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IF 4 3区 医学 Q1 Medicine
Dm Disease-A-Month
Pub Date : 2024-06-20 DOI: 10.1016/S0011-5029(24)00095-6
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引用次数: 0
Information for Readers 读者信息
IF 4 3区 医学 Q1 Medicine
Dm Disease-A-Month
Pub Date : 2024-06-20 DOI: 10.1016/S0011-5029(24)00096-8
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引用次数: 0
C2: Editorial Board C2：编辑委员会
IF 4 3区 医学 Q1 Medicine
Dm Disease-A-Month
Pub Date : 2024-06-20 DOI: 10.1016/S0011-5029(24)00094-4
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引用次数: 0
The efficacy and safety of gene therapy approaches in Parkinson's disease: A systematic review 帕金森病基因治疗方法的有效性和安全性：系统综述。
IF 4 3区 医学 Q1 Medicine
Dm Disease-A-Month
Pub Date : 2024-06-06 DOI: 10.1016/j.disamonth.2024.101754
Chiranjeevee R. Saravanan MBBS , Reem Faiz Hussein Eisa MBBS , Elizabeth Gaviria MD , Amani Algubari MD , Kiran Kishor Chandrasekar MBBS , Pugazhendi Inban MD , Priyadarshi Prajjwal MBBS , Hyma Bamba MBBS , Gurmehar Singh MBBS , Mohammed Dheyaa Marsool Marsool MBChB , Srikanth Gadam MD

Background

Parkinson's disease (PD) is a neurodegenerative disorder characterized by the progressive loss of dopaminergic neurons in the brain. Despite existing treatments, there remains an unmet need for therapies that can halt or reverse disease progression. Gene therapy has been tried and tested for a variety of illnesses, including PD. The goal of this systematic review is to assess gene therapy techniques' safety and effectiveness in PD clinical trials.

Methods

Online databases PubMed/Medline, and Cochrane were used to screen the studies for this systematic review. The risk of bias of the included studies was assessed using standard tools.

Results

Gene therapy can repair damaged dopaminergic neurons from the illness or deal with circuit anomalies in the basal ganglia connected to Parkinson's disease symptoms. Rather than only treating symptoms, this neuroprotective approach alters the illness itself. Medication for gene therapy is currently administered at the patient's bedside. It can hyperactivate specific brain circuits associated with motor dysfunction. PD therapies are developing quickly, and there aren't enough head-to-head trials evaluating the safety and effectiveness of available treatments. When choosing an advanced therapy, patient-specific factors should be considered in addition to the effectiveness and safety of each treatment option.

Conclusion

In comparison to conventional therapies, gene therapy may be advantageous for PD. It may minimize side effects, relieve symptoms, and offer dependable dopamine replacement.

背景：帕金森病（PD）是一种神经退行性疾病，其特征是大脑中多巴胺能神经元的逐渐丧失。尽管已有一些治疗方法，但对于能阻止或逆转疾病进展的疗法的需求仍未得到满足。基因疗法已在包括帕金森病在内的多种疾病中进行了尝试和测试。本系统综述旨在评估基因治疗技术在帕金森病临床试验中的安全性和有效性：本系统综述使用在线数据库 PubMed/Medline 和 Cochrane 筛选研究。采用标准工具评估了纳入研究的偏倚风险：基因疗法可以修复因疾病而受损的多巴胺能神经元，或治疗与帕金森病症状相关的基底节回路异常。这种神经保护方法不仅能治疗症状，还能改变疾病本身。基因治疗的药物目前是在病人床边使用的。它可以过度激活与运动功能障碍相关的特定脑回路。帕金森病疗法发展迅速，但目前还没有足够的正面试验来评估现有疗法的安全性和有效性。在选择先进疗法时，除了考虑每种治疗方案的有效性和安全性外，还应考虑患者的具体因素：结论：与传统疗法相比，基因疗法可能对帕金森病具有优势。结论：与传统疗法相比，基因疗法可能对帕金森病有优势，它可以最大限度地减少副作用，缓解症状，并提供可靠的多巴胺替代。
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引用次数: 0
Title Page 标题页
IF 4 3区 医学 Q1 Medicine
Dm Disease-A-Month
Pub Date : 2024-06-01 DOI: 10.1016/S0011-5029(24)00083-X
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引用次数: 0
Information for Readers 读者信息
IF 4 3区 医学 Q1 Medicine
Dm Disease-A-Month
Pub Date : 2024-06-01 DOI: 10.1016/S0011-5029(24)00084-1
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引用次数: 0
Ovarian tumors in the pediatric population: An update 儿童卵巢肿瘤：最新进展。
IF 4 3区 医学 Q1 Medicine
Dm Disease-A-Month
Pub Date : 2024-06-01 DOI: 10.1016/j.disamonth.2024.101691
Berrin Ergun-Longmire MD, Donald E. Greydanus MD, DrHC (Athens)

Research reveals that 1% of neoplasms in females under 17 years of age are ovarian neoplasms and though usually benign, malignant tumors may occur in the pediatric age group. This review considers various current concepts of these tumors including the epidemiology, risk factors, clinical presentations, diagnosis, differential diagnosis, and treatment options including the need to provide fertility-sparing surgery as well as their potential impacts on the psychological well-being of children and adolescents.

We gathered data from the published articles ranging from studies, meta-analyses, retrospective studies, and reviews. We focused on the articles published in English between January 1, 2000, and August 31, 2023. Only a few articles published prior to 2000 were included for historical perspective.

研究显示，17 岁以下女性的肿瘤中有 1%是卵巢肿瘤，虽然通常是良性的，但恶性肿瘤也可能发生在儿童群体中。这篇综述探讨了目前关于这些肿瘤的各种概念，包括流行病学、风险因素、临床表现、诊断、鉴别诊断、治疗方案（包括提供保胎手术的必要性）以及它们对儿童和青少年心理健康的潜在影响。我们从已发表的研究、荟萃分析、回顾性研究和综述等文章中收集数据。我们重点研究了 2000 年 1 月 1 日至 2023 年 8 月 31 日期间发表的英文文章。只有少数 2000 年之前发表的文章被纳入其中，以了解历史。
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引用次数: 0
Foreword for: Ovarian tumors in the pediatric population: An update 前言：儿科卵巢肿瘤：最新进展
IF 4 3区 医学 Q1 Medicine
Dm Disease-A-Month
Pub Date : 2024-06-01 DOI: 10.1016/j.disamonth.2024.101690
Jerrold B. Leikin MD (Senior EditorDisease-A-Month)
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引用次数: 0
C2: Editorial Board C2：编辑委员会
IF 4 3区 医学 Q1 Medicine
Dm Disease-A-Month
Pub Date : 2024-06-01 DOI: 10.1016/S0011-5029(24)00082-8
{"title":"C2: Editorial Board","authors":"","doi":"10.1016/S0011-5029(24)00082-8","DOIUrl":"https://doi.org/10.1016/S0011-5029(24)00082-8","url":null,"abstract":"","PeriodicalId":51017,"journal":{"name":"Dm Disease-A-Month","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0011502924000828/pdfft?md5=157f457b29d60be190f1b7ebf18135c3&pid=1-s2.0-S0011502924000828-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141313983","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Foreword: Autoimmune disorders and the eye 前言：自身免疫性疾病与眼睛。
IF 4 3区 医学 Q1 Medicine
Dm Disease-A-Month
Pub Date : 2024-05-01 DOI: 10.1016/j.disamonth.2024.101710
Jerrold B. Leikin MD (Senior Editor)
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引用次数: 0
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