Current gene therapy最新文献

{"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":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods and materials: </strong>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.</p><p><strong>Results and discussion: </strong>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.</p><p><strong>Conclusion: </strong>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}

{"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}

{"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}

{"title":"Imaged Capillary Isoelectric Focusing (icIEF) Platform for Characterization of Charge Variants of Adeno-Associated Virus (AAV) Capsids and Impact on Their Transduction Efficiency.","authors":"Brandon Hoyle, Dhimiter Bello, Jonathan Hill, Soumita Das, Jonghan Kim","doi":"10.2174/0115665232369708250327081305","DOIUrl":"https://doi.org/10.2174/0115665232369708250327081305","url":null,"abstract":"<p><strong>Objective: </strong>Adeno-Associated Virus (AAV) vectors are comprised of a capsid protein encapsulating a Deoxyribonucleic Acid (DNA) transgene that has been used in the gene therapy field showing potential to treat a range of genetic diseases. Methods in the field of gene therapy should be optimized or enhanced to deepen understanding of AAVs, specifically around charge heterogeneity of capsid species.</p><p><strong>Methods: </strong>In this study, a versatile approach was presented for investigating the charge heterogeneity of Adeno-Associated Virus (AAV) capsid proteins of a variety of serotypes. This method employs Imaged Capillary Isoelectric Focusing (icIEF) coupled with native fluorescence imaging detection and has undergone exhaustive validation.</p><p><strong>Results: </strong>Demonstrating its platform nature, this method analyzed seven different AAV serotypes from multiple manufacturing platforms. The distinctive profiles generated for each AAV serotype serve as valuable indicators for both identity confirmation and stability assessment. It was shown that thermal stress and pH conditions play a role in increasing acidic charged variants over time, affecting the charge heterogeneity of AAVs, which can be serotype-specific. Reverse phase LC-MS was used to identify and confirm the increased presence of Post-Translational Modifications (PTMs) that are linked to increasing acidic species variants relative to non-stressed AAVs.</p><p><strong>Conclusion: </strong>These PTMs have biological consequences reflected in the diminished expression of the protein of interest in vitro. This cIEF method successfully analyzed a variety of AAV serotypes, and increasing trends of acidic variants led to reduced in vitro potency.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144062560","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}

{"title":"Next-Generation Whole-Exome Pattern: Advanced Methods and Clinical Significance.","authors":"Sumel Ashique, Anas Islam, Navjot Kaur Sandhu, Bhavinee Sharma, Rashmi Pathak, Himanshu Sharma","doi":"10.2174/0115665232356780250331181436","DOIUrl":"https://doi.org/10.2174/0115665232356780250331181436","url":null,"abstract":"<p><p>NGS (Next-generation sequencing) has emerged as the primary approach for gene finding in uncommon hereditary disorders. Targeted gene panels, whole genome sequencing (WGS), and whole exome sequencing (WES) are uses of next-generation sequencing and other related technologies. It is possible to explain personal or individual genome sequencing using NGS technology, as well as to detect disease-causing mutations using NGS findings. NGS, deep sequencing or massively parallel are similar words that describe a method of DNA sequencing leading to revolutionary change in genomic research. Due to its cost-effectiveness, Whole-Exome sequencing (WES) using Next-Generation Sequencing (NGS) is becoming increasingly popular in the field of human genetics. As a diagnostic tool, this technology can reduce the duration of the diagnostic process for several patients and has mostly made a significant contribution to the identification of new genes responsible for causing diseases. Considering the diverse range of phenotypic presentations of the diagnosis, NGS has the potential to uncover causative mutations, including de novo, new, and familial variants, related to epileptic syndromes and significantly enhance molecular diagnosis. The present study centres on the potential applications of next-generation exome sequencing in clinical diagnostics and the challenges encountered in the data processing of such data.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143966130","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}

{"title":"CRISPR/Cas9 Technology for Modifying Immune Checkpoint in CAR-T Cell Therapy for Hematopoietic Malignancies.","authors":"Forough Shams, Elham Sharif, Hajar Abbasi-Kenarsari, Nader Hashemi, Masoumeh Sadat Hosseini, Neda Heidari, Effat Noori, Ali Hossein Amini, Maryam Bazrgar, Maryam Rouhani, Yong Teng","doi":"10.2174/0115665232357078250331180413","DOIUrl":"https://doi.org/10.2174/0115665232357078250331180413","url":null,"abstract":"<p><p>Hematologic malignancies, which arise from dysregulation of hematopoiesis, are a group of cancers originating in cells with diminished capacity to differentiate into mature progeny and accumulating immature cells in blood-forming tissues such as lymph nodes and bone marrow. Immune- targeted therapies, such as Immune Checkpoint Blockade (ICB), chimeric antigen receptor T (CAR-T) cell therapy, and the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system, a precise, popular, and versatile genome engineering tool, have opened new avenues for the treatment of malignancies. Targeting immune checkpoints has revolutionized FDA approval in cytotoxic T-lymphocyte-associated protein 4 (CTLA-4), PD-1 (programmed death-1), and PDL1. According to the ICB and CAR techniques, the production of efficient CAR-T cells depends on the successful genetic modification of T cells, making them less susceptible to immune escape and suppression by cancer cells, which results in reduced off-target toxicity. Therefore, CRISPR/Cas9 has revolutionized the immune checkpoint-based approach for CAR-T cell therapy of hematologic malignancy. Continued research and clinical trials will undoubtedly pave the way for further advances in this field, ultimately benefiting patients and improving outcomes.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143970339","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}

{"title":"Transforming RNA-Based Gene Therapy with Innovative Nanocarriers for siRNA and miRNA Delivery.","authors":"Jitendra Singh Chaudhary, Dilip Kumar Chanchal, Kuldeep Singh, Jeetendra Kumar Gupta, Divya Jain, Anubhav Dubey, Mahima Pandey, Shahbaz Khan","doi":"10.2174/0115665232355294250330002630","DOIUrl":"https://doi.org/10.2174/0115665232355294250330002630","url":null,"abstract":"<p><p>The cells have been given precise instructions proprio to the regulation of gene expression by the main genesis of Ryan-based gene therapy, which has revived cancer treatment and other disorders. The difficulty of delivering small interfering RNA (siRNA) and microRNA (miRNA) to a target cell is an enormous task and is often faced by researchers due to characteristic instabilities of these carriers and their poor uptake by the cell membrane. The new developments from nanocarrier technologies offer opportunities for better effectiveness of RNA therapy for its delivery and the effectiveness of the treatment regimen. The objective of this article is to provide an overview of the existing as well as the newest developments in nanocarrier technology, particularly as related to microRNA and small interfering RNA (siRNA) delivery. Their modes of operation and their uses in gene therapies are also examined as principles of their design. We focus on several nanocarrier technologies, which have shown proof of concept in multiple disciplines such as stability, controlled release profiles, and delivery. Lipid-based nanoparticles, polymeric systems, and hybrid nanocarriers are some of the platforms that fall under this category; however, this list is not exhaustive. We also study the idea that certain nanocarriers could have multiple functionalities, which would make it possible to improve cancer treatment by simultaneously carrying chemotherapy and genes. We aim to shed light on the future of RNA-based gene therapy by providing a thorough overview of recent research in the field. This will help us understand how novel nanocarrier technologies can tackle the delivery issues.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143968682","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}

{"title":"Non-coding RNAs-based Therapy and Angiogenesis: A New Era for the Management of Gliomas.","authors":"Ozal Beylerli, Ilgiz Gareev, Elmar Musaev, Tatiana Ilyasova, Sergey Roumiantsev, Vladimir Chekhonin","doi":"10.2174/0115665232356703250325075309","DOIUrl":"https://doi.org/10.2174/0115665232356703250325075309","url":null,"abstract":"<p><p>The relentless pursuit of understanding and combating glioblastoma (GBM), one of the most formidable foes in the realm of cancer, requires a deeper exploration of its intricate dynamics. Gliomas, particularly GBM, are known for their lethal nature, and a significant aspect of their pathogenesis lies in their ability to manipulate the blood vessels that sustain them. This complex relationship is governed by a multitude of molecular mechanisms involving a diverse array of cell types within the tumor microenvironment. Central to this intricate web of regulation are non-coding RNAs (ncRNAs), enigmatic molecules that have recently emerged as key players in cancer biology. These ncRNAs wield a remarkable influence on gene expression, often via epigenetic modifications and intricate control over angiogenesis-related molecules. Their role in GBM angiogenesis adds another layer of complexity to our understanding of this disease. In the realm of cancer therapeutics, targeting angiogenesis has become a prominent strategy. However, the efficacy of current antiangiogenic treatments against GBM is often transient, as these tumors can rapidly develop resistance, becoming even more aggressive. GBM employs a diverse set of strategies to foster its abnormal vasculature, which, in turn, holds the key to understanding why anti-angiogenic therapies often fall short of expectations. This review aims to shed light on potential strategies and novel perspectives to overcome GBM 's resistance to anti-angiogenic therapy. By exploring innovative approaches, including those centered on ncRNAs, we strive to chart a course toward more effective treatments. This journey into the depths of GBM 's complexities offers not only hope but also a blueprint for future research and therapeutic development. As we uncover the intricate mechanisms at play, we inch closer to the day when GBM is no longer an insurmountable adversary in the fight against cancer.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143979117","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}

{"title":"Expression of miR-92a in Green Tea EGCG Preconditioned Adiposederived Stem Cells Ameliorates Inflammatory Response and Increases Cartilage Regeneration in RA Rats through KLF4/IL-17/MMP-2 Axis Modulation.","authors":"Tung-Sheng Chen, Wei-Wen Kuo, Chih-Yang Huang","doi":"10.2174/0115665232339721250313075146","DOIUrl":"https://doi.org/10.2174/0115665232339721250313075146","url":null,"abstract":"<p><strong>Background: </strong>The global prevalence of rheumatoid arthritis (RA) is on the rise. Numerous studies have demonstrated the potential of stem cell-based therapies in RA treatment. Experimental evidence suggests that preconditioning enhances the regenerative capabilities of stem cells compared to their unconditioned counterparts.</p><p><strong>Objective: </strong>This study aimed to evaluate whether adipose-derived stem cells (ADSCs) preconditioned with green tea epigallocatechin gallate (EGCG) and miR-92a exhibit superior therapeutic effects in RA compared to unconditioned ADSCs.</p><p><strong>Methods: </strong>Both in vitro and in vivo models were employed. In the cellular model, ADSCs were preconditioned with EGCG and miR-92a. In the animal model, male Wistar rats were used, and RA was induced using the collagen-induced arthritis (CIA) model. Following RA induction, the animals were divided into six groups: Sham (healthy rats), RA (RA-induced rats), RA+ADSC (RA-induced rats receiving unconditioned ADSCs), RA+E-ADSC (RA-induced rats receiving EGCGpreconditioned ADSCs), RA+mic-ADSC (RA-induced rats receiving miR-92a mimicpreconditioned ADSCs), and RA+inh-ADSC (RA-induced rats receiving miR-92a inhibitorpreconditioned ADSCs).</p><p><strong>Results: </strong>In the cellular model, preconditioning with EGCG and miR-92a activated the CXCR4/p- Akt signaling pathway, thereby enhancing ADSC viability. In the animal model, RA induction caused several joint pathologies, including hind paw swelling, disrupted bone metabolism, immune cell infiltration, increased expression of IL-17, KLF4, and IL-6, as well as cartilage degradation. While transplantation of unconditioned ADSCs modestly improved these pathological features, the administration of E-ADSCs and mic-ADSCs significantly ameliorated these conditions in RA rats. Conversely, the therapeutic effects of E-ADSCs and mic-ADSCs were attenuated by the transplantation of inh-ADSCs.</p><p><strong>Conclusion: </strong>The therapeutic effects of E-ADSCs and mic-ADSCs in RA were strongly associated with the modulation of the KLF4/IL-17/MMP-2 axis. These findings suggest that ADSCs preconditioned with EGCG and miR-92a hold considerable clinical promise for the treatment of RA.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143779365","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}

{"title":"Riding the Wave of Progress: Examining the Current Landscape and Future Potential of MicroRNAs in Cancer Gene Therapy.","authors":"Saleem Ahmad, Hanif Khan, Njoka Irene Muthoni, Mohammed Alissa, Awaji Y Safhi, Fahad Y Sabei, Osama Abdulaziz, Khadijah Hassan Khan, Essam H Ibrahim, Safir Ullah Khan","doi":"10.2174/0115665232353538250318075057","DOIUrl":"10.2174/0115665232353538250318075057","url":null,"abstract":"<p><p>MicroRNAs, commonly referred to as miRNAs, exert a significant impact on cellular processes by coordinating post-transcriptional gene regulation. These non-coding RNAs, which are only 22 nucleotides long, form a part of the RNA-induced silencing complex (RISC) and play a crucial role in regulating gene expression. Their complex participation in cell proliferation, differentiation, and death highlights their crucial role in maintaining cellular balance. MicroRNAs have become significant contributors in the complex field of cancer biology, operating beyond the usual tasks of cells. Their dysregulation is closely intertwined with cancer initiation and development. miRNAs act as cellular regulators and regulate complex processes of gene expression. Disruption of this regulation can result in tumor development. This review article explores the intricate process of miRNA biosynthesis and its mechanisms, providing insights into its complex interactions with cancer. It also discusses the exciting field of miRNA-based cancer treatment. Exploring the therapeutic possibilities of these small RNA molecules presents opportunities for precision medicine, introducing a new age where miRNAs can be utilized to create targeted therapeutic interventions that mainly address the abnormal genetic characteristics that cause tumor formation. miRNAs provide a harmonious balance between understanding their biology and utilizing their therapeutic potential in cancer treatment. However, they also serve as conductors and possible therapeutic instruments in the symphony of molecular biology for gene therapy.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143699894","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}