Nucleosides, Nucleotides & Nucleic Acids最新文献

Investigating the interactions of Axitinib, a tyrosine kinase inhibitor, with DNA: experimental studies, molecular docking, and molecular dynamics simulations. 研究酪氨酸激酶抑制剂阿西替尼与DNA的相互作用：实验研究、分子对接和分子动力学模拟。

IF 1.1 4区生物学

Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-06-02 DOI: 10.1080/15257770.2025.2509977

Pelin Şenel, Abdullah Al Faysal, Soykan Agar, Mine Yurtsever, Ayşegül Gölcü

{"title":"Investigating the interactions of Axitinib, a tyrosine kinase inhibitor, with DNA: experimental studies, molecular docking, and molecular dynamics simulations.","authors":"Pelin Şenel, Abdullah Al Faysal, Soykan Agar, Mine Yurtsever, Ayşegül Gölcü","doi":"10.1080/15257770.2025.2509977","DOIUrl":"https://doi.org/10.1080/15257770.2025.2509977","url":null,"abstract":"Axitinib is an oral medication classified as a second-generation tyrosine kinase inhibitor. It serves as a primary treatment for metastatic renal cell carcinoma (RCC) due to its strong affinity for DNA, which leads to the disruption of the double helix structure. This disruption ultimately halts the cell cycle and induces senescence and mitotic catastrophe in RCC cells. Consequently, investigating the mechanism by which Axitinib binds to DNA is essential for comprehending its pharmacodynamic properties and for the advancement of more effective DNA-binding therapeutics. The present study aimed to examine the interaction between Axitinib and DNA through various analytical techniques, including UV-Vis spectroscopy, thermal denaturation assays, electrochemical methods, and fluorescence emission spectroscopy. According to the electrochemical studies, the binding constant (Kb) for Axitinib was calculated to be (5.13 ± 0.28) × 104, suggesting the potential for groove binding. This finding was further supported by in-silico analyses, where molecular docking and molecular dynamics simulations indicated that the drug selectively binds to the DNA minor groove through partial intercalation, forming new hydrogen bonds with its functional groups while separating the guanine and cytosine base pairs.","PeriodicalId":19343,"journal":{"name":"Nucleosides, Nucleotides & Nucleic Acids","volume":" ","pages":"1-24"},"PeriodicalIF":1.1,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144209051","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Biophysical and electrochemical studies on the interaction of arbutin drug with calf-thymus DNA. 熊果苷类药物与小牛胸腺DNA相互作用的生物物理和电化学研究。

IF 1.1 4区生物学

Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-06-02 DOI: 10.1080/15257770.2025.2512857

D S Bhuvaneshwari, Kandasamy Pavithra, Kuppanagounder P Elango

{"title":"Biophysical and electrochemical studies on the interaction of arbutin drug with calf-thymus DNA.","authors":"D S Bhuvaneshwari, Kandasamy Pavithra, Kuppanagounder P Elango","doi":"10.1080/15257770.2025.2512857","DOIUrl":"https://doi.org/10.1080/15257770.2025.2512857","url":null,"abstract":"Understanding the interaction of therapeutic drugs with DNA is crucial for designing highly selective DNA-targeted medicines that could overcome the current therapeutic limitations. In this endeavour, the DNA binding behaviour of arbutin (ATN) was explored using multi-spectroscopic, electrochemical and computational studies. The UV-Vis spectral studies authenticated the complexation of ATN with CT-DNA and exposed ATN as a moderately strong DNA binder with a binding constant of 8.029 × 103 M-1. The findings of fluorescence spectral studies not only revealed the spontaneous ground state complex formation between ATN and CT-DNA, but also emphasised the role of hydrogen bonding and Van der Waals interactions in stabilising the ATN/CT-DNA complex. Since the competitive dye displacement assay strongly excluded the plausibility of classical intercalation and conventional groove binding mode of ATN, viscosity studies provided clues regarding the external binding mode of ATN. The appreciable enhancement resulted in the fluorescence emission of the ATN/CT-DNA complex upon increasing NaCl concentration, which certified ATN as an external binder. The CD spectral results exposed the ATN-induced moderate conformational alterations in CT-DNA. Remarkably, the voltammetric titration results labelled the glucopyranoside moiety of ATN as a DNA binding unit with a formation constant of 2.57 × 104 M-1 rather than the hydroquinone moiety of ATN. Molecular docking and metadynamics simulation outcomes served as pictorial evidence of experimental results. They revealed the predominant contribution of hydrogen bonding interactions in stabilising ATN/DNA complexation.","PeriodicalId":19343,"journal":{"name":"Nucleosides, Nucleotides & Nucleic Acids","volume":" ","pages":"1-23"},"PeriodicalIF":1.1,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144199749","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

MiR-34a rs2666433 and cognitive function in major depressive disorder: a clinical correlation analysis. MiR-34a rs2666433与重度抑郁症认知功能的临床相关性分析

IF 1.1 4区生物学

Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-06-02 DOI: 10.1080/15257770.2025.2511106

Ning Li, Xiaochuan Zhao

{"title":"MiR-34a rs2666433 and cognitive function in major depressive disorder: a clinical correlation analysis.","authors":"Ning Li, Xiaochuan Zhao","doi":"10.1080/15257770.2025.2511106","DOIUrl":"https://doi.org/10.1080/15257770.2025.2511106","url":null,"abstract":"Background: Genetic factors play a crucial role in the development of major depressive disorder (MDD).Objectives: This study aimed to investigate the association between the microRNA (miR)-34a rs266643 polymorphism and MDD, as well as its impact on cognitive function.Materials and methods: Clinical data and blood samples were collected from 302 MDD patients and 306 healthy controls who met the predefined inclusion and exclusion criteria. The severity of MDD in patients was assessed using the Hamilton Rating Scale for Depression (HRSD). Cognitive function in MDD patients was evaluated using the Mini-Mental State Examination (MMSE), Stroop Color-Word Test (Stroop-C and Stroop-CW), and Montreal Cognitive Assessment (MoCA). Gene typing was performed using the Sanger sequencing method, while the relative expression level of miR-34a was quantified by RT-qPCR.Results: The MDD group exhibited a significantly higher miR-34a expression fold change compared to the control group (p < 0.001). Among the genotypes, the AA genotype demonstrated the highest expression, followed by GA, with both being significantly greater than GG. The expression of miR-34a was positively correlated with HRSD, Stroop-C, and Stroop-CW scores but negatively correlated with MMSE and MoCA scores (p < 0.001). Carrying the A allele (OR = 1.468, p = 0.002) or the AA genotype (OR = 2.382, p = 0.001) was associated with an increased risk of MDD. Furthermore, patients with the AA genotype exhibited significantly poorer cognitive function compared to other genotypes.Conclusion: The gene polymorphism of miR-34a rs2666433 was significantly associated with the severity of MDD as well as cognitive function.","PeriodicalId":19343,"journal":{"name":"Nucleosides, Nucleotides & Nucleic Acids","volume":" ","pages":"1-14"},"PeriodicalIF":1.1,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144209052","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Investigation of the expression levels of MEFV gene in patients with frequent MEFV pathogenic variants in Kahramanmaras (Turkey). 土耳其Kahramanmaras地区MEFV致病性变异体患者MEFV基因表达水平的研究

IF 1.1 4区生物学

Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-05-28 DOI: 10.1080/15257770.2025.2511104

Eda Ganiyusufoglu, Hasan Daglı, Metin Kılınc

{"title":"Investigation of the expression levels of MEFV gene in patients with frequent MEFV pathogenic variants in Kahramanmaras (Turkey).","authors":"Eda Ganiyusufoglu, Hasan Daglı, Metin Kılınc","doi":"10.1080/15257770.2025.2511104","DOIUrl":"https://doi.org/10.1080/15257770.2025.2511104","url":null,"abstract":"The main objective of this study is to detect the variants in patients who were diagnosed with familial Mediterranean fever (FMF) according to Tel-Hashomer diagnostic criteria and investigated the relationship between genotype-phenotype and the gene expression levels of the Mediterranean fever (MEFV) gene. Variant screening was achieved by automated sanger sequencing, and expression levels of the MEFV gene were analyzed by quantitative real time polymerase chain reaction (RT-PCR). A total of 46 patients with MEFV gene pathogenic variants and 8 control individuals without any variants were enrolled in the study. The most commonly encountered variants in heterozygote genotype were M694V (n = 4), E148Q (n = 3), and M680I (n = 2); in compound heterozygote genotype were M694V/R202Q (n = 4), and R202Q/E148Q (n = 3); in complex heterozygote genotype were R202Q/M694V/M680I (n = 4) and R202Q/M694V/V726A (n = 3); in homozygote genotype were M680I/M680I (n = 7) and M694V/M694V (n = 4). The gene expression levels of the patients with homozygous variants were found to be significantly lower than the healthy control group and patients with heterozygous variants. In patients with M694V homozygous variants, where clinical manifestations are severe, a remarkable decrease in the gene expression of the MEFV gene was observed. It was detected that there was a relationship between the genotype and gene expression level and that the level of gene expression and clinical symptoms were inversely correlated in patients with FMF.","PeriodicalId":19343,"journal":{"name":"Nucleosides, Nucleotides & Nucleic Acids","volume":" ","pages":"1-14"},"PeriodicalIF":1.1,"publicationDate":"2025-05-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144160676","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

miR-508-5p regulates macrophage polarization via targeting TSGA10 to promote malignant behavior in esophageal cancer cells. miR-508-5p通过靶向TSGA10调控巨噬细胞极化，促进食管癌细胞的恶性行为。

IF 1.1 4区生物学

Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-05-11 DOI: 10.1080/15257770.2025.2491561

Yuan Zhu, Zuojun Fu, Tianjiao Duan, Jing Wang, Lingjuan Zhang, Guisheng Liu, Xueyan Guo, Rong Zhang

{"title":"miR-508-5p regulates macrophage polarization via targeting TSGA10 to promote malignant behavior in esophageal cancer cells.","authors":"Yuan Zhu, Zuojun Fu, Tianjiao Duan, Jing Wang, Lingjuan Zhang, Guisheng Liu, Xueyan Guo, Rong Zhang","doi":"10.1080/15257770.2025.2491561","DOIUrl":"https://doi.org/10.1080/15257770.2025.2491561","url":null,"abstract":"Background: Esophageal cancer (EC) is among the deadliest malignancies in humans, with various miRNAs shown to regulate its progression by targeting distinct genes. miR-508-5p was identified as being linked to the malignant behavior of various tumors. Nevertheless, the precise role and mechanism of miR-508-5p in esophageal cancer (EC) remain ambiguous.Objective: This investigation focuses on the role and mechanism of the miR-508-5p/TSGA10 axis in the progression of EC.Methods: The expression of miR-508-5p and TSGA10 in EC cell lines was evaluated using quantitative Real-Time Polymerase Chain Reaction (qRT-PCR). Cell transfection techniques were used to knock down miR-508-5p and observe its effects on cell proliferation, migration, invasion, and apoptosis. A dual-luciferase reporter gene assay was conducted to verify the targeting relationship of miR-508-5p with TSGA10. Co-culture studies were undertaken to examine the regulatory effect of the miR-508-5p/TSGA10 axis on the polarization state of tumor-associated macrophages (TAMs) and the malignant behavior of EC cells.Results: The expression of miR-508-5p was significantly elevated in EC cells. Knocking down miR-508-5p curbed cell proliferation, migration, and invasion while promoting apoptosis. TSGA10 was validated as a primary target gene of miR-508-5p. miR-508-5p knockdown could inhibit the M2 polarization of TAMs by upregulating TSGA10, thereby suppressing the tumorigenic behavior of EC cells.Conclusion: miR-508-5p promotes the M2 polarization of TAMs and enhances the malignant behavior of EC cells by inhibiting TSGA10.","PeriodicalId":19343,"journal":{"name":"Nucleosides, Nucleotides & Nucleic Acids","volume":" ","pages":"1-15"},"PeriodicalIF":1.1,"publicationDate":"2025-05-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144012717","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Binding characterization of small protein-conjugated ssDNA aptamer to recombinant human ICAM-1. 小蛋白偶联的ssDNA适体与重组人ICAM-1的结合特性。

IF 1.1 4区生物学

Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-05-07 DOI: 10.1080/15257770.2025.2500049

Nik Abdul Aziz Nik Kamarudin, Nurfadhlina Musa, Nur Fatihah Mohd Zaidi, Basyirah Ghazali, Mariana Ahamad, Satvinder S Dhaliwal, Khairul Mohd Fadzli Mustaffa

{"title":"Binding characterization of small protein-conjugated ssDNA aptamer to recombinant human ICAM-1.","authors":"Nik Abdul Aziz Nik Kamarudin, Nurfadhlina Musa, Nur Fatihah Mohd Zaidi, Basyirah Ghazali, Mariana Ahamad, Satvinder S Dhaliwal, Khairul Mohd Fadzli Mustaffa","doi":"10.1080/15257770.2025.2500049","DOIUrl":"10.1080/15257770.2025.2500049","url":null,"abstract":"This study investigates the potential of a protein-DNA aptamer conjugate to enhance aptamer binding to recombinant human intercellular adhesion molecule 1 (rhICAM-1). Aptamers are single-stranded nucleic acids that bind target molecules through hydrogen bonding and hydrophobic interactions. Conjugating aptamers with antibodies or proteins has been shown to improve their binding affinity. Using Systematic Evolution of Ligands by Exponential Enrichment (SELEX), eight rounds of selection were performed with ICAM-1-coupled Dynabeads Protein A, identifying a DI05 as having the strongest binding affinity to rhICAM-1. An antibody inhibition assay showed a significant reduction in rhICAM-1 binding to immobilized aptamers (DI05, DI20, DI31, and DI33). Additionally, the binding affinity of eGFP-conjugated DI05 to rhICAM-1 was higher than that of unconjugated DI05. Docking simulations revealed close contact between DI05 and ICAM-1, with interactions primarily mediated by hydrogen bonds within three hairpin structures at ≤2.8 Å. These findings highlight the potential of aptamer-small protein conjugates as a promising strategy to enhance aptamer binding characteristics.","PeriodicalId":19343,"journal":{"name":"Nucleosides, Nucleotides & Nucleic Acids","volume":" ","pages":"1-23"},"PeriodicalIF":1.1,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144029611","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The magic bullet: a tribute to Fritz Eckstein. 神奇子弹：向弗里茨·埃克斯坦致敬。

IF 1.1 4区生物学

Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-05-06 DOI: 10.1080/15257770.2025.2500048

Erik De Clercq

引用次数: 0

Bacteriophage-based gene delivery: a novel approach for targeted breast cancer therapy. 基于噬菌体的基因传递：靶向乳腺癌治疗的新方法。

IF 1.1 4区生物学

Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-05-05 DOI: 10.1080/15257770.2025.2500042

Dilpreet Singh

{"title":"Bacteriophage-based gene delivery: a novel approach for targeted breast cancer therapy.","authors":"Dilpreet Singh","doi":"10.1080/15257770.2025.2500042","DOIUrl":"https://doi.org/10.1080/15257770.2025.2500042","url":null,"abstract":"Bacteriophage-based gene delivery systems are emerging as a promising alternative to traditional viral and non-viral vectors for targeted gene therapy in breast cancer. Their unique structural adaptability, low immunogenicity, and cost-effective production make them ideal candidates for precision medicine applications. Unlike conventional gene delivery platforms, bioengineered bacteriophages can be functionalized with tumor-specific ligands, modified for PEGylation to enhance circulation stability, and integrated with CRISPR/Cas9 gene-editing systems for precise genomic modifications. Additionally, bacteriophage vectors can be utilized in combination therapy, amplifying the effectiveness of chemotherapy and immunotherapy in breast cancer treatment. This mini-review discusses the bioengineering strategies used to enhance bacteriophage-based gene delivery, including surface modifications for tumor targeting, ligand-receptor binding for cellular uptake, and controlled genetic cargo release. We further examine in vitro and in vivo studies that demonstrate the potential of bacteriophage vectors in tumor suppression, gene expression efficiency, and immunomodulation. Furthermore, we explore the challenges and future directions of integrating bacteriophage-mediated gene therapy into clinical applications, addressing key issues such as systemic circulation half-life, off-target effects, and immune system interactions.","PeriodicalId":19343,"journal":{"name":"Nucleosides, Nucleotides & Nucleic Acids","volume":" ","pages":"1-19"},"PeriodicalIF":1.1,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144032570","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

LncRNA PGM5-AS1 inhibits the progression of breast cancer by inhibiting miR-182-5p. LncRNA PGM5-AS1通过抑制miR-182-5p抑制乳腺癌的进展。

IF 1.1 4区生物学

Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-04-29 DOI: 10.1080/15257770.2025.2498642

Yonghui Zhang, Mingxi Chen, Xuan Zheng, Kejia Li, Zhi Li, Xuelian Li

{"title":"LncRNA PGM5-AS1 inhibits the progression of breast cancer by inhibiting miR-182-5p.","authors":"Yonghui Zhang, Mingxi Chen, Xuan Zheng, Kejia Li, Zhi Li, Xuelian Li","doi":"10.1080/15257770.2025.2498642","DOIUrl":"https://doi.org/10.1080/15257770.2025.2498642","url":null,"abstract":"LncRNAs serve as crucial regulators in the survival and proliferation of tumors. This study is dedicated to exploring the functional significance of lncRNA PGM5-AS1 in breast cancer (BRCA). First, the expression level of PGM5-AS1 in BRCA patients and its diagnostic ability for BRCA were analyzed by RT-qPCR and Receiver Operating Characteristic curve. Subsequently, LnCAR database was used to preliminarily explore the relationship between PGM5-AS1 and prognosis. Moreover, we investigated the effects PGM5-AS1 on proliferation, apoptosis, and migration of BRCA cells by MTT assay, flow cytometry, and Transwell assay. More importantly, the regulation effect of PGM5-AS1 on the downstream target miR-182-5p was verified by dual luciferase reporting experiment, and the role of miR-182-5p was further explored in vitro experiments. PGM5-AS1 is significantly decreased in both BRCA patients and BRCA cell lines. In the diagnosis of BRCA, the sensitivity and specificity of PGM5-AS1 were 81.5% and 78.5%. Furthermore, lower levels of PGM5-AS1 are associated with a poor prognosis for affected patients. In vitro studies demonstrate that the upregulation of PGM5-AS1 confers a protective effect against BRCA, markedly inhibiting the viability and migratory capacity of tumor cells. More importantly, overexpression of PGM5-AS1 inhibited the high expression of miR-182-5p in tumor cells. In fact, inhibition of miR-182-5p is detrimental to the proliferation and migration of BRCA cells in vitro. lncRNA PGM5-AS1 has potential as a diagnostic marker for BRCA and acts as an inhibitor in BRCA. It inhibits tumor proliferation and metastasis by targeting miR-182-5p.","PeriodicalId":19343,"journal":{"name":"Nucleosides, Nucleotides & Nucleic Acids","volume":" ","pages":"1-14"},"PeriodicalIF":1.1,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144013236","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Gene therapy and gene therapy products introduced to market by 2022. 基因治疗和基因治疗产品将于2022年上市。

IF 1.1 4区生物学

Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-04-10 DOI: 10.1080/15257770.2025.2489495

Cengiz Bereket, Imge Kunter, Elaheh Ashrafian Bonab, Ghazal Footohi

{"title":"Gene therapy and gene therapy products introduced to market by 2022.","authors":"Cengiz Bereket, Imge Kunter, Elaheh Ashrafian Bonab, Ghazal Footohi","doi":"10.1080/15257770.2025.2489495","DOIUrl":"https://doi.org/10.1080/15257770.2025.2489495","url":null,"abstract":"Gene therapy has revolutionized the concept of treating genetic disorders by addressing the root causes at the genetic level, becoming one of the most quickly evolving fields in medicine today, especially due to its long-term effects. Gene therapy for the treatment of diseases relies on strategies of gene suppression, overexpression, and editing using different tools such as CRISPR and RNA interference. The gene transfer methods are broadly classified into three categories: physical, chemical, and biological. The use of viral vectors, such as adenoviruses, retroviruses, and adeno-associated viruses, is prevalent in clinical settings due to their high efficiency. Safety remains as an issue, and risk mitigation strategies will continue to evolve from clinical data to minimize complications related to gene silencing and immunotoxicity. In this review, various aspects of gene therapy have been covered, such as in-vivo and ex-vivo gene therapy, gene transfer methods, safety issues, as well as the gene therapy products approved until 2022. This review lists 35 licensed gene therapy products, detailing their therapeutic uses, mechanism of action, and vectors employed. Each product illustrates the various applications and potentials of gene therapy against untreatable conditions. Continuous improvements in gene transfer methods, vector safety, and clinical applications will increase the impact of the technology and offer hope for effective treatment and possible cures for different genetic disorders.","PeriodicalId":19343,"journal":{"name":"Nucleosides, Nucleotides & Nucleic Acids","volume":" ","pages":"1-39"},"PeriodicalIF":1.1,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143991797","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0