Current gene therapy最新文献

{"title":"Exploring the Epigenetic Mechanisms Underlying Chronic Diseases: A Comprehensive Review.","authors":"Afaf Aldahish, Rajalakshimi Vasudevan, Shaima Y Al Qasim, Malath Saeed Alshahrani, Lujain Khalid Almohsen, Salma Mohammed Alghanoom, Arwa Ali AlQahtani, Wajan Khalid Alshahrani","doi":"10.2174/0115665232443611260121063601","DOIUrl":"https://doi.org/10.2174/0115665232443611260121063601","url":null,"abstract":"<p><p>Epigenetics, the study of heritable changes in gene expression without changes to the DNA sequence, has emerged as an important regulator of disease risk and development. Epigenetic alterations, such as DNA methylation, histone modifications, and non-coding RNAs, act as molecular bridges between genetic predisposition and environmental factors. Understanding these changes is critical for determining the pathophysiology of chronic illnesses. This article looks at the involvement of epigenetic mechanisms in chronic diseases such as cardiovascular disease, obesity, depression, Alzheimer's disease, and diabetes mellitus. The emphasis is on recent epigenetic discoveries, their implications for disease risk assessment, and the prospect of epigenetic-based therapeutics in precision medicine. Epigenome-Wide Association Studies (EWAS) and next-generation sequencing technologies have revealed disease-specific epigenetic patterns, underlining their potential as biomarkers for early detection and risk stratification. Epigenetic changes affect important molecular pathways that control metabolic regulation, neuroplasticity, inflammatory response, and cellular homeostasis. Epigenetic modification-targeting therapies, such as DNA methylation inhibitors, histone deacetylase inhibitors, and RNA-based treatments, have shown promise in preclinical and early clinical trials. Epigenetic research offers a transformational framework for understanding the intricate interplay of genetic and environmental variables in chronic illness etiology. Epigenetic alterations are reversible, which opens up new possibilities for therapeutic intervention and individualized therapy. Future research should concentrate on improving epigenetic biomarkers, determining causal links in disease progression, and incorporating epigenetic findings into clinical practice. The advancement of epigenetic medicines has the potential to transform disease prevention, treatment, and global health policies.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2026-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147456202","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":"Molecular Complexities of Dementia: PAISA Mutations and Targeting TAF2N as Therapeutic Avenues.","authors":"Siva Prasad Panda, Sanjesh Kumar, Mansi Singh, Vikrant Singh","doi":"10.2174/0115665232389636251115111503","DOIUrl":"https://doi.org/10.2174/0115665232389636251115111503","url":null,"abstract":"<p><p>Millions of individuals worldwide are affected by Alzheimer's disease dementia (ADD) and frontotemporal dementia (FTD), with FTD characterized by degeneration of the frontal and temporal lobes leading to cognitive and behavioral impairments. A subset of Alzheimer's cases exhibits familial inheritance, with the PAISA mutation, a glutamic acid to alanine substitution at codon 280 (E280A) in the PSEN1 gene, being a primary cause of early-onset dementia. PSEN1 encodes a key component of the γ-secretase complex, which cleaves amyloid precursor protein (APP) to generate beta-amyloid (Aβ) peptides. The PAISA mutation disrupts normal Aβ processing, leading to overproduction or accumulation of Aβ, formation of amyloid plaques, and accelerated progression of dementia. Its prevalence is particularly high in Colombian families, giving rise to the term \"PAISA mutation.\" The APOE genotype further modulates the clinical manifestation in PAISA carriers, with APOE2 potentially delaying disease onset, whereas APOE4 is associated with earlier onset. Recent research highlights TAF2N (also known as RBP56, encoded by TAF15) as a promising therapeutic target, as its modulation may regulate AD-associated genes, reduce toxic Aβ isoforms, modulate tau and APP pathways, protect neurons, and enhance synaptic function. Overall, understanding the molecular effects of PAISA mutations and exploring TAF2N-targeted therapies offers novel avenues for addressing early-onset familial AD, providing insights into broader mechanisms of disease pathogenesis.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2026-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147442419","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":"Exploiting the Warburg Effect: A Potential Ally for Cancer Treatment.","authors":"Vandana Soni","doi":"10.2174/0115665232387693251111102627","DOIUrl":"https://doi.org/10.2174/0115665232387693251111102627","url":null,"abstract":"<p><p>Mitochondrial dysfunction and the Warburg effect, characterized by aerobic glycolysis, are hallmarks of cancer metabolism, facilitating tumor progression and resistance to therapies. These metabolic shifts allow cancer cells to prioritize glycolysis over oxidative phosphorylation, contributing to rapid proliferation, immune evasion, and metastasis. This review explores the intricate regulation of the Warburg effect by enzymes, transcription factors, and non-coding RNAs. Key players, such as hexokinase, pyruvate kinase M2, and glucose transporters, are discussed as central drivers of glycolysis. This review highlights therapeutic strategies targeting these pathways, including small-molecule inhibitors, combination therapies, and traditional medicines. Advanced diagnostic tools, such as FDG-PET imaging and metabolic profiling, are evaluated for their potential to personalize cancer treatment. The role of synthetic lethality, immunotherapy, and novel drug combinations in addressing metabolic vulnerabilities is also examined. Furthermore, the review underscores the impact of metabolic reprogramming on the tumor microenvironment and its implications for immune modulation. Targeting the Warburg effect presents a promising avenue for overcoming drug resistance and enhancing cancer therapy. This review provides a comprehensive framework for integrating metabolic reprogramming into advanced therapeutic and diagnostic strategies, paving the way for personalized cancer treatment approaches.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2026-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147442387","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":"Folic Acid-Conjugated Nanocarriers for Targeted Cancer Therapy: Recent Patents and Future Prospects.","authors":"Nandini Sharma, Yukta Garg, Raj Kumar Narang, Amandeep Singh","doi":"10.2174/0115665232425332251126045318","DOIUrl":"https://doi.org/10.2174/0115665232425332251126045318","url":null,"abstract":"<p><p>As folate receptors are overexpressed on the surface of many tumor cells, folic acidconjugated nanocarriers have emerged as a promising approach for targeted cancer therapy. These nanocarriers enhance therapeutic efficacy while reducing systemic toxicity and adverse effects by enabling selective drug delivery. The design, synthesis, and application of folic acid-functionalized nanocarriers such as polymeric nanoparticles, liposomes, dendrimers, and micelles have advanced significantly, as evidenced by recent patents. Key objectives of these developments include improving stability, drug loading capacity, controlled release, and receptor-mediated endocytosis. To translate these targeted nanocarriers from bench to bedside, this study examines significant patents submitted over the past ten years, evaluates their clinical potential, and discusses current challenges and potential future directions.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2026-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147442410","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":"Revolutionizing Drug and Gene Delivery: Cutting-Edge Smart Polymers for Precision Release.","authors":"Atul Pratap Singh, Shamim, Dhananjay Taumar, Himanchal Sharma, Anshu Gujjar, Vatan Chaudhary, Iram Rajpoot","doi":"10.2174/0115665232390238251121074917","DOIUrl":"https://doi.org/10.2174/0115665232390238251121074917","url":null,"abstract":"<p><p>Smart polymer-based drug and gene delivery systems are revolutionizing contemporary therapeutics with their increased specificity, efficacy, and safety. Compared to traditional carriers, smart polymers exhibit stimuli-responsive behavior, enabling controlled and site-specific drug release in response to physiological cues, such as pH, temperature, or enzyme activity. Such dynamic materials allow accurate drug delivery and minimize systemic toxicity, along with providing improved bioavailability. This review delves into some of the latest developments in stimuliresponsive polymers, nanoplatforms with multiple functions, and hybrid polymer systems, highlighting their potential in controlled drug delivery and theragnostics. Challenges, including biocompatibility, stability, and overcoming biological barriers, such as the blood-brain barrier, are also covered. By combining smart polymers with nanotechnology and targeted personalized medicine strategies, the discipline is set to redefine targeted therapy and maximize therapeutic outcomes.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2026-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147442404","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":"Discovery of Genes Related to Cuproptosis and Mitophagy to Improve Myocardial Infarction Diagnosis and Treatment.","authors":"Fenlong Xue, Yuhui Zhang, Chao Chang, Qingliang Chen","doi":"10.2174/0115665232440699251209120654","DOIUrl":"https://doi.org/10.2174/0115665232440699251209120654","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Introduction: &lt;/strong&gt;Emerging evidence links cuproptosis and mitophagy to the progression of myocardial infarction (MI). This study explored cuproptosis- and mitophagy-related gene modules in MI, aiming to identify potential biomarkers to improve MI management.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;The GSE66360 dataset, containing 50 control and 49 MI samples, was obtained from GEO. Differentially expressed genes (DEGs) were identified using the \"limma\" 3.42.2 package, and pathway differences were analyzed via gene set enrichment analysis (GSEA). After clustering gene modules related to cuproptosis and mitophagy using weighted gene co-expression network analysis (WGCNA), core modular genes were selected. LASSO regression and random forest were employed for feature selection. Immune microenvironment profiling was conducted using singlesample GSEA (ssGSEA) and CIBERSORT algorithms. Finally, potential therapeutic targets were identified using DSigDB and molecular docking.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;We identified the DEGs between MI and control samples. GSEA analysis showed that these genes were associated with the cell cycle, glycolysis, and the inflammatory signaling pathway. The dark green module identified by WGCNA was correlated with both cuproptosis and mitophagy and enriched in oxidative phosphorylation and immune processes. By combining the core modular genes and DEGs, six hub genes were selected using LASSO and random forests. A 3-gene diagnostic model established based on GPCPD1, S100A8, and CD55 achieved an Area under the Curve (AUC) of 0.959. The three genes were upregulated in MI and correlated with immune infiltration. Molecular docking showed Corbadrine and Dicyclomine were potential therapeutic agents targeting CD55.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Discussion: &lt;/strong&gt;This study highlights the broader implications of linking cuproptosis and mitophagy in MI, proposing a novel perspective on mitochondrial dysfunction as a central hub connecting metabolic stress, immune dysregulation, and cell death. The identified core genes-S100A8, CD55, and GPCPD1-not only serve as potential diagnostic markers but may represent functional nodes at the intersection of copper-dependent cell death and mitochondrial quality control. Their strong association with inflammatory cell infiltration suggests that these genes could influence the post-MI immune microenvironment, potentially affecting both injury progression and repair. Moreover, the feasibility of targeting CD55, as indicated by molecular docking, opens new avenues for therapeutic interventions to modulate complement activation and inflammation. While the findings are computationally derived, they generate testable hypotheses about crosstalk between emerging cell death pathways and immune-metabolic pathways in MI, underscoring the need for future experimental studies to validate these interactions and explore their translational potential in cardiovascular disease.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;Th","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2026-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147321379","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":"Padlock Assay in Transthyretin Amyloidosis: A Feasibility Study.","authors":"Maria Grazia Di Certo, Francesca Gabanella, Claudio Passananti, Nicoletta Corbi, Annalisa Onori, Cinzia Pisani, Marco Fiore, Marco de Vincentiis, Giampiero Ferraguti, Silvia Francati, Irene Bottillo, Valentina Lodato, Mara Riminucci, Chiara Cambieri, Laura Libonati, Federica Moret, Cristina Chimenti, Alessandro Corsi, Maurizio Inghilleri, Marco Ceccanti","doi":"10.2174/0115665232404606251128044439","DOIUrl":"https://doi.org/10.2174/0115665232404606251128044439","url":null,"abstract":"<p><strong>Introduction: </strong>The ability to distinguish between transcripts that differ by a single nucleotide positions our padlock assay as a highly accurate imaging tool for detecting disorders associated with structural variations in the human genome. In this study, we evaluated the effectiveness of the padlock assay in identifying TTR gene variants in a case of transthyretin amyloidosis (ATTR), a rare multisystemic disease. ATTR may result from autosomal dominant mutations in the TTR gene or occur in a wild-type form.</p><p><strong>Methods: </strong>We applied the padlock assay in combination with rolling circle amplification (RCA) and fluorescence microscopy, using peripheral blood mononuclear cells (PBMCs) as clinical samples.</p><p><strong>Results: </strong>Using the padlock assay, 1) we detected intracellular TTR transcripts in 80% of PBMCs, including a benign variant caused by a single nucleotide substitution in intron 3, 2) we visualized the subcellular localization of both coding and non-coding regions of TTR transcripts, and 3) through dual staining, we simultaneously detected both wild-type and mutated TTR intron 3 in patient- derived cells. Additionally, we found that PBMCs and platelets are immunoreactive to TTR antibodies, suggesting that immunocompetent cells may contribute to the distribution of TTR protein across tissues and organs.</p><p><strong>Discussion: </strong>We demonstrate that the padlock assay can serve as a non-invasive imaging test capable of spatially detecting genomic variants in ATTR.</p><p><strong>Conclusion: </strong>These findings suggest that the padlock assay has potential application in evaluating the efficacy of disease-modifying therapies in extra-hepatic cells. Moreover, this study is the first to highlight PBMCs as a valuable source for advancing our understanding of ATTR pathogenesis and for supporting the development of improved therapeutic approaches.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2026-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147282812","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":"Nano-vaccines in Pandemic Preparedness: Pioneering Rapid Development and Deployment for Global Immunity.","authors":"Dhananjay Taumar, Atul Pratap Singh, Himanchal Sharma, Anshu Gujjar, Iram Rajpoot, Vatan Chaudhary","doi":"10.2174/0115665232391895251117041913","DOIUrl":"https://doi.org/10.2174/0115665232391895251117041913","url":null,"abstract":"<p><strong>Background: </strong>The increasing frequency of global pandemics, such as COVID-19, H1N1, and Ebola, highlights the urgent need for innovative vaccine technologies that enable rapid response and broader accessibility. Nanovaccines, leveraging nanotechnology for targeted antigen delivery and enhanced immune activation, have emerged as a promising solution for improving vaccine efficacy, stability, and distribution.</p><p><strong>Objective: </strong>This review examines the role of nano vaccines in pandemic preparedness, focusing on their benefits, mechanisms of action, recent advancements, and the challenges associated with their development and deployment.</p><p><strong>Methods: </strong>A comprehensive literature review was conducted using databases such as PubMed, Scopus, and Web of Science. Research articles and clinical trial data from the past decade were analyzed, with a focus on nano-vaccine platforms, immune response mechanisms, and global deployment strategies.</p><p><strong>Key findings: </strong>Nanovaccines offer accelerated development timelines, enhanced antigen presentation, and prolonged immune responses via platforms including lipid-based nanoparticles, polymeric systems, virus-like particles, and inorganic nanoparticles. The success of mRNA vaccines during COVID-19 has demonstrated the transformative potential of nanotechnology in vaccine development. However, significant challenges remain, including safety concerns, large-scale manufacturing, regulatory approval procedures, and equitable access, particularly in low- and middle-income countries.</p><p><strong>Conclusion: </strong>Nanovaccines offer substantial promise for global pandemic preparedness. Tackling current challenges through international collaboration, policy support, and increased investment will be essential for ensuring their widespread adoption. Developing nanotechnology-driven vaccine solutions can strengthen global health resilience and enable faster, more effective responses to future pandemics.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2026-01-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146050512","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":"Advancements in CAR-T Cell Therapy for Ovarian Cancer: Current Strategies and Future Directions.","authors":"Ying Liu, Hao Ai","doi":"10.2174/0115665232414033251124073755","DOIUrl":"https://doi.org/10.2174/0115665232414033251124073755","url":null,"abstract":"<p><p>Chimeric Antigen Receptor (CAR) T-cell therapy has emerged as a transformative approach in oncology, particularly for hematologic malignancies. However, its application to solid tumors, such as ovarian cancer, remains challenging. This review discusses recent advancements in CAR-T cell therapy specifically targeting ovarian cancer, with a focus on current strategies and future directions. We first introduce the fundamental structure of CARs, detailing the core components including the antigen-binding domain, the transmembrane domain, and the signaling domains. The optimization of CAR-T design is then examined, highlighting innovative strategies such as bispecific CAR-Ts, co-expression CAR-Ts, fine-tuning of CAR constructs, and cytokine-modified CAR-Ts. The review further explores a comprehensive array of antigen targets relevant to ovarian cancer, ranging from HER2 and mesothelin to more novel targets like CD47 and L1CAM. Additionally, we investigate how nanotechnology is enhancing CAR-T cell therapy for solid tumors, with specific attention to mRNA delivery systems, liposomal nanoparticles, hydrogel-based platforms, and the integration of photothermal therapy.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2026-01-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146050474","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":"Glioblastoma Organoid Technology: Approach to Target the Complex Tumor Microenvironment with Promising Drug Responsiveness.","authors":"Priya Bisht, Ruchi Pandey, Tanveer A Tabish, Nanasaheb D Thorat, Murali Kumarasamy, Krishna Murti, Nitesh Kumar","doi":"10.2174/0115665232400556251120095720","DOIUrl":"https://doi.org/10.2174/0115665232400556251120095720","url":null,"abstract":"<p><p>Glioblastoma remains one of the most lethal brain tumors with limited therapeutic options and a dismal survival rate, largely due to its highly complex Tumor Microenvironment (TME) and frequent drug resistance. In response, three-dimensional (3D) organoid technology has emerged as a powerful tool for modeling glioblastoma, offering a physiologically relevant in vitro system that closely mimics the in vivo architecture, cellular heterogeneity, and drug response of human tumors. Unlike traditional 2D cultures or animal models, glioblastoma organoids enable highthroughput drug screening, personalized therapy testing, and early diagnostic research by preserving key features of the TME. However, despite their immense promise, current organoid models face substantial limitations, including the absence of immune components, functional vasculature, and region-specific neuronal subtypes, thereby restricting their full translational potential, especially for immunotherapy studies. Existing models, such as genetically engineered cerebral organoids (Neo- Cor) and glioblastoma spheroid co-cultures (GLICO), either fail to reflect patient heterogeneity or are constrained by time-intensive preparation. Additionally, patient-derived organoids may lose genetic fidelity over prolonged culture. The novelty of this work lies in its advocacy for engineered cell-based strategies and Adult Stem Cell (AdSC)-derived organoids to overcome these challenges. By enhancing the intrinsic properties of organoid cells and integrating endothelial and immune components, this approach offers a next-generation platform for more accurate modeling of glioblastoma, with greater relevance for drug responsiveness, chemosensitization studies, gene editing, and regenerative applications. This distinguishes the present work from previous studies by not only identifying the gaps in current organoid technologies but also proposing specific, actionable improvements that bring organoid culture closer to clinical translation in glioblastoma research.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2026-01-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146050553","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}