Current gene therapy最新文献

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Immune Modulation Strategies in Gene Therapy: Overcoming Immune Barriers and Enhancing Efficacy. 基因疗法中的免疫调节策略:克服免疫障碍,提高疗效。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2024-10-02 DOI: 10.2174/0115665232305409240918040639
Sivaprakasam S Amsaveni, Radha Mahendran, Vidhya C S, Dilip Kumar Chanchal, Sojomon Mathew, Mukesh Chandra Sharma, Jailani S, Syed Salman Ali
{"title":"Immune Modulation Strategies in Gene Therapy: Overcoming Immune Barriers and Enhancing Efficacy.","authors":"Sivaprakasam S Amsaveni, Radha Mahendran, Vidhya C S, Dilip Kumar Chanchal, Sojomon Mathew, Mukesh Chandra Sharma, Jailani S, Syed Salman Ali","doi":"10.2174/0115665232305409240918040639","DOIUrl":"https://doi.org/10.2174/0115665232305409240918040639","url":null,"abstract":"<p><p>The immune system presents significant obstacles to gene therapy, which has limited its use in treating many illnesses. New approaches are needed to overcome these problems and improve the effectiveness of gene therapy. This study explores several techniques to immune regulation within gene therapy, a cutting-edge discipline that aims to optimise results by fine-tuning the immune response. We cover new ways to control the immune system and deliver therapeutic genes just where they are needed, including influencing immunological checkpoints, causing immunotolerance, and making smart use of immunomodulatory drugs. In addition, the study provides insight into new developments in the design of less immunogenic gene delivery vectors, which allow for the extension of transgene expression with minimal adverse immune reactions. In order to maximise the efficacy of gene-based therapies, this review analyses these novel approaches and gives a thorough overview of the present state of the art by addressing obstacles and pointing the way toward future developments in immune regulation. Not only does their integration provide new opportunities for the creation of safer and more effective gene treatments, but it also contains the key to overcome current obstacles.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142364742","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 Gene Signatures Associated with COVID-19 across Children, Adolescents, and Adults in the Nasopharynx and Peripheral Blood by Using a Machine Learning Approach. 利用机器学习方法识别儿童、青少年和成人鼻咽部和外周血中与 COVID-19 相关的基因特征。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2024-09-27 DOI: 10.2174/0115665232316769240912061652
YuSheng Bao, JingXin Ren, Lei Chen, Wei Guo, KaiYan Feng, Tao Huang, Yu-Dong Cai
{"title":"Identification of Gene Signatures Associated with COVID-19 across Children, Adolescents, and Adults in the Nasopharynx and Peripheral Blood by Using a Machine Learning Approach.","authors":"YuSheng Bao, JingXin Ren, Lei Chen, Wei Guo, KaiYan Feng, Tao Huang, Yu-Dong Cai","doi":"10.2174/0115665232316769240912061652","DOIUrl":"https://doi.org/10.2174/0115665232316769240912061652","url":null,"abstract":"<p><strong>Background: </strong>Significant variations in immune profiles across different age groups manifest distinct clinical symptoms and prognoses in Coronavirus Disease 2019 (COVID-19) patients. Predominantly, severe COVID-19 cases that require hospitalization occur in the elderly, with the risk of severe illness escalating with age among young adults, children, and adolescents.</p><p><strong>Objective: </strong>This study aimed to delineate the unique immune characteristics of COVID-19 across various age groups and evaluate the feasibility of detecting COVID-19-induced immune alterations through peripheral blood analysis.</p><p><strong>Methods: </strong>By employing a machine learning approach, we analyzed gene expression data from nasopharyngeal and peripheral blood samples of COVID-19 patients across different age brackets. Nasopharyngeal data reflected the immune response to COVID-19 in the upper respiratory tract, while peripheral blood samples provided insights into the overall immune system status. Both datasets encompassed COVID-19 patients and healthy controls, with patients divided into children, adolescents, and adult age groups. The analysis included the expression levels of 62,703 genes per patient. Then, 9 feature-sequencing methods (least absolute shrinkage and selection operator, light gradient boosting machine, Monte Carlo feature selection, random forest, ridge regression, adaptive boosting, categorical boosting, extremely randomized trees, and extreme gradient boosting) were employed to evaluate the association of the genes with COVID-19. Key genes were then utilized to develop efficient classification models.</p><p><strong>Results: </strong>The findings identified specific markers: insulin-like growth factor binding protein 3 (downregulated in the peripheral blood of COVID-19 patients), interferon alpha-inducible protein 27 (upregulated), and SERPING1 (upregulated in nasopharyngeal tissues). In addition, fibulin-2 was downregulated in adolescent patients, but upregulated in the other groups, while epoxide hydrolase 3 was upregulated in healthy controls, but downregulated in children and adolescents.</p><p><strong>Conclusion: </strong>This study offers valuable insights into the local and systemic immune responses of COVID-19 patients across age groups, aiding in identifying potential therapeutic targets and formulating personalized treatment strategies.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142343126","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
Pan-Cancer Single-Cell Analysis Revealing the Heterogeneity of Cancer-Associated Fibroblasts in Skin Tumors. 泛癌单细胞分析揭示皮肤肿瘤中癌症相关成纤维细胞的异质性
IF 3.8 4区 医学
Current gene therapy Pub Date : 2024-09-25 DOI: 10.2174/0115665232331353240911080642
Yichi Zhang, Zhijie Zhao, Wenyi Huang, Byeong Seop Kim, Li Lin, Xin Li, Mengyuan Hou, Li Li, Yan Zhang, Wenjing Xi, Gang Chai
{"title":"Pan-Cancer Single-Cell Analysis Revealing the Heterogeneity of Cancer-Associated Fibroblasts in Skin Tumors.","authors":"Yichi Zhang, Zhijie Zhao, Wenyi Huang, Byeong Seop Kim, Li Lin, Xin Li, Mengyuan Hou, Li Li, Yan Zhang, Wenjing Xi, Gang Chai","doi":"10.2174/0115665232331353240911080642","DOIUrl":"https://doi.org/10.2174/0115665232331353240911080642","url":null,"abstract":"<p><strong>Background: </strong>Cancer-Associated Fibroblasts (CAFs) constitute a heterogeneous group of cells critical for the remodeling of the tumor microenvironment (TME). Given their significant impact on tumor progression, particularly in skin cancers, a deeper understanding of their characteristics and functions is essential.</p><p><strong>Methods: </strong>This study employed a single-cell transcriptomic analysis to explore the diversity of CAFs within three major types of skin cancer: basal cell carcinoma, melanoma, and head and neck squamous cell carcinoma. We applied analytical techniques, including Gene Ontology (GO), Kyoto Encyclopedia of Genes and Genomes (KEGG), Gene Set Enrichment Analysis (GSEA), pseudotime tracking, metabolic profiling, and stemness assessment to delineate and define the functional attributes of identified CAF subgroups.</p><p><strong>Results: </strong>Our analysis successfully delineated nine distinct CAF subgroups across the studied tumor types. Of particular interest, we identified a novel CAF subtype, designated as C0, exclusive to basal cell carcinoma. This subtype exhibits phenotypic traits associated with invasive and destructive capabilities, significantly correlating with the progression of basal cell carcinoma. The identification of this subgroup provides new insights into the role of CAFs in cancer biology and opens avenues for targeted therapeutic strategies.</p><p><strong>Conclusion: </strong>A pan-cancer analysis was performed on three cancers, BCC, MA, and HNSCC, focusing on tumor fibroblasts in TME. Unsupervised clustering categorized CAF into nine subpopulations, among which the C0 subpopulation had a strong correspondence with BCC-CAF and an invasive- destructive-related phenotype.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142343127","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
Target and Gene-Based Therapeutic Strategies against Pancreatic Cancer: Current and Future Prospects. 基于靶点和基因的胰腺癌治疗策略:当前和未来前景。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2024-09-24 DOI: 10.2174/0115665232320846240910055032
Medha Dwivedi, Sayak Sanyal, Sakshi Singh, Manish Dwivedi, Somali Sanyal
{"title":"Target and Gene-Based Therapeutic Strategies against Pancreatic Cancer: Current and Future Prospects.","authors":"Medha Dwivedi, Sayak Sanyal, Sakshi Singh, Manish Dwivedi, Somali Sanyal","doi":"10.2174/0115665232320846240910055032","DOIUrl":"https://doi.org/10.2174/0115665232320846240910055032","url":null,"abstract":"<p><p>Despite tremendous advancements in knowledge, diagnosis, and availability of both traditional and innovative treatments, pancreatic cancer remains a dangerous disease with a high death rate and dismal prognosis. The traditional strategy in adjuvant and palliative settings is still cytotoxic chemotherapy predicated on the purine derivative gemcitabine; nevertheless, there is an increasing need for new medicines that target the primary molecular pathways and pathophysiological abnormalities implicated. There is now just a tiny amount of evidence of therapeutic benefit when the targeted drug erlotinib is added to the conventional gemcitabine treatment. In preclinical and clinical trials, novel medications targeting mTOR, NF-κB, and proteasome, including the enzyme histone deacetylase, are currently being studied alongside the well-established monoclonal antibody treatments and small-molecule protein tyrosine kinase inhibitors. These novel medications may change the negative natural progression of this illness in conjunction with gene therapy and immunotherapy, both of which are undergoing clinical study. In this regard, leveraging miRNA manipulation to combat cancer is appealing due to its promise to deliver personalized treatment tailored to an individual's distinct gene or miRNA expression profile. Preclinical studies involving animals have showcased the effectiveness of miRNA-based therapies, with several of these treatments now progressing into human clinical trials for various malignancies and other medical conditions. This review describes the important developments of targeted therapeutics that are associated with pancreatic cancer and the discoveries which can help in dealing with this fatal malignancy in a more significant manner.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142343128","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
Characteristics of Oxidative Phosphorylation-Related Subtypes and Construction of a Prognostic Signature in Ovarian Cancer 氧化磷酸化相关亚型的特征及卵巢癌预后特征的构建
IF 3.6 4区 医学
Current gene therapy Pub Date : 2024-09-18 DOI: 10.2174/0115665232323373240905104033
Jiaojiao Lu, Shuai Zhen, Xu Li
{"title":"Characteristics of Oxidative Phosphorylation-Related Subtypes and Construction of a Prognostic Signature in Ovarian Cancer","authors":"Jiaojiao Lu, Shuai Zhen, Xu Li","doi":"10.2174/0115665232323373240905104033","DOIUrl":"https://doi.org/10.2174/0115665232323373240905104033","url":null,"abstract":"Background: Ovarian cancer is associated with a high mortality rate. Oxidative Phosphorylation (OXPHOS) is an active metabolic pathway in cancer; nevertheless, its role in ovarian cancer continues to be ambiguous. Therefore, the objective of this study was to identify the prognostic value of OXPHOS-related genes and the immune landscape in ovarian cancer. Methods: We obtained public ovarian cancer-related datasets from The Cancer Genome Atlas (TCGA) and Gene Expression Omnibus (GEO) databases and recognized OXPHOS-related genes from the GeneCards database and literature. Cox regression analyses were conducted to identify prognostic OXPHOS-related genes and develop a prognostic nomogram based on the OXPHOS score and clinicopathological features of patients. Functional enrichment analyses were employed to identify related processes. Results: A 12-gene signature was identified to classify the ovarian cancer patients into high- and low-risk groups. The Immunophenoscore (IPS) was higher in the OXPHOS score-high group than in the OXPHOS score-low group, suggesting a better response to immune checkpoint inhibitors. Functional enrichment analyses unveiled that OXPHOS-related genes were considerably abundant in a series of immune processes. The calibration curves of the constructed prognostic nomograms at 1, 2, and 3 years exhibited strong concordance between the anticipated and observed survival probabilities of ovarian cancer patients. Conclusion: We have constructed a prognostic model containing 12 OXPHOS-related genes and demonstrated its strong predictive value in ovarian cancer patients. OXPHOS has been found to be closely linked to immune infiltration and the reaction to immunotherapy, which may contribute to improving individualized treatment and prognostic evaluation in ovarian cancer.","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142260000","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
DeepLCRmiRNA: A Hybrid Neural Network Approach for Identifying Lung Cancer-Associated miRNAs. DeepLCRmiRNA:识别肺癌相关 miRNA 的混合神经网络方法
IF 3.8 4区 医学
Current gene therapy Pub Date : 2024-09-13 DOI: 10.2174/0115665232312364240902060458
Nitao Cheng, Chen Chen, Junliang Liu, Xuanchun Wang, Ziqi Gao, Ming Mao, Jingyu Huang
{"title":"DeepLCRmiRNA: A Hybrid Neural Network Approach for Identifying Lung Cancer-Associated miRNAs.","authors":"Nitao Cheng, Chen Chen, Junliang Liu, Xuanchun Wang, Ziqi Gao, Ming Mao, Jingyu Huang","doi":"10.2174/0115665232312364240902060458","DOIUrl":"https://doi.org/10.2174/0115665232312364240902060458","url":null,"abstract":"<p><strong>Introduction: </strong>Lung cancer stands as one of the most prevalent malignant neoplasms, with microRNAs (miRNAs) playing a pivotal role in the modulation of gene expression, impacting cancer cell proliferation, invasion, metastasis, immune escape, and resistance to therapy.</p><p><strong>Method: </strong>The intricate role of miRNAs in lung cancer underscores their significance as biomarkers for early detection and as novel targets for therapeutic intervention. Traditional approaches for the identification of miRNAs related to lung cancer, however, are impeded by inefficiencies and complexities.</p><p><strong>Results: </strong>In response to these challenges, this study introduced an innovative deep-learning strategy designed for the efficient and precise identification of lung cancer-associated miRNAs. Through comprehensive benchmark tests, our method exhibited superior performance relative to existing technologies.</p><p><strong>Conclusion: </strong>Further case studies have also confirmed the ability of our model to identify lung cancer-associated miRNAs that have undergone biological validation.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142281656","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
Proteogenomic Identification and Analysis of KIF5B as a Prognostic Signature for Hepatocellular Carcinoma. 作为肝细胞癌预后特征的 KIF5B 蛋白基因组鉴定与分析
IF 3.8 4区 医学
Current gene therapy Pub Date : 2024-09-06 DOI: 10.2174/0115665232308821240826075513
Lishuang Qi, Yilong Tan, Yunfei Zhou, Yihong Dong, Xun Yang, Shuyuan Chang, Lei Yu, Dabin Liu
{"title":"Proteogenomic Identification and Analysis of KIF5B as a Prognostic Signature for Hepatocellular Carcinoma.","authors":"Lishuang Qi, Yilong Tan, Yunfei Zhou, Yihong Dong, Xun Yang, Shuyuan Chang, Lei Yu, Dabin Liu","doi":"10.2174/0115665232308821240826075513","DOIUrl":"https://doi.org/10.2174/0115665232308821240826075513","url":null,"abstract":"<p><strong>Background: </strong>Metabolic disorders are significant risk factors for liver cancer, particularly Hepatocellular Carcinoma (HCC). However, the molecular genetic basis of metabolic reprogramming in the liver remains largely uncertain.</p><p><strong>Objective: </strong>This study aimed to investigate some novel prognostic biomarkers in HCC by using proteogenomic and transcriptomic analysis and explore the potential role of specific prognostic genes in HCC.</p><p><strong>Methods: </strong>Here, we have presented a proteogenomic analysis of 10 pairs of HCC. Protein co-expression and pathway analysis were performed to investigate the biological characteristics of HCC. Protein and mRNA expression profiles of multi-cohorts were integrated to detect novel prognostic protein markers of HCC. The carcinogenic roles of candidate prognostic markers were further evaluated by MTS assay, colony formation, monolayer wound healing assay, and xenograft models.</p><p><strong>Results: </strong>A total of 2086 proteins with significantly different expressions were detected in HCC. Pathways related to oncogenic signaling and insulin-related metabolism have been found to be dysregulated and differentially regulated in HCC. We have identified the novel prognostic biomarkers, KIF5B, involved in liver metabolic reprogramming. The biomarkers were identified using multivariable COX regression analysis from two independent proteomic datasets (Fudan Cohort and our recruited cohort) and the TCGA mRNA database. Both the protein and mRNA up-regulation of KIF5B have been found to be associated with a poor clinical outcome in HCC. Insulin activated the protein expression of KIF5B in HCC. Knocking out KIF5B expression by sgRNA decreased the protein expression of FASN and SCD1 and the intracellular triglyceride concentration. Silencing KIF5B suppressed HCC cell proliferation and colony formation in vitro, as well as HCC growth in xenograft models.</p><p><strong>Conclusion: </strong>Our findings have suggested KIF5B protein to function as a novel prognostic biomarker in HCC. KIF5B expression has been found to activate the AKT/mTOR pathway and reprogram triglyceride metabolism, leading to HCC development. Targeting KIF5B may be an effective strategy in the clinical treatment of HCC.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142153352","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
Delivery of Superoxide Dismutase 3 Gene with Baculoviruses Inhibits TNF-α Triggers Vascular Smooth Muscle Cell Proliferation and Inflammation. 用巴库洛病毒传递超氧化物歧化酶 3 基因可抑制 TNF-α 引发的血管平滑肌细胞增殖和炎症。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2024-09-05 DOI: 10.2174/0115665232308789240823052607
Shoa-Lin Lin, Song-Tay Lee, Shang-En Huang, Tsung-Hsien Chang, Yong-Jian Geng, Erna Sulistyowati, Jwu-Lai Yeh
{"title":"Delivery of Superoxide Dismutase 3 Gene with Baculoviruses Inhibits TNF-α Triggers Vascular Smooth Muscle Cell Proliferation and Inflammation.","authors":"Shoa-Lin Lin, Song-Tay Lee, Shang-En Huang, Tsung-Hsien Chang, Yong-Jian Geng, Erna Sulistyowati, Jwu-Lai Yeh","doi":"10.2174/0115665232308789240823052607","DOIUrl":"https://doi.org/10.2174/0115665232308789240823052607","url":null,"abstract":"<p><strong>Background: </strong>Superoxide dismutase 3 (SOD3), recognized as a potent free radical scavenger, exhibits antioxidant, anti-inflammatory, and anti-angiogenic properties. However, the molecular mechanisms underlying the protective effects of SOD3 on the vascular smooth muscle cell during atherosclerosis remain unclear.</p><p><strong>Objectives: </strong>This study aimed to investigate the efficacy of the baculovirus expressing SOD3 gene delivery to vascular smooth muscle cells (VSMCs) and investigate whether the overexpression of SOD3 mitigates cell proliferation and migration induced by tumor necrosis factor-α (TNF-α).</p><p><strong>Methods: </strong>A baculoviral vector containing SOD3 cDNA (vAcMBac-CMV-IE-SOD3) was constructed and utilized to deliver the SOD3 gene into primary rat VSMCs. Cells were stimulated with recombinant TNF-α, and then cell proliferation and migration were evaluated using the bromodeoxyuridine and wound healing assay. Western blot was used to verify the expression of cell cycle regulators, cellular mediators, and proliferative biomarkers. Zymography, immunofluorescence staining, and ELISA assay were conducted to assess the expression levels of matrix metalloproteinases.</p><p><strong>Results: </strong>The results demonstrated efficient and non-cytotoxic transduction of vAcMBac- CMV-IE-SOD3 in VSMCs. SOD3 overexpression significantly suppressed cell proliferation and motility by inhibiting cell cycle regulators in TNF-α-induced cells. TNF-α elevated protein levels of phospho-ERK and phospho-Akt were reduced markedly by SOD3-overexpressing. Additionally, SOD3 overexpression attenuated the elevation of MMP-2 and MMP-9, the pro-inflammatory and proliferative biomarkers. Overall, the SOD3 gene delivery exhibited potent anti-proliferation and anti-inflammation effects on TNF-α-induced VSMCs.</p><p><strong>Conclusion: </strong>An effective SOD3 gene delivery using a recombinant baculoviral vector has been successfully established and is useful for overexpression of the SOD gene family. This approach provides new therapeutic strategies in gene therapy against atherosclerosis.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142139560","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
Recent Advances in Therapeutics and Manufacturing Processes of Recombinant Adeno-Associated Virus for the Treatment of Lung Diseases. 用于治疗肺部疾病的重组腺相关病毒疗法和制造工艺的最新进展。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2024-09-02 DOI: 10.2174/0115665232294935240826061311
Luis Sorroza-Martinez, Mia Pelletier, David Guay, Bruno Gaillet
{"title":"Recent Advances in Therapeutics and Manufacturing Processes of Recombinant Adeno-Associated Virus for the Treatment of Lung Diseases.","authors":"Luis Sorroza-Martinez, Mia Pelletier, David Guay, Bruno Gaillet","doi":"10.2174/0115665232294935240826061311","DOIUrl":"https://doi.org/10.2174/0115665232294935240826061311","url":null,"abstract":"<p><p>Developing delivery vectors capable of transducing genetic material across the lung epithelia and mucus barrier is a major challenge and of great interest to enable gene therapies to treat pulmonary diseases. Recombinant Adeno-associated Viruses (rAAVs) have emerged as attractive candidates among viral and non-viral vectors due to their broad tissue tropism, ability to transduce dividing and quiescent cells, and their safety profile in current human applications. While rAAVs have demonstrated safety in earlier clinical trials for lung disease applications, there are still some limitations regarding rAAV-transgene delivery in pulmonary cells. Thus, further improvements in rAAV engineering are needed to enhance the effectiveness of rAAV-based therapies for lung diseases. Such therapies could benefit patients with chronic lung diseases, such as asthma, chronic obstructive pulmonary disease, pulmonary hypertension, and cystic fibrosis, among others, by regulating hereditary gene mutations or acquired gene deregulations causing these conditions. Alongside therapeutic development, advances in the rAAV production process are essential to meet increasing production demands, while reducing manufacturing costs. This review discusses current challenges and recent advances in the field of rAAV engineering and manufacturing to encourage the clinical development of new pulmonary gene therapy treatments.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142119189","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
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 : 2024-08-30 DOI: 10.2174/0115665232316708240828063527
Abhinay Dhiman, Sidharth Mehan, Zuber Khan, Aarti Tiwari, Ghanshyam Das Gupta, Acharan S 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 S Narula","doi":"10.2174/0115665232316708240828063527","DOIUrl":"https://doi.org/10.2174/0115665232316708240828063527","url":null,"abstract":"<p><p>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.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2024-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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