Current gene therapy最新文献

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Bridging Mind and Gut: The Molecular Mechanisms of microRNA, Microbiota, and Cytokine Interactions in Depression. 架起心灵和肠道的桥梁:抑郁症中microRNA、微生物群和细胞因子相互作用的分子机制。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-06-27 DOI: 10.2174/0115665232361169250617192348
Himanshu Sharma, Abdullah Al Noman, Iftakhar Ahmad, Susmita Deb Tonni, Tasmia Jahin Mim, Fahmida Afrose, Pranab Dev Sharma, Anwar Parvez, Samanta Tamanna, Md Al Azad, Rashmi Pathak
{"title":"Bridging Mind and Gut: The Molecular Mechanisms of microRNA, Microbiota, and Cytokine Interactions in Depression.","authors":"Himanshu Sharma, Abdullah Al Noman, Iftakhar Ahmad, Susmita Deb Tonni, Tasmia Jahin Mim, Fahmida Afrose, Pranab Dev Sharma, Anwar Parvez, Samanta Tamanna, Md Al Azad, Rashmi Pathak","doi":"10.2174/0115665232361169250617192348","DOIUrl":"https://doi.org/10.2174/0115665232361169250617192348","url":null,"abstract":"<p><p>Depression is a complex psychiatric disorder that arises from various underlying biological mechanisms. In this review, the role of microRNAs (miRNAs) in modulating gut microbiotacytokine communication and their potential to unravel the pathophysiology of depression and develop novel therapeutic strategies are discussed. MiRNAs are small non-coding RNA molecules that have emerged as key regulators in the bidirectional signaling of the gut-brain axis by modulating gene expression and fine-tuning an intricate dialogue between the microbiota, immune system, and central nervous system. Results show how gut microbiota can shape miRNA expression in brain regions involved in mood regulation; conversely, evidence is accumulating, elucidating how miRNA perturbations can shape microbial ecology. Gut bacteria-derived short-chain fatty acids (SCFAs) fuel this nexus by exerting effects on neurogenesis, neurotransmitter synthesis, neuroinflammation, affective behavior alterations, and depressive-like phenotypes. Pro-inflammatory cytokines such as IL-6, TNF-α, and IL-1β are also known to be associated with depressive symptoms related to altered expression patterns of specific miRNAs across these disorders. This review exposes the novel potential biomarkers and therapeutic targets/strategies to develop innovative methods in the diagnosis and treatment of depression by exploring bidirectional relations among miRNAs, gut microbiota, and cytokines. The knowledge of these molecular networks and pathways has provided the opportunity for designing new-generation therapeutics such as phytobiotics, probiotics, psychobiotics, diet therapies, and nanomedicine based on miRNAs from a future perspective, which will revolutionize the management of mental disorders.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144526739","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 Genetic and Epidemiological Dimensions of Gallbladder Cancer: Toward Effective Therapeutic Strategies. 胆囊癌的遗传和流行病学维度:走向有效的治疗策略。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-06-24 DOI: 10.2174/0115665232366089250610083533
Afrin Siddiqui, Somali Sanyal, Debalina Mukherjee, Medha Dwivedi, Manish Dwivedi
{"title":"The Genetic and Epidemiological Dimensions of Gallbladder Cancer: Toward Effective Therapeutic Strategies.","authors":"Afrin Siddiqui, Somali Sanyal, Debalina Mukherjee, Medha Dwivedi, Manish Dwivedi","doi":"10.2174/0115665232366089250610083533","DOIUrl":"https://doi.org/10.2174/0115665232366089250610083533","url":null,"abstract":"<p><p>Gallbladder Cancer (GBC) is a highly concerning malignancy, particularly prevalent in the Asian continent, attributed to irregularities in the bile tract. As of 2022, GLOBOCAN data ranks GBC as the 22nd most common cause of cancer-related mortality globally and the 6th among gastrointestinal cancers. According to recent World Cancer Research statistics, approximately 122,491 new cases of gallbladder cancer were reported by the end of 2022, ranking it 23rd among cancers in men and 20th in women worldwide. Towards the therapy of GBC, genetic studies have provided valuable insights into the molecular mechanisms driving GBC. Mutations in TP53, KRAS, ERBB2 (HER2), CDKN2A, and PIK3CA play crucial roles in tumor initiation and progression. Additionally, epigenetic modifications and aberrant signaling pathways, including Wnt/β-catenin, Notch, and PI3K/AKT/mTOR, have been implicated in GBC pathogenesis. Exploring these genetic alterations has led to targeted therapies, such as HER2 inhibitors (trastuzumab, pertuzumab) and immune checkpoint inhibitors, offering new treatment prospects. Further, current treatment approaches, including surgical resection, chemotherapy (gemcitabine-cisplatin), and radiation therapy, offer suboptimal outcomes in advanced stages of GBC. Despite its prevalence, effective therapeutic approaches and early-stage diagnostic methods remain elusive. This review provides a comprehensive overview of GBC, including its genetic mutations, epidemiology, risk factors, prevention, diagnosis, treatment options, and challenges. This work aims to offer valuable insights into the various factors directly or indirectly associated with GBC, which may assist in preparing an effective strategy against this growing malignancy.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144539348","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
Therapeutic Effects of Mesenchymal Stem Cells Carrying Echovirus in Mouse Models of Breast Cancer. 携带埃可病毒的间充质干细胞对小鼠乳腺癌模型的治疗作用。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-06-20 DOI: 10.2174/0115665232347036250610065753
Fatemeh Aminian, Nahid Babaei, Hadi Esmaeili Gouvarchin Ghaleh, Gholamreza Khamisipour, Mahdieh Farzanehpour
{"title":"Therapeutic Effects of Mesenchymal Stem Cells Carrying Echovirus in Mouse Models of Breast Cancer.","authors":"Fatemeh Aminian, Nahid Babaei, Hadi Esmaeili Gouvarchin Ghaleh, Gholamreza Khamisipour, Mahdieh Farzanehpour","doi":"10.2174/0115665232347036250610065753","DOIUrl":"https://doi.org/10.2174/0115665232347036250610065753","url":null,"abstract":"<p><strong>Introduction: </strong>Breast Cancer (BC) is treatable in early stages but has high mortality rates in advanced cases, highlighting the need for better treatment methods. Oncolytic Viruses (OVs) have emerged as a promising approach to specifically target and kill BC tumor cells, although their effectiveness is limited by the immune response. To overcome this challenge, researchers are investigating the use of cell carriers. This study aims to evaluate the effects of mesenchymal stem cells carrying Echovirus (MSCs-ECHO) in a BC mouse model.</p><p><strong>Method: </strong>The effectiveness of MSCs-ECHO was evaluated in a mouse model of BC induced by the subcutaneous injection of live 4T1 cells (1×104) in female Balb/c mice. Its effects were assessed using several parameters, including Tumor Size (TS), Survival Probability (SP), and indicators of immune system response, such as the Splenocyte Proliferation Index (SPI), Nitric Oxide (NO), Lactate Dehydrogenase (LDH), and cytokines (IL-4, IL-10, IFN-γ, and TGF-β) in the supernatant of splenocytes.</p><p><strong>Results: </strong>Our findings revealed that treatment with MSCs-ECHO significantly increased SP, SPI, LDH, NO, and IFN-γ levels, while reducing TS, TGF-β, IL-4, and IL-10 levels in treated mice compared to the control group. Additionally, MSCs-ECHO demonstrated superior therapeutic effects compared to treatment with cell-free virus.</p><p><strong>Conclusion: </strong>These findings indicate that ECHO treatment may represent a promising therapeutic approach for BC. Based on the results of the present study, the utilization of MSCs as carriers for OV appears to be a viable complementary strategy in the management of BC.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144505055","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
Novel Perspective of Regulating P53/Bcl2/Caspase-3 via In vitro Targeted AFP Gene Knocks Out in HepG2 Cells Using CRISPR/Cas9 Editing Tool. 利用CRISPR/Cas9编辑工具通过体外靶向敲除HepG2细胞中的AFP基因调控P53/Bcl2/Caspase-3的新视角
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-06-12 DOI: 10.2174/0115665232366303250529164610
Fatma Khorshed, Amina M Medhat, Germine M Hamdy, Ehab El-Dabaa, Hanaa Hammad, Heba A H Abd Elhameed, Mohamed Saber
{"title":"Novel Perspective of Regulating P53/Bcl2/Caspase-3 via In vitro Targeted AFP Gene Knocks Out in HepG2 Cells Using CRISPR/Cas9 Editing Tool.","authors":"Fatma Khorshed, Amina M Medhat, Germine M Hamdy, Ehab El-Dabaa, Hanaa Hammad, Heba A H Abd Elhameed, Mohamed Saber","doi":"10.2174/0115665232366303250529164610","DOIUrl":"https://doi.org/10.2174/0115665232366303250529164610","url":null,"abstract":"<p><strong>Introduction: </strong>Hepatocellular carcinoma (HCC) is a major health burden worldwide, with a persistent need for molecular target drugs. Alpha-fetoprotein (AFP) is a major concern during HCC, as it has an incompletely solved action. CRISPR/Cas9 is a gene editing tool that aids in cancer treatment research; thus, this study evaluated the effect of in vitro knockout of AFP on HCC using CRISPR/Cas9 technique.</p><p><strong>Methods: </strong>Two sgRNAs targeting specific sites in AFP exon 2 were separately cloned to the mammalian expression vector pSpCas9 (BB)-2a-GFP (PX458). HepG2 cells were transfected with CRISPR constructs I and II, and a pool of the two constructs (M) for 6 -, 24- and 39 hours using liopfectamine3000. AFP editing was evaluated regarding genomic DNA sequence, RNA, and protein expression levels. In addition, the effect of AFP knocking out on HepG2 viability, and apoptotic genes mRNA and protein expression levels were evaluated using crystal violet assay, real-time PCR, and western blot analysis respectively.</p><p><strong>Results: </strong>The results revealed efficient delivery of the AFP/CRISPR constructs to HepG2 cells. Insertion and deletion mutations introduced to the AFP genomic sequence were analyzed using TIDE software analysis and the Expasy translation tool. The viability of the HepG2 cells was reduced 39 hours post-transfection with significant modulation in the expression of the apoptotic markers P53, BAX, Bcl2, and caspase-3.</p><p><strong>Conclusion: </strong>This study succeeded in developing AFP/CRISPR constructs that could disrupt the AFP genomic sequence, reduce its expression, and restore the activity of cell-specific apoptotic factors, demonstrating the potential inhibitory effect of AFP downregulation on HCC progression.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144282791","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 Novel Biomarkers of Bacterial Lipopolysaccharides in Diabetic Nephropathy via Transcriptomics and Mendelian Randomization. 通过转录组学和孟德尔随机化鉴定糖尿病肾病细菌脂多糖的新生物标志物。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-06-03 DOI: 10.2174/0115665232353811250527053550
Yaxian Ning, Jianqin Wang, Xiaochun Zhou, Gouqin Wang, Lili Zhang
{"title":"Identification of Novel Biomarkers of Bacterial Lipopolysaccharides in Diabetic Nephropathy via Transcriptomics and Mendelian Randomization.","authors":"Yaxian Ning, Jianqin Wang, Xiaochun Zhou, Gouqin Wang, Lili Zhang","doi":"10.2174/0115665232353811250527053550","DOIUrl":"https://doi.org/10.2174/0115665232353811250527053550","url":null,"abstract":"<p><strong>Background: </strong>Dysbiosis of Intestinal Flora Lipopolysaccharides (LPS) is implicated in Diabetic Nephropathy (DN), yet the underlying mechanisms remain unclear. This study aims to elucidate the causal relationship between bacterial LPS and DN, with the goal of informing targeted therapeutic strategies.</p><p><strong>Methods: </strong>DN datasets GSE30528 and GSE96804 were analyzed. Bacterial LPS-related genes (LPS-RGs) were retrieved from the Gene Set Enrichment Analysis (GSEA) database. Differential expression analysis identified differentially expressed genes (DEGs), which were cross-referenced with LPS-RGs to derive DE-LPS-RGs. Mendelian randomization (MR) was applied to explore correlations between exposure factors and outcomes using GWAS data. miRNA-mRNA and TFmRNA regulatory networks were constructed using data from the TarBase and ENCODE databases, and potential therapeutic agents were identified through the DGIdb database.</p><p><strong>Results: </strong>Seven DE-LPS-RGs were identified, with CD14 and LY86 selected as biomarkers. GSEA and GeneMANIA analyses indicated that these genes participate in signal transduction and chargelike receptor signaling pathways. The regulatory networks demonstrated that LY86 interacts with miRNA hsa-mir-26a-5p, while TF ELK1 regulates both CD14 and LY86. Additionally, CD14 was associated with three potential drugs: VB-201, IC14, and Lovastatin.</p><p><strong>Conclusion: </strong>CD14 and LY86 represent promising biomarkers for DN, offering new perspectives for its prediction, diagnosis, and therapeutic intervention.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-06-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144559466","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
Targeting Extrachromosomal DNA (ecDNA) in Cancer: A New Era of CHK1 Inhibition and Personalized Treatments. 靶向肿瘤染色体外DNA (ecDNA): CHK1抑制和个性化治疗的新时代。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-05-27 DOI: 10.2174/0115665232390083250526072441
Komal, Lovekesh Singh, Subramanyam Sarma Ganti
{"title":"Targeting Extrachromosomal DNA (ecDNA) in Cancer: A New Era of CHK1 Inhibition and Personalized Treatments.","authors":"Komal, Lovekesh Singh, Subramanyam Sarma Ganti","doi":"10.2174/0115665232390083250526072441","DOIUrl":"https://doi.org/10.2174/0115665232390083250526072441","url":null,"abstract":"","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144186746","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
Next-Generation Nucleic Acid Delivery: A Review of Nanobiosystem Design and Applications. 新一代核酸传递:纳米生物系统设计与应用综述。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-05-27 DOI: 10.2174/0115665232367377250519114910
Ashish Kumar Parashar, Anu Hardenia, Sunil Kumar Dwivedi, Gaurav Kant Saraogi, Shiv Hardenia
{"title":"Next-Generation Nucleic Acid Delivery: A Review of Nanobiosystem Design and Applications.","authors":"Ashish Kumar Parashar, Anu Hardenia, Sunil Kumar Dwivedi, Gaurav Kant Saraogi, Shiv Hardenia","doi":"10.2174/0115665232367377250519114910","DOIUrl":"https://doi.org/10.2174/0115665232367377250519114910","url":null,"abstract":"<p><p>The increasing approval of nucleic acid therapeutics has led to a significant advancement in medicines, demonstrating their potential to revolutionize the prevention and treatment of numerous diseases. However, challenges like nuclease degradation and difficult cellular delivery hinder their use as therapeutic agents. The rising demand for precise gene therapy delivery has positioned nanobiosystems as a groundbreaking solution, with their customizable properties enabling targeted and efficient delivery. Nucleic Acid therapeutics, encompassing antisense DNA, mRNA, small interfering RNA (siRNA), and microRNA (miRNA), have been rigorously investigated for their capacity to modulate gene expression. Notably, integrating these gene therapies with nanoscale delivery platforms has significantly broadened their scope, facilitating sophisticated advancements in bioanalysis, gene silencing, protein replacement therapies, and the development of vaccines. This review provides a thorough review of recent advancements in nanobiosystems for therapeutic nucleic acid delivery. We explore the unique characteristics of various nanobiosystems, including gene therapy-based delivery, nanoparticles, stimuli-responsive systems, smart nanocarriers, and extracellular vesicle-based delivery. We offer a detailed overview of their applications in nucleic acid delivery. Furthermore, we address biological barriers and strategies for the therapeutic delivery of nucleic acids. Ultimately, this review provides critical insights into the strategic development of nextgeneration delivery vectors for nucleic acid therapeutics.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144186745","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
Elucidating the Predominant Role of AEBP1 in Different Types of Cancers with a Focus on Glioblastoma Progression - A Review. 阐明AEBP1在胶质母细胞瘤进展中不同类型癌症中的主导作用-综述
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-05-14 DOI: 10.2174/0115665232365878250503091118
Rangaraj Kaviyaprabha, Sridhar Muthusami, Thandaserry Vasudevan Miji, Palanisamy Arulselvan, Muruganantham Bharathi
{"title":"Elucidating the Predominant Role of AEBP1 in Different Types of Cancers with a Focus on Glioblastoma Progression - A Review.","authors":"Rangaraj Kaviyaprabha, Sridhar Muthusami, Thandaserry Vasudevan Miji, Palanisamy Arulselvan, Muruganantham Bharathi","doi":"10.2174/0115665232365878250503091118","DOIUrl":"https://doi.org/10.2174/0115665232365878250503091118","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Introduction: &lt;/strong&gt;Glioblastoma multiforme (GBM) is a highly deleterious lesion with an increased recurrence rate even after radiotherapy and chemotherapy. In this context, additional biomarkers are needed to curb chemoresistance. Computational approaches help us process the RNA-seq and identify the Differentially Expressed Genes (DEGs) in tumors and adjacent normal regions to identify the diagnostic and therapeutic biomarkers.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods and materials: &lt;/strong&gt;In this study, we extensively reviewed the role of AEBP1 in different types of cancer, highlighting its significance as a novel target to prevent collagen deposition. Specifically, the underlying mechanisms of AEBP1 in Glioblastoma were analyzed extensively using computational approaches that include Gene Expression Omnibus (GEO), GEPIA to obtain the TCGA-GBM dataset, and Glioma-BioDP to identify the survival rate in the context of AEBP1 expression associated with patients' age. Meanwhile, Tumor Immune Single-cell Hub 2 was implemented to identify the expression of AEBP1 in immunologically lineaged, cancerous, and stromal cells. In addition to that, the miRNA regulation associated with the AEBP1 expression was predicted by implementing NetworkAnalyst, TarBase v8.0, and CancerMIRNome. We identified the DEGs by examining the GSE121723, GSE184643, and GSE14824 datasets with P-values ≤ 0.05 as statistically significant. Furthermore, we predicted and analyzed the highly expressed genes and identified the survival rate, which significantly stated that the overexpression of AEBP1 was associated with decreased survival rates in GBM patients. The Protein-Protein Interaction network was constructed to identify the correlated gene expression.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results and discussion: &lt;/strong&gt;We identified 3695, 37001, and 8855 significantly differentially expressed genes (DEGs). The DEGs were filtered by applying a log2 fold-change cut-off of ≥2.0. Finally, 139 common genes were mapped with the identified DEGs (1338 genes) and SDEs (500 genes) estimated from the TCGA-GBM dataset. The analysis revealed that 155 genes are commonly upregulated, and survival analyses were performed that described the AEBP1 significantly reduced the GBM patients' survival rate among other genes. The constructed PPI network and correlated expression analysis associated with the AEBP1 expression revealed that COL6A2 and THBS2 might play a significant role in the GBM stage advancements by depositing collagens in the matrix environment. Also, the miRNA analysis revealed that the hsa-miR-128-3p and hsa-miR-512-3p could be targeted as a miRNA marker gene to prevent the GBM progression associated with the AEBP1 expression.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;AEBP1 is a multi-cancer drug target, underscoring its diagnostic and prognostic value in different types of cancer preventive medicine. It influences tumor growth, metastasis, and immune evasion in cancers like adrenocortical, oral, breast, bladder, ga","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144076483","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
Advances in Gene Therapy for X-Linked Myotubular Myopathy: Current Progress and Future Challenges. 基因治疗x连锁肌小管肌病的进展:当前进展和未来挑战。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-05-06 DOI: 10.2174/0115665232388661250430062849
Md Sadique Hussain, Gyas Khan, Gaurav Gupta
{"title":"Advances in Gene Therapy for X-Linked Myotubular Myopathy: Current Progress and Future Challenges.","authors":"Md Sadique Hussain, Gyas Khan, Gaurav Gupta","doi":"10.2174/0115665232388661250430062849","DOIUrl":"https://doi.org/10.2174/0115665232388661250430062849","url":null,"abstract":"","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143972631","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 and Validation of Amino Acid Metabolism-Related Biomarkers and Investigation of their Potential Mechanisms in Lung Adenocarcinoma. 肺腺癌中氨基酸代谢相关生物标志物的鉴定和验证及其潜在机制的研究。
IF 3.8 4区 医学
Current gene therapy Pub Date : 2025-04-18 DOI: 10.2174/0115665232364091250203090710
Xu Zhu, Ying Zhang, Peiying Pan, Xinlei Liu, Jian Zhang, Xiaojun Du, Tao Wang, Yin Teng, Chao Fan, Jianglun Li, Jieheng Wu, Zhu Zeng, Siyuan Yang
{"title":"Identification and Validation of Amino Acid Metabolism-Related Biomarkers and Investigation of their Potential Mechanisms in Lung Adenocarcinoma.","authors":"Xu Zhu, Ying Zhang, Peiying Pan, Xinlei Liu, Jian Zhang, Xiaojun Du, Tao Wang, Yin Teng, Chao Fan, Jianglun Li, Jieheng Wu, Zhu Zeng, Siyuan Yang","doi":"10.2174/0115665232364091250203090710","DOIUrl":"https://doi.org/10.2174/0115665232364091250203090710","url":null,"abstract":"<p><strong>Background: </strong>In lung adenocarcinoma (LUAD), the metabolism of amino acids (AAs) plays a crucial role in the growth, infiltration, and metastasis of tumor cells. Nevertheless, the potential of AA metabolism-associated genes (AAMRGs) to serve as prognostic indicators in LUAD remains ambiguous. Thus, this study sought to evaluate the prognostic value of AAMRGs in LUAD patients.</p><p><strong>Methods: </strong>Herein, we extracted LUAD transcriptomic information from two key repositories, namely The Cancer Genome Atlas Program (TCGA) and Gene Expression Omnibus. The non-negative matrix factorization (NMF) clustering technique was used to categorize the LUAD cases based on their AAM profiles before assessing the survival rates and composition of immune cells. Using limma software, shared dysregulated transcripts were identified across subgroups before functional annotation via DAVID, which comprised exploration of gene ontology and the Kyoto Encyclopedia of Genes and Genomes pathway. The prognostic framework was developed using five prognostic indicators through TCGA-derived LUAD specimens. We performed the analysis using singlevariable Cox, least absolute shrinkage and selection operator regression, and multi-factorial Cox regression. Molecular pathways between cohorts were compared with gene set enrichment analysis (GSEA). Real-time quantitative polymerase chain reaction (RT-qPCR) and immunohistochemical (IHC) analysis were utilized to validate the key genetic components of the model.</p><p><strong>Results: </strong>NMF clustering analysis was performed to categorize 497 LUAD patients into three distinct subgroups with obvious variations in the survival rates. The subtypes exhibited substantial disparities in immune cell populations, particularly in monocytes and mast cells. Analysis of 176 shared differentially expressed genes (DEGs) revealed enrichment in T lymphocyte stimulation, immunological reactions, and extra immune-related processes within the subgroups. The prognostic framework was constructed using biomarkers, such as ERO1LB, HPGDS, LOXL2, TMPRSS11E, and SLC34A2. Moreover, GSEA demonstrated a correlation between elevated risk and cell cycle processes, but lower risk was linked with arachidonic acid metabolic pathways. Analysis of 1128 DEGs revealed enrichment in various physiological processes, including cellular division, p53 signaling cascades, immunological responses, and additional pathways upon the comparison of high and low-risk cohorts. The RT-qPCR analysis confirmed elevated expression levels of ERO1LB and TMPRSS11E in LUAD specimens. Consistent with RT-qPCR analysis, the IHC results affirmed that the expression levels of ERO1LB and TMPRSS11E were increased in LUAD specimens.</p><p><strong>Conclusion: </strong>The five identified AAMRGs in LUAD were validated and appropriately utilized to construct a risk assessment model that could potentially act as prognostic biomarkers for LUAD patients.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143983958","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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