Current gene therapy最新文献_第5页

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 3.8 4区医学

Current gene therapy Pub Date : 2024-07-31 DOI: 10.2174/0115665232305431240726113347

Abhijit Debnath, Rupa Mazumder, Avijit Mazumder, Soumya Tripathi, Arpita Dua, Rajesh 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 Singh, Saloni Mangal, Jahanvi Sanchitra, Pratibha Pandey, Biplab Pal, Hema Chaudhary, Parul Sharma, Shikha Srivastava","doi":"10.2174/0115665232305431240726113347","DOIUrl":"https://doi.org/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":""},"PeriodicalIF":3.8,"publicationDate":"2024-07-31","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

Alternative Splicing of Pre-mRNA Matters in Oral Diseases. 口腔疾病中的前核糖核酸替代剪接问题。

IF 3.8 4区医学

Current gene therapy Pub Date : 2024-07-23 DOI: 10.2174/0115665232302948240718050212

Mingyang Yue, Rong Jia

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 : 2024-07-03 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":"https://doi.org/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.Aim: 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":""},"PeriodicalIF":3.8,"publicationDate":"2024-07-03","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

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 3.6 4区医学

Current gene therapy Pub Date : 2024-06-10 DOI: 10.2174/0115665232291487240603093218

Uttam Prasad Panigrahy, Rahul Subhash Buchade, Sandhya S, Anoop Kumar N, Sachinkumar Dnyaneshwar Gunjal, E 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 S, Anoop Kumar N, Sachinkumar Dnyaneshwar Gunjal, E Selvakumari, Narendra Kumar Pandey, Ankita Wal","doi":"10.2174/0115665232291487240603093218","DOIUrl":"https://doi.org/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.Result: 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":""},"PeriodicalIF":3.6,"publicationDate":"2024-06-10","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

The Role and Treatment Strategies of Ammonia-Related Metabolism in Tumor Microenvironment. 氨相关代谢在肿瘤微环境中的作用和治疗策略

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-06-10 DOI: 10.2174/0115665232301222240603100840

Qizhen Ye, Dan Li, Yi Zou, Ying Yuan

{"title":"The Role and Treatment Strategies of Ammonia-Related Metabolism in Tumor Microenvironment.","authors":"Qizhen Ye, Dan Li, Yi Zou, Ying Yuan","doi":"10.2174/0115665232301222240603100840","DOIUrl":"https://doi.org/10.2174/0115665232301222240603100840","url":null,"abstract":"Tumor cells achieve their adaptability through various metabolic reprogramming processes. Among them, ammonia, as a traditional metabolic waste, plays an increasingly important role in the tumor microenvironment along with its associated metabolites. Other cells in the microenvironment can also reshape the immune status of the microenvironment by regulating ammonia-related metabolism, and targeting this metabolic aspect has emerged as a potential strategy for tumor treatment. In this study, we have systematically reviewed the source and destination of ammonia in tumor cells, as well as the links between ammonia and other biological processes. We have also analyzed the ammonia-related metabolic regulation of other cells (including T cells, macrophages, dendritic cells, natural killer cells, myeloid-derived suppressor cells, and stromal cells) in the tumor microenvironment, and summarized the tumor treatment methods that target this metabolism. Through ammonia-related metabolic reprogramming, tumor cells obtain the energy they need for rapid growth and proliferation. Multiple immune cells and stromal cells in the microenvironment also interact with each other through this metabolic regulation, ultimately leading to immune suppression. Despite the heterogeneity of tumors and the complexity of cellular functions, further research into therapeutic interventions targeting ammonia-related metabolism is warranted. This review has focused on the role and regulation of ammonia-related metabolism in tumor cells and other cells in the microenvironment, and highlighted the efficacy and prospects of targeted ammonia-related metabolism therapy.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141300274","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 3.6 4区医学

Current gene therapy Pub Date : 2024-05-24 DOI: 10.2174/0115665232303436240515071754

Aya Al Othman, Anna Polyanskaya, Mikhail Durymanov

引用次数: 0

Development of Cell and Gene Therapies for Clinical Use in the US and EU: Summary of Regulatory Guidelines 在美国和欧盟开发用于临床的细胞和基因疗法：监管指南摘要

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-04-27 DOI: 10.2174/0115665232306205240419091414

Anand Rotte

{"title":"Development of Cell and Gene Therapies for Clinical Use in the US and EU: Summary of Regulatory Guidelines","authors":"Anand Rotte","doi":"10.2174/0115665232306205240419091414","DOIUrl":"https://doi.org/10.2174/0115665232306205240419091414","url":null,"abstract":"Recent decades have seen advancements in the management and treatment of difficult-- to-treat diseases such as cancer. A special class of therapeutics called cell and gene therapy has been introduced in the past 10 years. Cell and gene therapy products have strengthened the treatment options for life-threatening diseases with unmet clinical needs and also provided the possibility of a potential cure for the disease in some of the patients. Cell and gene therapy products are gaining recognition, and the interest in clinical development of cell and gene therapy products is increasing. Moreover, as the class of cell and gene therapy products is relatively new, there is a limited regulatory experience in the development, and the developers of the cell and gene therapy products can often be puzzled with an array of questions on regulations. The current review intends to provide a basic understanding of regulatory guidelines from the FDA and EMA that are applicable to cell and gene therapy products. Essentials such as which office is responsible for the evaluation of applications, which regulatory class/pathway is appropriate for development, and what are the quality, nonclinical and clinical studies that are needed to support the application are discussed in the article. In addition, a summary of regulatory designations and the post-approval requirements, such as Risk Evaluation and Mitigation Strategies (REMS) and long-term follow- up, is included in the article. Developers (referred to as ‘sponsors’ in this article) of cell and gene therapies can use the respective guidance documents and other specific review articles cited in this review for detailed information on the topics.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":"33 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-04-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140810163","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

Role of Non-coding RNAs on the Radiotherapy Sensitivity and Resistance in Cancer Cells 非编码 RNA 对癌症细胞放疗敏感性和耐受性的作用

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-04-27 DOI: 10.2174/0115665232301727240422092311

Fatemeh Jalali-Zefrei, Seyed Mehdi Mousavi, Kourosh Delpasand, Mohammad Shourmij, Soghra Farzipour

引用次数: 0

Evolution of Prime Editing Systems: Move Forward to the Treatment of Hereditary Diseases 基因编辑系统的演变：向治疗遗传性疾病迈进

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-04-16 DOI: 10.2174/0115665232295117240405070809

Olga V. Volodina, Anastasia R. Fabrichnikova, Arina A. Anuchina, Olesya S. Mishina, Alexander V. Lavrov, Svetlana A. Smirnikhina

{"title":"Evolution of Prime Editing Systems: Move Forward to the Treatment of Hereditary Diseases","authors":"Olga V. Volodina, Anastasia R. Fabrichnikova, Arina A. Anuchina, Olesya S. Mishina, Alexander V. Lavrov, Svetlana A. Smirnikhina","doi":"10.2174/0115665232295117240405070809","DOIUrl":"https://doi.org/10.2174/0115665232295117240405070809","url":null,"abstract":": The development of gene therapy using genome editing tools recently became relevant. With the invention of programmable nucleases, it became possible to treat hereditary diseases due to introducing targeted double strand break in the genome followed by homology directed repair (HDR) or non-homologous end-joining (NHEJ) reparation. CRISPR-Cas9 is more efficient and easier to use in comparison with other programmable nucleases. To improve the efficiency and safety of this gene editing tool, various modifications CRISPR-Cas9 basis were created in recent years, such as prime editing – in this system, Cas9 nickase is fused with reverse transcriptase and guide RNA, which contains a desired correction. Prime editing demonstrates equal or higher correction efficiency as HDR-mediated editing and much less off-target effect due to inducing nick. There are several studies in which prime editing is used to correct mutations in which researchers reported little or no evidence of off-target effects. The system can also be used to functionally characterize disease variants. However, prime editing still has several limitations that could be further improved. The effectiveness of the method is not yet high enough to apply it in clinical trials. Delivery of prime editors is also a big challenge due to their size. In the present article, we observe the development of the platform, and discuss the candidate proteins for efficiency enhancing, main delivery methods and current applications of prime editing.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":"44 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140586457","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

Efficacy and Safety of Pembrolizumab Monotherapy or Combined Therapy in Patients with Metastatic Triple-negative Breast Cancer: A Systematic Review and Meta-Analysis of Randomised Controlled Trials Pembrolizumab单药或联合疗法对转移性三阴性乳腺癌患者的疗效和安全性：随机对照试验的系统回顾和元分析

IF 3.6 4区医学

Current gene therapy Pub Date : 2024-03-12 DOI: 10.2174/0115665232283880240301035621

Mahmood Araghi, Farshad Gharebakhshi, Fatemeh Faramarzi, Alireza mafi, Tahoora Mousavi, Mina Alimohammadi, Hussein Soleimantabar

{"title":"Efficacy and Safety of Pembrolizumab Monotherapy or Combined Therapy in Patients with Metastatic Triple-negative Breast Cancer: A Systematic Review and Meta-Analysis of Randomised Controlled Trials","authors":"Mahmood Araghi, Farshad Gharebakhshi, Fatemeh Faramarzi, Alireza mafi, Tahoora Mousavi, Mina Alimohammadi, Hussein Soleimantabar","doi":"10.2174/0115665232283880240301035621","DOIUrl":"https://doi.org/10.2174/0115665232283880240301035621","url":null,"abstract":"Background: Metastatic Triple-negative Breast Cancer (mTNBC) is the most aggressive form of breast cancer, with a greater risk of metastasis and recurrence. Research studies have published in-depth analyses of the advantages and disadvantages of pembrolizumab, and early data from numerous trials suggests that patients with mTNBC have had remarkable outcomes. This meta-analysis compares the data from numerous relevant studies in order to evaluate the safety and efficacy of pembrolizumab monotherapy or combination therapies for mTNBC. Methods: To identify eligible RCTs, a thorough literature search was carried out using electronic databases. CMA software was utilized to perform heterogeneity tests using fixed and random-effects models. Results: According to our pooled data, the median Progression-free Survival (PFS) was 2.66 months, and the median overall survival (OS) was 12.26 months. Furthermore, by comparing efficacy indicators between PD-L1–positive and PD-L1–negative groups, a correlation was found between the overexpression of PD-L1 with OS, PFS, and ORR. Patients with PD-L1-positive tumors had a higher response rate, with an ORR of 21.1%, compared to the patients with PD-L1-negative tumors. The ORR for first-line immunotherapy was higher than that of ≥second-line immunotherapy. In addition, pembrolizumab plus combination treatment resulted in a pooled incidence of immune-related adverse events of 22.7%. Conclusion: A modest response to pembrolizumab monotherapy was detected in the mTNBC patients. Furthermore, a better outcome from pembrolizumab treatment may be predicted by PD-L1-- positive status, non-liver/lung metastases, combination therapy, and first-line immunotherapy. Pembrolizumab, in combination with chemotherapy, may be more beneficial for patients whose tumors are PD-L1 positive.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":"290 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140115293","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