Current gene therapyPub Date : 2026-01-01DOI: 10.2174/0115665232342211250207064205
M Suhas, Sunil Kumar Kadiri
{"title":"Parkinson's Disease: From Bench to Bedside-Advancements in Diagnosis and Therapeutics using Pharmacogenomic Approach.","authors":"M Suhas, Sunil Kumar Kadiri","doi":"10.2174/0115665232342211250207064205","DOIUrl":"10.2174/0115665232342211250207064205","url":null,"abstract":"<p><p>This study provides a detailed overview of Parkinson's disease (PD), a neurodegenerative ailment mostly known for movement difficulties such tremor, stiffness, and bradykinesia, which affects approximately 1% of persons over the age of 60. Although the precise cause of PD is still unknown, various factors such as pesticide exposure, genetics, and lifestyle choices like smoking and caffeine consumption are thought to play a role in its development. The presence of Lewy bodies characterizes the disease, the aggregation of alpha-synuclein, the loss of dopaminergic neurons in the substantia nigra, and disruptions in basal ganglia circuitry, resulting in both motor and nonmotor symptoms. This review is structured into several key sections, beginning with an exploration of the pathophysiological mechanisms behind PD, including how genetic mutations can lead to deficits in the Ubiquitin Proteasome System and mitochondrial function, which are linked to familial cases of the disease. Following this, the review explores diagnostic methods, such as the UK Brain Bank Criteria, advanced imaging techniques, olfactory testing, and innovative technologies like machine learning, all of which support early detection and accurate diagnosis of PD. Treatment strategies are also comprehensively reviewed, focusing on traditional pharmacological options like levodopa and dopamine agonists, as well as surgical interventions such as deep brain stimulation. Additionally, the review discusses promising new therapies, including immunotherapy aimed at neuroinflammation and gene therapy for disease modification. The impact of lifestyle changes such as exercise and diet on reducing PD risk and enhancing symptom management are also considered. In conclusion, this review highlights the complex nature of Parkinson's disease and underscores the need for a holistic approach that combines pharmacotherapy, advanced treatments, and lifestyle adjustments. By addressing both symptom management and disease modification, these strategies provide hope for improving quality of life.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"27-52"},"PeriodicalIF":3.3,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143406063","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":"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, has 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":"1-29"},"PeriodicalIF":3.3,"publicationDate":"2026-01-01","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}
Current gene therapyPub Date : 2026-01-01DOI: 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":"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":"173-186"},"PeriodicalIF":3.3,"publicationDate":"2026-01-01","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}
Current gene therapyPub Date : 2026-01-01DOI: 10.2174/0115665232356703250325075309
Ozal Beylerli, Ilgiz Gareev, Elmar Musaev, Tatiana Ilyasova, Sergey Roumiantsev, Vladimir Chekhonin
{"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":"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":"85-115"},"PeriodicalIF":3.3,"publicationDate":"2026-01-01","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":"Association of KIM-1 (HAVCR1) Expression with the Tumor Immune Microenvironment in Clear Cell Renal Cell Carcinoma.","authors":"Panagiotis J Vlachostergios, Athanasios Karathanasis, Foteini Karasavvidou, Vassilios Tzortzis","doi":"10.2174/0115665232402424250721114133","DOIUrl":"10.2174/0115665232402424250721114133","url":null,"abstract":"<p><strong>Introduction: </strong>Kidney injury molecule 1 (KIM-1) is a cell-surface glycoprotein expressed in the proximal tubules and encoded by the hepatitis A virus cellular receptor 1 (HAVCR1) gene. It is also expressed in renal cell carcinoma (RCC).</p><p><strong>Objective: </strong>This study examined the immune landscape of clear cell RCC in association with HAVCR1 expression.</p><p><strong>Methods: </strong>Next-generation sequencing (NGS) data from ccRCC tumor samples of patients from The Cancer Genome Atlas (TCGA) were interrogated for enrichment of immune infiltrates and checkpoints in tumors harboring high HAVCR1 mRNA expression or/and amplification.</p><p><strong>Results: </strong>HAVCR1 mRNA expression was positively associated with presence of CD8<sup>+</sup> (r = 0.254, p = 3.03 x 10<sup>-8</sup>) and CD4 T-cells (r = 0.329, p = 3.98 x 10<sup>-13</sup>), while it was negatively associated with T-regulatory (T-regs) (r = ̶ 0.2, p = 1.47 x 10<sup>-5</sup>) and myeloid-derived suppressor cells (MDSCs) (r = ̶.0.285, p = 4.92 x 10<sup>-10</sup>). HAVCR1 amplification was also associated with CD8<sup>+</sup> (p = 0.0019), CD4<sup>+</sup> T cells (p = 0.0002) while expression of HAVCR1 gene was positively associated with immune checkpoints PD-L1 (CD274) (r = 0.331, p = 4.64 x 10<sup>-15</sup>) and CTLA4 mRNA expression (r = 0.085, p = 0.05). HAVCR1 transcript levels were directly correlated with those of Polybromo-1 (PBRM1) (r = 0.276, p = 9.36 x 10<sup>-11</sup>) while inversely related with BRCA-associated protein 1 (BAP1) gene expression (r = ̶ 0.134, p = 1.94 x 10<sup>-3</sup>).</p><p><strong>Discussion: </strong>The study reveals that high HAVCR1 (KIM-1) expression in clear cell RCC is associated with a distinct immune profile characterized by increased CD8<sup>+</sup>/CD4<sup>+</sup> T-cell infiltration and immune checkpoint expression, suggesting a potential role in predicting immunotherapy response, though the observational nature and reliance on TCGA data limit causal inference.</p><p><strong>Conclusions: </strong>Collectively, a potential immune-regulatory role of KIM-1 in clear cell RCC is implicated. This could be exploited for predicting benefit from adjuvant immunotherapy.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"11-16"},"PeriodicalIF":3.3,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144741444","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}
Current gene therapyPub Date : 2026-01-01DOI: 10.2174/0115665232417125251021114250
Nai Liang, Hui Chen, Yi Yang, Benlong Guo, Zehai Xu, Li Li, Yunfeng Jin
{"title":"Foxp3<sup>+</sup> Tregs Promote M2 Macrophage Polarization via Sirt1-ERK1/2- STAT3 Pathway in Ovarian Cancer Progression.","authors":"Nai Liang, Hui Chen, Yi Yang, Benlong Guo, Zehai Xu, Li Li, Yunfeng Jin","doi":"10.2174/0115665232417125251021114250","DOIUrl":"10.2174/0115665232417125251021114250","url":null,"abstract":"<p><strong>Introduction: </strong>Epithelial Ovarian Cancer (EOC) is a highly aggressive gynecological malignancy with a high mortality rate primarily due to late-stage diagnosis and metastatic dissemination. Regulatory T cells (Tregs) have emerged as critical mediators of immune evasion, yet the role of Foxp3<sup>+</sup> Tregs in modulating Tumor-Associated Macrophage (TAM) polarization and the underlying molecular mechanisms in EOC remains unclear.</p><p><strong>Methods: </strong>An orthotopic EOC mouse model and in vitro co-culture systems were employed to investigate the effects of Foxp3<sup>+</sup> Tregs on TAM polarization. Quantitative Real-Time PCR (qRTPCR), flow cytometry, Western blotting, wound healing, and transwell assays were performed to assess gene expression, immune cell infiltration, and tumor cell migration/invasion. Foxp3 knockdown was achieved using Adeno-Associated Virus (AAV)-mediated delivery to evaluate its effects in vivo.</p><p><strong>Results: </strong>Foxp3<sup>+</sup> Tregs induced macrophage polarization toward the M2 phenotype, characterized by downregulation of M1 markers (IL-1β, iNOS) and upregulation of M2 markers (IL-10, Arg-1). Mechanistically, Foxp3<sup>+</sup> Tregs activated the Sirt1-ERK1/2-STAT3 signaling pathway while suppressing NF-κB activity. In vitro, Foxp3<sup>+</sup> Tregs enhanced the migratory and invasive capacities of ovarian cancer cells, whereas in vivo Foxp3 knockdown significantly reduced tumor growth and M2 macrophage infiltration.</p><p><strong>Discussion: </strong>These findings suggest that Foxp3<sup>+</sup> Tregs play a pivotal role in shaping the immunosuppressive tumor microenvironment in EOC by promoting M2 macrophage polarization through Sirt1-ERK1/2-STAT3 signaling and NF-κB suppression, ultimately facilitating tumor progression.</p><p><strong>Conclusion: </strong>Foxp3<sup>+</sup> Tregs drive immunosuppressive macrophage polarization and ovarian cancer progression, highlighting Foxp3 as a potential therapeutic target for EOC treatment.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"1-11"},"PeriodicalIF":3.3,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145430313","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":"Gene Therapy: Transforming the Battle Against Pancreatic Cancer.","authors":"Rohit Sharma, Sourabh Kumar, Rashmi Ghosh, Kumari Komal, Manish Kumar","doi":"10.2174/0115665232364196250131102330","DOIUrl":"10.2174/0115665232364196250131102330","url":null,"abstract":"<p><p>Pancreatic cancer remains one of the most aggressive and lethal malignancies, with a dismal prognosis despite advancements in conventional treatment modalities. Gene therapy has emerged as a promising approach to combat pancreatic cancer by targeting the underlying genetic alterations and harnessing the power of the immune system. This review explores the current landscape of gene therapy strategies for pancreatic cancer, including gene replacement therapy, gene silencing, immunotherapy enhancement, and oncolytic virotherapy. Gene replacement therapy aims to restore the function of tumor suppressor genes, such as TP53, while gene silencing targets oncogenes like KRAS (Kirsten rat sarcoma viral oncogene homolog) to inhibit tumor growth. Immunotherapy enhancement, particularly through chimeric antigen receptor (CAR) T-cell therapy, has shown potential in overcoming the immunosuppressive tumor microenvironment. Oncolytic viruses, engineered to replicate in and destroy cancer cells selectively, have demonstrated efficacy in preclinical models and are being evaluated in clinical trials. Recent advances, including the successful treatment of a patient with advanced pancreatic cancer using neoantigen T-cell receptor gene therapy, highlight the potential of personalized gene therapy approaches. However, challenges such as precise gene delivery, tumor heterogeneity, and ethical considerations must be addressed to realize the potential of gene therapy for pancreatic cancer fully. Ongoing research and clinical trials are expected to facilitate the way for the development of safe and effective gene therapies, offering hope for improved outcomes in pancreatic cancer.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"152-159"},"PeriodicalIF":3.3,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143398543","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":"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":"1-14"},"PeriodicalIF":3.3,"publicationDate":"2026-01-01","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}
Current gene therapyPub Date : 2026-01-01DOI: 10.2174/0115665232412138250722020114
Ying Shi, Qirui Shen, Aimin Jiang, Hong Yang, Kexin Li, Jian Zhang, Anqi Lin, Peng Luo
{"title":"DrugSurvPlot: A Novel Web-Based Platform Harnessing Drug Sensitivity Scores as Molecular Biomarkers for Pan-Cancer Survival Prognosis.","authors":"Ying Shi, Qirui Shen, Aimin Jiang, Hong Yang, Kexin Li, Jian Zhang, Anqi Lin, Peng Luo","doi":"10.2174/0115665232412138250722020114","DOIUrl":"10.2174/0115665232412138250722020114","url":null,"abstract":"<p><strong>Background: </strong>Using predicted drug sensitivity scores as survival biomarkers may improve precision medicine and overcome the limitations of genomically guided approaches in clinical trials.</p><p><strong>Methods: </strong>Pan-Cancer Drug Sensitivity Score Survival Analysis (DrugSurvPlot) is an interactive, login-free web analyzer built with R (v4.3.1), leveraging the Shiny package for interface/server logic, the DT package for data table queries/downloads, and the survival package for survival analysis. Data preprocessing was performed using OncoPredict, enabling users to export processed tables and results.</p><p><strong>Results: </strong>DrugSurvPlot integrates 189 GEO datasets (including 10 immune checkpoint inhibitor treatment datasets) and 33 TCGA datasets, totaling 85,531 records across 52 cancer types and 13 survival status data types, while incorporating 198 anticancer drugs from GDSC2. This tool supports two cutoff strategies for drug sensitivity scores, offers advanced survival analysis methods, and enables customizable high-definition visualization of results.</p><p><strong>Discussion: </strong>DrugSurvPlot represents a significant advancement in computational oncology by establishing predicted drug sensitivity scores as novel prognostic biomarkers for tumor survival analysis. This interactive platform integrates comprehensive datasets spanning 198 anticancer drugs and 52 cancer types, while providing researchers with intuitive tools for generating publication-ready Kaplan-Meier analyses. Current limitations in drug repertoire coverage and dataset diversity will be addressed through ongoing expansion of pharmacological databases and incorporation of emerging data modalities, including single-cell transcriptomics.</p><p><strong>Conclusions: </strong>In summary, DrugSurvPlot offers a no-code platform with comprehensive datasets, diverse cancer coverage, and customizable survival analysis, addressing critical research gaps. Continuous enhancements will improve predictive accuracy and clinical utility, establishing it as an evolving powerhouse in drug-survival investigations.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":"187-202"},"PeriodicalIF":3.3,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144741445","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}