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Latest Articles on Gene Therapy

Overview of latest articles and publications on gene therapy in PubMed, including Human Gene Therapy, Journal of Molecular Medicine and Journal of Gene Medicine. PubMed is a service of the US National Library of Medicine that includes over 18 million citations from MEDLINE and other life science journals.

  • A novel NFKB1 agonist remodels tumor microenvironment and activates dendritic cells to promote anti-tumor immunity in colorectal cancer Tue, 20 May 2025 06:00:00 -0400
    CONCLUSION: Our study identified a novel NFKB1 agonist that promotes anti-tumor immunity by remodeling the TME and activating DCs and that may provide a new way to overcome resistance to current anti-tumor immunotherapy in colorectal cancer.

  • Gene therapy for age-related macular degeneration: a promising frontier in vision preservation Tue, 20 May 2025 06:00:00 -0400
    Age-related macular degeneration (AMD) is a leading cause of central vision loss, progressively impairing the retina and affecting millions worldwide. By 2040, global cases of AMD are projected to reach 300 million, posing a significant public health challenge. While early AMD may only cause mild visual impairment, advanced stages, particularly neovascular (wet) and non-neovascular (dry) AMD, can lead to severe vision loss or legal blindness, substantially affecting daily life. The introduction...

  • Advancing cancer gene therapy: the emerging role of nanoparticle delivery systems Tue, 20 May 2025 06:00:00 -0400
    Gene therapy holds immense potential due to its ability to precisely target oncogenes, making it a promising strategy for cancer treatment. Advances in genetic science and bioinformatics have expanded the applications of gene delivery technologies beyond detection and diagnosis to potential therapeutic interventions. However, traditional gene therapy faces significant challenges, including limited therapeutic efficacy and the rapid degradation of genetic materials in vivo. To address these...

  • FGA can be used as a promising therapeutic target in osteoarthritis Tue, 20 May 2025 06:00:00 -0400
    CONCLUSIONS: FGA plays a pivotal role in the molecular pathology of OA and may represent a promising therapeutic target for the development of precision treatments for OA.

  • Genetic influence of the brain imaging phenotypes, brain and cerebrospinal fluid metabolites and brain genes on migraine subtypes: a Mendelian randomization and multi-omics study Tue, 20 May 2025 06:00:00 -0400
    CONCLUSIONS: Our study integrates cross-omics analyses to investigate the causal links between brain structure, metabolic alterations, gene expression, and migraine including its MA and MO subtypes. These findings provide novel insights into the pathophysiological mechanisms and potential targets for intervention across migraine subtypes.

  • miRNAs in secondary hyperparathyroidism: literature review Tue, 20 May 2025 06:00:00 -0400
    Secondary hyperparathyroidism (SHPT) is a common complication of Chronic kidney disease (CKD), which is mainly manifested by the overproduction of Parathyroid hormone (PTH), leading to multi-system pathologies such as calcium and phosphorus metabolism disorders, skeletal lesions, and cardiovascular diseases, which seriously affects the quality of life of patients. In recent years, the role of microRNAs (miRNAs) in the pathogenesis of SHPT has been gradually revealed, providing new research...

  • SUMOylation-regulated genes in colon cancer: expression patterns and clinical implications Tue, 20 May 2025 06:00:00 -0400
    Colon cancer (CRC) demonstrates significant heterogeneity, and identifying effective biomarkers can advance the development of precision therapies. Emerging evidence implicates SUMOylation-regulated genes as pivotal regulators of cancer-associated pathways, yet their prognostic potential and therapeutic implications in CRC remain unexplored. A comprehensive analysis of SUMOylation-regulated gene expression, clinical and prognostic value in CRC was performed using transcriptomic data from...

  • METTL3-mediated m⁶A methylation in cardiac diseases: pathogenic roles and therapeutic potential Tue, 20 May 2025 06:00:00 -0400
    Cardiac dysfunction is a leading cause of death each year, putting heavy burdens on the global healthcare system. To improve our understanding of cardiac disease, novel perspectives for exploring their pathogenesis mechanisms are needed, which contributes to finding novel diagnoses and therapy targets for cardiac disease. To be noteworthy, researchers have paid great attention to understanding the pathogenesis of cardiac diseases from the perspective of methyltransferase-like 3 (METTL3, the...

  • Genome Editing in Gynecological Oncology: The Emerging Role of CRISPR/Cas9 in Precision Cancer Therapy Tue, 20 May 2025 06:00:00 -0400
    CONCLUSION: Genome editing, particularly CRISPR/Cas9, holds substantial capacity for revolutionizing the treatment landscape of gynecological cancers by enabling highly specific, gene-targeted therapies that can overcome conventional treatment limitations such as chemoresistance and tumor recurrence. Emerging preclinical studies demonstrate the feasibility of correcting oncogenic mutations and enhancing the sensitivity of tumors to existing therapies. However, before clinical translation can be...

  • Synergistic targeting strategies for prostate cancer Tue, 20 May 2025 06:00:00 -0400
    Prostate cancer is the second most commonly diagnosed cancer and the fifth leading cause of death among men worldwide. Androgen deprivation therapy is a common prostate cancer treatment, but its efficacy is often hindered by the development of resistance, which results in reducing survival benefits. Immunotherapy showed great promise in treating solid tumours; however, clinically significant improvements have not been demonstrated for patients with prostate cancer, highlighting specific...

  • Hemorrhagic and ischemic risks of anti-VEGF therapies in glioblastoma Tue, 20 May 2025 06:00:00 -0400
    Glioblastoma (GBM) is one of the most aggressive primary brain tumors, characterized by extensive neovascularization and a highly infiltrative phenotype. Anti-vascular endothelial growth factor (VEGF) therapies, such as bevacizumab, have emerged as significant adjunct treatments for recurrent and high-grade GBM by targeting abnormal tumor vasculature. Despite demonstrated benefits in slowing tumor progression and alleviating peritumoral edema, these agents are associated with notable vascular...

  • Mass spectrometric insights into the protein composition of human cutaneous neurofibromas: comparison of neurofibromas with the overlying skin Tue, 20 May 2025 06:00:00 -0400
    CONCLUSION: The cNFs are relatively quiescent, consistent with their benign nature and limited growth potential. The development of pharmacological therapy for cNFs requires overcoming the high similarity between cNFs and the overlying skin. The present dataset can serve as a resource for future research on cNFs.

  • Genetic surveillance of Plasmodium falciparum populations following treatment policy revisions in the Greater Mekong Subregion Tue, 20 May 2025 06:00:00 -0400
    Genetic surveillance of Plasmodium falciparum (Pf) can track antimalarial-resistant strains, to inform decision-making by National Malaria Control Programmes (NMCPs). The GenRe-Mekong project prospectively collected 5982 samples in the Greater Mekong Subregion (GMS) between 2017 and 2022, genotyping drug resistance markers, and barcodes that recapitulate genetic variation. Genotypes were analyzed with the grcMalaria R package, first described in this paper, to translate genetic epidemiology data...

  • DNA nanoflower Oligo-PROTAC for targeted degradation of FUS to treat neurodegenerative diseases Tue, 20 May 2025 06:00:00 -0400
    Oligonucleotide-based medicine faces challenges in efficiently crossing the blood-brain barrier and rapidly reducing toxic proteins. To address these challenges, here we establish an integrated modality, brain-penetrant DNA nanoflowers incorporated with oligonucleotide-based proteolysis targeting chimeras. Using FUS as a proof-of-concept, mutations of which cause frontotemporal dementia and amyotrophic lateral sclerosis, we demonstrate that a FUS-engaging RNA oligonucleotide crosslinked to a...

  • The clinical and molecular landscape of breast cancer in women of African and South Asian ancestry Tue, 20 May 2025 06:00:00 -0400
    Addressing existing racial disparity in breast cancer is crucial to ensure equitable benefit across diverse communities. We evaluate the molecular and clinical effects of genetic ancestry in African and South Asian women compared to European using a combined cohort of 7136 breast cancer patients. We find that non-European patients present significantly earlier and die at a younger age. The African group has an increased prevalence of higher grade and hormone receptor negative disease. The South...

  • PKMYT1 kinase ameliorates cisplatin sensitivity in osteosarcoma Tue, 20 May 2025 06:00:00 -0400
    Cisplatin (DDP) remains a cornerstone therapy for osteosarcoma (OS); however, pervasive resistance severely limits its clinical efficacy and worsens patient outcomes. Developing strategies to enhance the chemotherapeutic responsiveness of OS cells is therefore of critical importance. Here, we conducted a kinome-wide clustered regularly interspaced short palindromic repeats (CRISPR) screen, coupled with transcriptome sequencing, to identify regulators of DDP sensitivity. This approach revealed...

  • Reactive oxygen species responsive nanomotors for gene edited metabolic disruption and immunotherapy Tue, 20 May 2025 06:00:00 -0400
    While gene-editing-based tumor therapy holds promise, conventional passive-diffusion vectors face limited penetration in dense solid tumors. Here, we developed a ROS-driven gene editing nanomotor (RDN@PL), which takes hemin as the core and encapsulates CRISPR/Cas9 plasmids targeting LDHA (A glycolysis key enzyme). In tumor microenvironments, RDN@PL consumes extracellular ROS to fuel self-diffusiophoresis, achieving higher intratumoral accumulation than passive particles. Upon internalization,...

  • Analysis of clinical characteristics in 4 pediatric cases of glycogen storage disease type Ⅸa Tue, 20 May 2025 06:00:00 -0400
    Objective: To investigate the clinical manifestations, pathological features, and genetic variant characteristics of children with glycogen storage disease type Ⅸa (GSD Ⅸa). Methods: A retrospective case series analysis was conducted to collected and analyzed the medical history, biochemical markers, liver ultrasound results, liver histopathological findings, genotypes, treatment regimens, and follow-up data of 4 pediatric patients diagnosed with GSD IXa in the Department of Infectious Diseases...

  • Intervention effect analysis of TPMT and NUDT15 genotyping on the tolerability of azathioprine or 6-mercaptopurine therapy in pediatric inflammatory bowel disease Tue, 20 May 2025 06:00:00 -0400
    Objective: To investigate the impact of pre-treatment TPMT and NUDT15 genotyping on medication selection, tolerability and discontinuation rates of azathioprine or 6-mercaptopurine therapy in children with inflammatory bowel disease (IBD). Methods: A retrospective cohort study was conducted on 181 children with IBD who were scheduled for azathioprine or 6-mercaptopurine therapy at the Department of Gastroenterology, Children's Hospital, Zhejiang University School of Medicine between January 2010...

  • Hyaluronan-decorated-phloroglucinol-loaded mesoporous silica downregulates GLI1 and SMO genes of hedgehog signaling pathway in gastrointestinal cancer stem-like cells Tue, 20 May 2025 06:00:00 -0400
    Cancer stem-like cells (CSCs), a small subset of cells within the tumor microenvironment, favor tumor relapse and remain a major obstacle in cancer therapy. Hence, hyaluronan-based mesoporous silica nano-formulation with phloroglucinol (MSN-PG-HA) was employed in our study to address the cancer relapse by targeting CD44. Various in vitro cellular assays were conducted to assess the nano-formulation's effect on suppressing CSC characteristics in AGS, HCT-116 and SW-620. The CUA assay indicated...