Current gene therapy最新文献_第7页

Untangling the Genetic Threads of Alzheimer's: Insights into Risk Factors and Biomarkers. 解开阿尔茨海默氏症的遗传线索：洞察风险因素和生物标志物。

IF 3.8 4区医学

Current gene therapy Pub Date : 2025-01-07 DOI: 10.2174/0115665232338503241227111640

Atabak Naiyeri, Amin Moqadami, Mohammad Khalaj-Kondori

{"title":"Untangling the Genetic Threads of Alzheimer's: Insights into Risk Factors and Biomarkers.","authors":"Atabak Naiyeri, Amin Moqadami, Mohammad Khalaj-Kondori","doi":"10.2174/0115665232338503241227111640","DOIUrl":"https://doi.org/10.2174/0115665232338503241227111640","url":null,"abstract":"Dementia is a comprehensive term that refers to illnesses characterized by a decline in cognitive memory and other cognitive functions, affecting a person's overall ability to operate. The exact causes of dementia are unknown to this day. The heterogeneity of Alzheimer's indicates the contribution of genetic polymorphism to this disease. This disease is the most prevalent and damaging illness. Studies indicate that the global prevalence of Alzheimer's disease (AD) exceeds 26 million individuals. Investigation of variations in many genes indicates that these variations may be linked to the susceptibility to AD. Additional genetic factors could potentially influence AD. Analysis of several single-nucleotide polymorphisms in this context reveals a correlation between certain variants and AD. Regardless, Alzheimer's disease is always influenced by a particular APOE gene allele. The study's findings indicate that risk of Alzheimer's disease (AD) is linked to polymorphisms in the following genes: BDNF, presenilin-1 (PS-1), presenilin-2 (PS-2), LRP, APP, CTSD,5-6HT, TREM2, TNF-α, LPL, Clusterin (CLU), SORL1 (Sortilin-Related Receptor), PICALM, Complement Receptor 1 (CR1), and APOE genes.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142946279","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

CRISPR/Cas System: A Powerful Strategy to Improve Monogenic Human Diseases as Therapeutic Delivery; Current Applications and Challenges. CRISPR/Cas系统：改善人类单基因疾病治疗的有力策略当前的应用和挑战。

IF 3.8 4区医学

Current gene therapy Pub Date : 2025-01-07 DOI: 10.2174/0115665232345516241119070150

Fatemeh Saberi, Zeinab Yousefi-Najafabadi, Forough Shams, Zeinab Dehghan, Sepideh Ahmadi, Tayyebeh Pilehchi, Effat Noori, Zeinab Esmaeelzadeh, Maryam Bazgiri, Rezvan Mohammadi, Farzaneh Khani, Marzieh Sameni, Parisa Moradbeigi, Gholam Ali Kardar, Mohammad Salehi, Yong Teng, Vahid Jajarmi

{"title":"CRISPR/Cas System: A Powerful Strategy to Improve Monogenic Human Diseases as Therapeutic Delivery; Current Applications and Challenges.","authors":"Fatemeh Saberi, Zeinab Yousefi-Najafabadi, Forough Shams, Zeinab Dehghan, Sepideh Ahmadi, Tayyebeh Pilehchi, Effat Noori, Zeinab Esmaeelzadeh, Maryam Bazgiri, Rezvan Mohammadi, Farzaneh Khani, Marzieh Sameni, Parisa Moradbeigi, Gholam Ali Kardar, Mohammad Salehi, Yong Teng, Vahid Jajarmi","doi":"10.2174/0115665232345516241119070150","DOIUrl":"https://doi.org/10.2174/0115665232345516241119070150","url":null,"abstract":"The 5,000 to 8,000 monogenic diseases are inherited disorders leading to mutations in a single gene. These diseases usually appear in childhood and sometimes lead to morbidity or premature death. Although treatments for such diseases exist, gene therapy is considered an effective and targeted method and has been used in clinics for monogenic diseases since 1989. Monogenic diseases are good candidates for novel therapeutic technologies like gene editing approaches to repair gene mutations. Clustered regularly interspaced short palindromic repeats (CRISPR)-based systems, the pioneer and effective gene editing tool, are utilized for ex vivo and in vivo treatment of monogenic diseases. The current review provides an overview of recent therapeutic applications of CRISPR-based gene editing in monogenic diseases in in vivo and ex vivo models. Furthermore, this review consolidates strategies aimed at providing new treatment options with gene therapy, thereby serving as a valuable reference for advancing the treatment landscape for patients with monogenic disorders.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142946275","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

Identification of the Metabolic Characteristic of Organ Fibrosis Using Microbial Analysis on RNA-seq Data. 利用RNA-seq数据的微生物分析鉴定器官纤维化的代谢特征。

IF 3.8 4区医学

Current gene therapy Pub Date : 2025-01-03 DOI: 10.2174/0115665232257596231011110813

Zitong Wang, Ping Wang, Guoyou He, Liang Cheng, Tianyu Li, Yan Wang, Hong Li

{"title":"Identification of the Metabolic Characteristic of Organ Fibrosis Using Microbial Analysis on RNA-seq Data.","authors":"Zitong Wang, Ping Wang, Guoyou He, Liang Cheng, Tianyu Li, Yan Wang, Hong Li","doi":"10.2174/0115665232257596231011110813","DOIUrl":"https://doi.org/10.2174/0115665232257596231011110813","url":null,"abstract":"Background: Fibrosis refers to abnormal deposition of extracellular matrix, which leads to organ dysfunction. Metabolic alterations, especially enhanced glycolysis and suppressed fatty acid oxidation, are recognized as an essential pathogenic process of fibrosis. Recently, several reports indicate that the changes in microbiota composition are associated with metabolic disorders, suggesting microbes may contribute to organ fibrosis by regulating metabolic processes.Method: In this study, microbial reannotation was carried out on the RNA-seq data of fibrotic organs. Then, the microbial composition differences among healthy and fibrotic organ samples were determined by alpha and beta diversity analysis. Common and specific microbial markers of fibrosis were also identified by LEfSe. After that, the correlation analysis of the characteristic microbe-- gene-functional pathway was conducted to confirm the effects of microbes on host metabolism.Result: The results showed that the microbial composition significantly differed between healthy and diseased organs. Besides, the common characteristic microbes interacted closely with each other and contributed to fibrosis through symbiosis or inhibition. The largest proportion in fibrosis organs was Proteobacteria, which was the main source of pathogenic microbes.Conclusion: Further study found that the metabolic alteration driven by common and special characteristic microbes in fibrotic organs focused on the processes related to glycolysis and fatty acid metabolism.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142946276","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

Acetylresveratrol (AC-Res): An Evolving Frontier in Modulating Gene Expression. 乙酰白藜芦醇（AC-Res）：调节基因表达的前沿技术。

IF 3.8 4区医学

Current gene therapy Pub Date : 2025-01-01 DOI: 10.2174/0115665232291487240603093218

Uttam Prasad Panigrahy, Rahul Subhash Buchade, Sandhya Subhadra, Anoop Kumar Narayanan, Sachinkumar Dnyaneshwar Gunjal, Ezhilarasan Selvakumari, Narendra Kumar Pandey, Ankita Wal

{"title":"Acetylresveratrol (AC-Res): An Evolving Frontier in Modulating Gene Expression.","authors":"Uttam Prasad Panigrahy, Rahul Subhash Buchade, Sandhya Subhadra, Anoop Kumar Narayanan, Sachinkumar Dnyaneshwar Gunjal, Ezhilarasan Selvakumari, Narendra Kumar Pandey, Ankita Wal","doi":"10.2174/0115665232291487240603093218","DOIUrl":"10.2174/0115665232291487240603093218","url":null,"abstract":"Background: Acetylresveratrol (AC-Res), to date, is a powerful stilbene phytoalexin generated organically or as a component of a plant's defensive system, is a significant plant phenolic chemical portion and is investigated as a therapy option for a number of disorders. Owing to its inadequate stabilisation and considerable conformation rigidity, the utility of AC-Res as a medication is limited.Objective: The current review article outlined the structure of AC-Res, their methods of activity, and the latest technological progress in the administration of these molecules. It is conceivable to deduce that AC-Res has a variety of consequences for the cellular functions of infected cells.Methods: The literature survey for the present article was gathered from the authentic data published by various peer-reviewed publishers employing Google Scholar and PubMedprioritizing Scopus and Web of Science indexed journals as the search platform focusing on AC-Res pharmacological actions, particularly in the English language.Results: Despite its extensive spectrum of biological and therapeutic applications, AC-Res has become a source of increasing concern. Depending on the researchers, AC-Res possesses radioprotective, cardioprotective, neurological, anti-inflammatory, and anti-microbial potential. It also has anti-cancer and antioxidant properties.Conclusion: To avoid non-specific cytotoxicity, optimization efforts are presently emphasizing the possible usage of AC-Res based on nanocrystals, nanoparticles and dendrimers, and nanocrystals. Finally, while using AC-Res in biology is still a way off, researchers agree that if they continue to explore it, AC-Res and similar parts will be recognized as actual possibilities for a variety of things in the next years.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"210-226"},"PeriodicalIF":3.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141300273","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

Exploring Gene Therapy: The Next Generation of Colorectal Cancer Treatment. 探索基因疗法：下一代结直肠癌治疗。

IF 3.8 4区医学

Current gene therapy Pub Date : 2025-01-01 DOI: 10.2174/0115665232326072240809061901

Raj Kamal, Priyanka Paul, Diksha, Ankit Awasthi

引用次数: 0

Hereditary Patterns and Genetic Associations in Obsessive-Compulsive Disorder (OCD): Neuropsychiatric Insights, Genetic Influences, and Treatment Perspectives. 强迫症（OCD）的遗传模式和遗传关联：神经精神洞察力、遗传影响和治疗视角》（Neuropsychiatric Insights, Genetic Influences, and Treatment Perspectives）。

IF 3.8 4区医学

Current gene therapy Pub Date : 2025-01-01 DOI: 10.2174/0115665232316708240828063527

Abhinay Dhiman, Sidharth Mehan, Zuber Khan, Aarti Tiwari, Ghanshyam Das Gupta, Acharan Singh Narula

{"title":"Hereditary Patterns and Genetic Associations in Obsessive-Compulsive Disorder (OCD): Neuropsychiatric Insights, Genetic Influences, and Treatment Perspectives.","authors":"Abhinay Dhiman, Sidharth Mehan, Zuber Khan, Aarti Tiwari, Ghanshyam Das Gupta, Acharan Singh Narula","doi":"10.2174/0115665232316708240828063527","DOIUrl":"10.2174/0115665232316708240828063527","url":null,"abstract":"Obsessive-Compulsive Disorder (OCD), a prevalent neuropsychiatric condition, affects approximately 2%-3% of the global population. This paper provides an extensive overview of OCD, detailing its clinical manifestations, neurobiological underpinnings, and therapeutic approaches. It examines OCD's classification shift in the DSM-5, the role of the cortico-striatothalamo- cortical pathway in its development, and the various factors contributing to its etiology, such as genes, environmental factors, and genetic predispositions. The challenges in diagnosing OCD and the effectiveness of both psychological and pharmacotherapeutic treatments are discussed. The paper also highlights the significant overlap between OCD and other mental health disorders, emphasizing its impact on global disability. Moreover, the role of genetic factors in OCD, including twin studies and gene association studies, is elaborated, underscoring the complex interplay of hereditary and environmental influences in its manifestation. The review further delves into the polygenic nature of OCD, illustrating how multiple genes contribute to its development, and explores the implications of genetic studies in understanding the disorder's complexity. Additionally, this research study delves into the concept of polygenic inheritance in complex diseases, highlighting the role of multiple genes in increasing OCD risk. A Genome-wide Association Study (GWAS) is employed to assess Single Nucleotide Polymorphisms (SNPs) to unearth genetic associations with OCD. This comprehensive analysis provides valuable insights into OCD's genetic landscape, paving the way for enhanced diagnostic approaches and treatment modalities.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"257-316"},"PeriodicalIF":3.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12246748/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105187","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

miRNA-Targeted Vaccines: A Promising Approach for Viral Attenuation and Immunogenicity Enhancement. miRNA 靶向疫苗：病毒衰减和免疫原性增强的有效方法。

IF 3.3 4区医学

Current gene therapy Pub Date : 2025-01-01 DOI: 10.2174/0115665232305431240726113347

Abhijit Debnath, Rupa Mazumder, Avijit Mazumder, Soumya Tripathi, Arpita Dua, Rajesh Kumar Singh, Saloni Mangal, Jahanvi Sanchitra, Pratibha Pandey, Biplab Pal, Hema Chaudhary, Parul Sharma, Shikha Srivastava

{"title":"miRNA-Targeted Vaccines: A Promising Approach for Viral Attenuation and Immunogenicity Enhancement.","authors":"Abhijit Debnath, Rupa Mazumder, Avijit Mazumder, Soumya Tripathi, Arpita Dua, Rajesh Kumar Singh, Saloni Mangal, Jahanvi Sanchitra, Pratibha Pandey, Biplab Pal, Hema Chaudhary, Parul Sharma, Shikha Srivastava","doi":"10.2174/0115665232305431240726113347","DOIUrl":"10.2174/0115665232305431240726113347","url":null,"abstract":"MicroRNAs (miRNAs) have emerged as a significant tool in the realm of vaccinology, offering novel approaches to vaccine development. This study investigates the potential of miRNAs in the development of advanced vaccines, with an emphasis on how they regulate immune response and control viral replication. We go over the molecular features of miRNAs, such as their capacity to direct post-transcriptional regulation toward mRNAs, hence regulating the expression of genes in diverse tissues and cells. This property is harnessed to develop live attenuated vaccines that are tissue-specific, enhancing safety and immunogenicity. The review highlights recent advancements in using miRNA-targeted vaccines against viruses like influenza, poliovirus, and tick-borne encephalitis virus, demonstrating their attenuated replication in specific tissues while retaining immunogenicity. We also explored the function of miRNAs in the biology of cancer, highlighting their potential to develop cancer vaccines through targeting miRNAs that are overexpressed in tumor cells. The difficulties in developing miRNA vaccines are also covered in this work, including delivery, stability, off-target effects, and the requirement for individualized cancer treatment plans. We wrap off by discussing the potential of miRNA vaccines and highlighting how they will influence the development of vaccination techniques for cancer and infectious diseases in the future.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"360-373"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141874377","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

Human Virus-Like Proteins: Implications for Gene Therapy. 人类病毒样蛋白：对基因疗法的影响。

IF 3.8 4区医学

Current gene therapy Pub Date : 2025-01-01 DOI: 10.2174/0115665232303436240515071754

Aya Al Othman, Anna Polyanskaya, Mikhail Durymanov

引用次数: 0

Knockdown of hsa_circ_0102231 Impedes the Progression of Liver Cancer through the miR-873-SOX4 Axis. 敲除 hsa_circ_0102231 会通过 miR-873-SOX4 轴阻碍肝癌的进展

IF 3.8 4区医学

Current gene therapy Pub Date : 2025-01-01 DOI: 10.2174/0115665232301878240627051455

Jingyu Qian, Banghong Jiang, Zhongqiang Qin, Yulin Tan

{"title":"Knockdown of hsa_circ_0102231 Impedes the Progression of Liver Cancer through the miR-873-SOX4 Axis.","authors":"Jingyu Qian, Banghong Jiang, Zhongqiang Qin, Yulin Tan","doi":"10.2174/0115665232301878240627051455","DOIUrl":"10.2174/0115665232301878240627051455","url":null,"abstract":"Background: Hepatocellular carcinoma (HCC) is one of the most intractable tumors in the world due to its high rate of recurrence and heterogeneity.Aims: The objective of this study was to investigate the role of circular RNA 0102231 (hsa_circ_ 0102231) in the progression of liver cancer.Methods: In this study, quantitative polymerase chain reaction experiments were performed to quantify the hsa_circ_0102231 level in different liver cancer cell lines. Bioinformatics analysis, as well as a dual-luciferase reporter and RNA pull-down assay, were used to identify putative hsa_circ_ 0102231 downstream targets. Colony formation and CCK8 assays were utilized to examine cell proliferation, whereas Transwell assays were employed to monitor cell migration. Lastly, the role of hsa_circ_0102231 in liver cancer was assessed in a subcutaneous xenograft model.Results: The expression of hsa_circ_0102231 increased significantly in HepG2 and Huh-7 cells compared with controls, and hsa_circ_0102231 knockdown inhibited cell proliferation and migration in vitro and in vivo. Bioinformatics analysis, as well as a dual-luciferase reporter and RNA pulldown assay, revealed that miR-873 and SOX4 were hsa_circ_0102231 downstream targets. miR-873 inhibition or SOX4 overexpression rescued the proliferation and migration of HepG2 and Huh-7 cells after hsa_circ_0102231 knockdown. Furthermore, SOX4 overexpression reversed the miR-873-induced inhibition of cell migration and proliferation in vitro.Conclusion: These results show that hsa_circ_0102231 knockdown impedes the progression of liver cancer by regulating the miR-873/SOX4 axis. However, further studies are needed to determine whether hsa_circ_0102231 may be a therapeutic target in liver cancer.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"317-326"},"PeriodicalIF":3.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141497366","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

Gene Therapy for Skin Aging. 皮肤老化基因疗法。

IF 3.8 4区医学

Current gene therapy Pub Date : 2025-01-01 DOI: 10.2174/0115665232286489240320051925

Fawzy A Saad

{"title":"Gene Therapy for Skin Aging.","authors":"Fawzy A Saad","doi":"10.2174/0115665232286489240320051925","DOIUrl":"10.2174/0115665232286489240320051925","url":null,"abstract":"Extrinsic and intrinsic factors contribute to skin aging; nonetheless, they are intertwined. Moreover, intrinsic skin aging mirrors age-related declines in the entire human body's internal organs. There is evidence that skin appearance is an indicator of the general health of somebody or a visual certificate of health. Earlier, it was apparent that the intrinsic factors are unalterable, but the sparkling of skin aging gene therapy on the horizon is changing this narrative. Skin aging gene therapy offers tools for skin rejuvenation, natural beauty restoration, and therapy for diseases affecting the entire skin. However, skin aging gene therapy is an arduous and sophisticated task relying on precise interim stimulation of telomerase to extend telomeres and wend back the biological clock in the hopes to find the fountain of youth, while preserving cells innate biological features. Finding the hidden fountain of youth will be a remarkable discovery for promoting aesthetics medicine, genecosmetics, and healthy aging. Caloric restriction offers ultimate health benefits and a reproducible way to promote longevity in mammals, while delaying age-related diseases. Moreover, exercise further enhances these health benefits. This article highlights the potential of skin aging gene therapy and foretells the emerging dawn of the genecosmetics era.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"2-9"},"PeriodicalIF":3.8,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140287092","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