studies. Ongoing investigation of short-chain and replacement PFAS compounds is also critically needed. Additionally, directly connecting the mechanistic insights from animal models to human fertility impacts remains challenging. While controlled toxicology studies have described pathways by which PFAS could impair cellular functioning in the testes, uncertainty persists in extrapolating these experimental effects to real-world human exposures and sperm parameter declines reported epidemiologically. Overall, current findings suggest PFAS may contribute to declining male reproductive function, but additional clarification through well-designed longitudinal cohort studies integrated with mechanistic animal work is still warranted to confirm exposure-fertility links across a range of PFAS types and inform evidence-based public health mitigation strategies.","PeriodicalId":7270,"journal":{"name":"Advances in experimental medicine and biology","volume":"1469 ","pages":"127-162"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143956067","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Intraflagellar Transport (IFT) and Sperm Formation. 鞭毛内运输（IFT）与精子形成。

4区医学

Advances in experimental medicine and biology Pub Date : 2025-01-01 DOI: 10.1007/978-3-031-82990-1_17

Yunhao Liu, Yu Fang, Opeyemi Dhikhirullahi, Ling Zhang, Zhibing Zhang

引用次数: 0

A Comprehensive Bibliometric Analysis of Orchitis Research from 1980 to 2023. 1980 - 2023年睾丸炎研究文献计量学综合分析

4区医学

Advances in experimental medicine and biology Pub Date : 2025-01-01 DOI: 10.1007/978-3-031-82990-1_10

Haoyang Qu, Qiubei Liu, Dongwang Zheng, Ya Ni, Xiang Xiao

{"title":"A Comprehensive Bibliometric Analysis of Orchitis Research from 1980 to 2023.","authors":"Haoyang Qu, Qiubei Liu, Dongwang Zheng, Ya Ni, Xiang Xiao","doi":"10.1007/978-3-031-82990-1_10","DOIUrl":"https://doi.org/10.1007/978-3-031-82990-1_10","url":null,"abstract":"Orchitis, an inflammation of the testes, presents significant implications for male fertility and has been a focal area of scientific inquiry over the past four decades. This study employs a comprehensive bibliometric analysis to assess the progression of global research on orchitis from 1980 to 2023. Drawing from a dataset of 1586 publications indexed in the Web of Science Core Collection, we uncover emerging patterns, collaborations, and pivotal works that have shaped the field. The United States, China, and Germany emerge as leading contributors, while the Journal of Urology stands out as a primary publishing avenue. The results highlight the increasing recognition of autoimmune responses, alongside infectious agents, as key contributors to orchitis. Moreover, molecules such as TNF-α, IL-6, and IFN-γ are identified as central to the disease's pathology. The dynamic interplay of testosterone and regulatory T cells is underscored as a determinant of the testicular immune milieu. Notably, disruptions in the blood-testis barrier (BTB) and germ cell apoptosis emerge as pivotal consequences of the condition. This analysis underscores the expansive and multidisciplinary nature of orchitis research, revealing a consistent growth in collaborative endeavors. In summary, our findings catalog the evolution of orchitis research, providing a consolidated perspective on past achievements and signposting future research avenues. Such insights are instrumental for researchers aiming to navigate the complexities of orchitis and its multifaceted impact on male reproductive health.","PeriodicalId":7270,"journal":{"name":"Advances in experimental medicine and biology","volume":"1469 ","pages":"207-243"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143965872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

MicroRNAs Fine-Tune Brain and Body Communication in Health and Disease : MicroRNAs in Brain-Body Communication. MicroRNAs微调健康和疾病中的脑和身体通讯：脑-体通讯中的MicroRNAs。

4区医学

Advances in experimental medicine and biology Pub Date : 2025-01-01 DOI: 10.1007/978-3-031-89525-8_12

David C Henshall

{"title":"MicroRNAs Fine-Tune Brain and Body Communication in Health and Disease : MicroRNAs in Brain-Body Communication.","authors":"David C Henshall","doi":"10.1007/978-3-031-89525-8_12","DOIUrl":"https://doi.org/10.1007/978-3-031-89525-8_12","url":null,"abstract":"MicroRNAs are short noncoding RNAs that post-transcriptionally regulate gene expression. Their function is to fine-tune protein levels, thereby reducing gene expression noise. MicroRNAs serve critical functions during brain development, sculpting the transcript landscapes that direct cell proliferation, migration, and differentiation. In the mature brain, microRNAs impose stability on gene expression patterns to protect the constancy of network behavior while also facilitating the mechanics of synaptic plasticity. Brain diseases feature alterations in levels of specific microRNAs which influence injury and repair processes including changes to neuronal and glia morphology, immune and inflammatory responses, and cellular metabolism. MicroRNAs are also found within the extracellular space, including circulating in the cerebrospinal fluid and plasma. Characterization of biofluid microRNAs indicates some have CNS origins leading to the idea that microRNAs function in local and long-distance signalling. This chapter reviews the microRNA pathway and functions in the brain, the physical forms of extracellular microRNAs including encapsulation within extracellular vesicles, the changes to circulating microRNAs in brain diseases and evidence that circulating microRNAs come from the brain, and the effects, if any, of extracellular microRNA on recipient cells. The chapter also reviews the clinical applications of these findings, including diagnostic point-of-care testing and as therapeutic targets.","PeriodicalId":7270,"journal":{"name":"Advances in experimental medicine and biology","volume":"1477 ","pages":"311-337"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144180675","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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 Connection Between the Appetite-Regulatory Peptides Ghrelin and GLP-1 and Alcohol Use Disorder. 食欲调节肽Ghrelin和GLP-1与酒精使用障碍的关系

4区医学

Advances in experimental medicine and biology Pub Date : 2025-01-01 DOI: 10.1007/978-3-031-89525-8_8

Elisabet Jerlhag

{"title":"The Connection Between the Appetite-Regulatory Peptides Ghrelin and GLP-1 and Alcohol Use Disorder.","authors":"Elisabet Jerlhag","doi":"10.1007/978-3-031-89525-8_8","DOIUrl":"https://doi.org/10.1007/978-3-031-89525-8_8","url":null,"abstract":"The body-brain connection is well-established, further evidenced by studies on the orexigenic peptide ghrelin and the anorexigenic peptide glucagon-like peptide-1 (GLP-1). The ghrelin pathway consists of several substrates, which has been studied in relation to alcohol-related responses. Preclinical studies have found that central ghrelin infusions elevate alcohol intake, whereas suppression of the ghrelin receptors (GHSR) attenuates alcohol-related responses. On a similar note, the endogenous GHSR inverse agonist, LEAP2, and the precursor of ghrelin, DAG, block the stimulatory properties of alcohol and reduce alcohol intake. GLP-1 receptor agonists are currently approved for the treatment of type 2 diabetes and obesity. Recent advances in rodents have extended the pharmacological relevance of these GLP-1 receptor agonists as they mitigate alcohol-related responses. Specifically, in animal models reflecting alcohol use disorder (AUD) all tested GLP-1 receptor agonists reduce alcohol intake, suppress the motivation to consume alcohol, and prevent relapse drinking, effects most likely driven by an attenuation of alcohol-induced reward. Additional experiments were conducted in attempts to define areas and circuits responsible for ghrelin and GLP-1 to control alcohol-related responses. Specifically, areas associated with reward were defined as important modulatory areas. In summary, the ghrelin pathway and GLP-1 participate in the pathophysiology of AUD, thereby providing tentative treatment targets.","PeriodicalId":7270,"journal":{"name":"Advances in experimental medicine and biology","volume":"1477 ","pages":"229-241"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144179696","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Hematopoietic Stem Cell Transplantation in Sickle Cell Disease. 造血干细胞移植治疗镰状细胞病。

4区医学

Advances in experimental medicine and biology Pub Date : 2025-01-01 DOI: 10.1007/978-3-031-84988-6_10

Mohammed Essa, Mohsen Alzahrani, Ali D Alahmari, Mahmoud Aljurf

{"title":"Hematopoietic Stem Cell Transplantation in Sickle Cell Disease.","authors":"Mohammed Essa, Mohsen Alzahrani, Ali D Alahmari, Mahmoud Aljurf","doi":"10.1007/978-3-031-84988-6_10","DOIUrl":"https://doi.org/10.1007/978-3-031-84988-6_10","url":null,"abstract":"Sickle cell disease (SCD) is the most common inherited hemoglobinopathy worldwide with more than 300,000 babies in the world born every year with this disease. The clinical presentation and severity can be variable depending on many factors such as disease genotype, geographical location, environmental factors, and inheritance of other genetic abnormalities. The survival of patients with SCD have improved over last 3 decades where the expected median survival is more than 50 years for patients living in the developed countries. This improved survival is secondary to simple prophylactic and therapeutic interventions such as blood transfusions, prophylactic antibiotics, and vaccination. Disease modifying agents such as hydroxyurea contributed to the improved all disease outcome, survival, and quality of life. Over last 2 decades, curative options such as allogeneic stem cell transplant have gained popularity and increased evidence of safety and efficacy as the only curative option for SCD. However, there are many challenges to consider in patients who undergo hematopoietic stem cell transplantation (HSCT) that may affect the outcome. In this chapter, multiple challenges will be discussed including the indications of HSCT, choosing the appropriate donor and how to prevent and manage the unique or common post-transplant complications. Elaborative sections will focus on conditioning regimens choices in matched related donor HSCT. In addition, challenges in regards to various approaches of alternative donor transplant will be thoroughly discussed. Finally, long term effects and recommended follow up will be described.","PeriodicalId":7270,"journal":{"name":"Advances in experimental medicine and biology","volume":"1475 ","pages":"177-191"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144245646","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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