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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.

  • Rare dual MYH9-ROS1 fusion variants in a patient with lung adenocarcinoma: A case report Fri, 24 Jan 2025 06:00:00 -0500
    RATIONALE: ROS proto-oncogene 1 (ROS1) fusion is a rare but important driver mutation in non-small cell lung cancer, which usually shows significant sensitivity to small molecule tyrosine kinase inhibitors. With the widespread application of next-generation sequencing (NGS), more fusions and co-mutations of ROS1 have been discovered. Non-muscle myosin heavy chain 9 (MYH9) is a rare fusion partner of ROS1 gene as reported. Here, we report an even rare case with coexistence of short and long...

  • Three cases of hemoglobin M disease in a family lineage: Case report and literature review Fri, 24 Jan 2025 06:00:00 -0500
    RATIONALE: This study presents a case of hemoglobin M disease (HMD), a rare inherited disorder characterized by persistent cyanosis and hypoxemia, observed across 3 generations within a single family. The diagnosis of HMD poses significant challenges, particularly in asymptomatic individuals, due to its rarity and the subtlety of its symptoms. Notably, there is a scarcity of reports on methemoglobinemia in pediatric populations, which further complicates early detection and intervention. The...

  • One hundred thirty-four germ line PU.1 variants and the agammaglobulinemic patients carrying them Fri, 24 Jan 2025 06:00:00 -0500
    Leukopoiesis is lethally arrested in mice lacking the master transcriptional regulator PU.1. Depending on the animal model, subtotal PU.1 loss either induces acute myeloid leukemia or arrests early B cell and dendritic cell development. Although humans with absolute PU.1 deficiency have not been reported, a small cadre of congenital agammaglobulinemia patients with sporadic, inborn PU.1 haploinsufficiency were recently described. To better estimate the penetrance, clinical complications,...

  • Bioprinted optoelectronically active cardiac tissues Fri, 24 Jan 2025 06:00:00 -0500
    Electrical stimulation of existing three-dimensional bioprinted tissues to alter tissue activities is typically associated with wired delivery, invasive electrode placement, and potential cell damage, minimizing its efficacy in cardiac modulation. Here, we report an optoelectronically active scaffold based on printed gelatin methacryloyl embedded with micro-solar cells, seeded with cardiomyocytes to form light-stimulable tissues. This enables untethered, noninvasive, and damage-free...

  • Chemopreventive role of β-caryophyllene in DMBA-induced skin cancer: Modulation of apoptotic pathways and PI3K/Akt signaling in Swiss albino mice Fri, 24 Jan 2025 06:00:00 -0500
    CONCLUSIONS: This study provides compelling evidence that the antioxidant and pro-apoptotic effects of β-caryophyllene contribute to its chemopreventive properties in DMBA-induced skin carcinogenesis in mice. The modulation of key apoptotic signaling pathways and the suppression of the PI3K/Akt pathway by BCP underscores its potential as a therapeutic agent for preventing skin cancer. These findings pave the way for further exploration of BCP as a promising candidate for skin cancer prevention...

  • Unraveling Mechanisms of Genetic Risks in Metabolic Dysfunction-Associated Steatotic Liver Diseases: A Pathway to Precision Medicine Fri, 24 Jan 2025 06:00:00 -0500
    Metabolic dysfunction-associated steatotic liver disease (MASLD) is a growing global health problem, affecting ∼1 billion people. This condition is well established to have a heritable component with strong familial clustering. With the extraordinary breakthroughs in genetic research techniques coupled with their application to large-scale biobanks, the field of genetics in MASLD has expanded rapidly. In this review, we summarize evidence regarding genetic predisposition to MASLD drawn from...

  • Case Series of Anesthetic Management of Gene Therapy in Children With Aromatic L-Amino Acid Decarboxylase Deficiency Fri, 24 Jan 2025 06:00:00 -0500
    CONCLUSION: Using an anesthetic plan based on atropine, ketamine, sevoflurane, and a titrated infusion of norepinephrine, prolonged anesthesia appeared to be safe and reproductible in this population.

  • Integrative Transcriptome-Wide Association Study With Expression Quantitative Trait Loci Colocalization Identifies a Causal VAMP8 Variant for Nasopharyngeal Carcinoma Susceptibility Fri, 24 Jan 2025 06:00:00 -0500
    Nasopharyngeal carcinoma (NPC) is an Asia-prevalent malignancy, yet its genetic underpinnings remain incompletely understood. Here, a transcriptome-wide association study (TWAS) is conducted on NPC, leveraging gene expression prediction models based on epithelial tissues and genome-wide association study (GWAS) summary statistics from 1577 NPC cases and 6359 controls of southern Chinese descent. The TWAS identifies VAMP8 on chromosome 2p11.2 as a novel susceptibility gene for NPC. Further...

  • WFDC3 sensitizes colorectal cancer to chemotherapy by regulating ATM/ATR kinase signaling pathway Fri, 24 Jan 2025 06:00:00 -0500
    Chemoresistance is an ongoing challenge for colorectal cancer (CRC) that significantly compromises the anti-tumor efficacy of current drugs. Identifying effective targets or drugs for overcoming chemoresistance is urgently needed. Our previous study showed that WFDC3 served as a tumor suppressor that hindered CRC metastasis. However, the function of WFDC3 in chemotherapy remains unknown. Here, we found that high WFDC3 expression in CRC patients treated with oxaliplatin was associated with a...

  • Metrnl and Cardiomyopathies: From Molecular Mechanisms to Therapeutic Insights Fri, 24 Jan 2025 06:00:00 -0500
    Cardiomyopathies, a diverse group of diseases affecting the heart muscle, continue to pose significant clinical challenges due to their complex aetiologies and limited treatment options targeting underlying genetic and molecular dysregulations. Emerging evidence indicates that Metrnl, a myokine, adipokine and cardiokine, plays a significant role in the pathogenesis of various cardiomyopathies. Therefore, the objective of this review is to examine the role and mechanism of Metrnl in various...

  • Rhanterium Epapposum Essential Oil and Its Primary Compounds Control Infection, Inflammation, and Serum Electrolyte Imbalance in Mice with Giardiasis Fri, 24 Jan 2025 06:00:00 -0500
    CONCLUSION: The current investigation showed that R. epapposum essential oil and its main compounds controlled the Giardia infection in mice by inhibiting inflammation, and serum electrolyte imbalance. After confirmation in further studies, these compounds may be considered for development as a novel therapeutic option for the treatment of giardiasis.

  • Integrated Analysis of Bulk RNA Sequencing, eQTL, GWAS, and Single-Cell RNA Sequencing Reveals Key Genes in Hepatocellular Carcinoma Fri, 24 Jan 2025 06:00:00 -0500
    Hepatocellular carcinoma (HCC) poses a continual therapeutic challenge owing to its elevated incidence and unfavourable prognosis, underscoring the critical need for the discovery of new molecular targets for detection and therapy. This work included the analysis of three publically accessible HCC datasets from TCGA and GEO. Instrumental variables (IVs) were derived via expression quantitative trait loci (eQTL) analysis, then followed by two-sample Mendelian randomisation (MR) analysis utilising...

  • Epigenomics-guided precision oncology: Chromatin variants in prostate tumor evolution Fri, 24 Jan 2025 06:00:00 -0500
    Prostate cancer is a common malignancy that in 5%-30% leads to treatment-resistant and highly aggressive disease. Metastasis-potential and treatment-resistance is thought to rely on increased plasticity of the cancer cells-a mechanism whereby cancer cells alter their identity to adapt to changing environments or therapeutic pressures to create cellular heterogeneity. To understand the molecular basis of this plasticity, genomic studies have uncovered genetic variants to capture clonal...

  • Niraparib Maintenance Therapy in Patients with Platinum-Sensitive Recurrent Ovarian Cancer: Real-World Experience at Hospitals in Spain Fri, 24 Jan 2025 06:00:00 -0500
    CONCLUSION: This large real-world analysis corroborates the tolerability and activity of niraparib maintenance for platinum-sensitive recurrent OC and economic savings.

  • Targeting breast cancer stem cells in ER-positive breast cancer by repurposing the benzoporphyrin derivative verteporfin as a YAP/TAZ small molecule inhibitor Fri, 24 Jan 2025 06:00:00 -0500
    CONCLUSION: This study highlights the potential of repurposing verteporfin, an FDA-approved drug originally formulated for age-related macular degeneration, as a therapeutic agent for targeting YAP/TAZ-mediated stemness and redox balance in brCSCs, thereby reducing their viability in ER-positive breast cancers.

  • Bacopa monnieri Extract Diminish Hypoxia-Induced Anxiety by Regulating HIF-1α Signaling and Enhancing the Antioxidant Defense System in Hippocampus Fri, 24 Jan 2025 06:00:00 -0500
    Hypoxia is a significant stressor, and stabilized hypoxia-inducible factor-1α (HIF-1α) regulates the expression of numerous genes, leading to various biochemical, molecular, physiological and genomic changes. The body's oxygen-sensing system activates gene expression to protect brain tissues from hypoxia. Gamma-aminobutyric acid, an inhibitory neurotransmitter, regulates brain excitability during hypoxia through the activation of HIF-1 α. Herbal medicines have been widely used for managing...

  • Marine natural products as a source of novel anticancer drugs: an updated review (2019-2023) Fri, 24 Jan 2025 06:00:00 -0500
    Marine natural products have long been recognized as a vast and diverse source of bioactive compounds with potential therapeutic applications, particularly in oncology. This review provides an updated overview of the significant advances made in the discovery and development of marine-derived anticancer drugs between 2019 and 2023. With a focus on recent research findings, the review explores the rich biodiversity of marine organisms, including sponges, corals, algae, and microorganisms, which...

  • Identification of a metabolic-immune signature associated with prognosis in colon cancer and exploration of potential predictive efficacy of immunotherapy response Fri, 24 Jan 2025 06:00:00 -0500
    The role of metabolic reprogramming of the tumor immune microenvironment in cancer development and immune escape has increasingly attracted attention. However, the predictive value of differences in metabolism-immune microenvironment on the prognosis of colon cancer (CC) and the response to immunotherapy have not been elucidated. The aim of this study was to investigate changes in metabolism and immune profile of CC and to identify a reliable signature for predicting prognosis and therapeutic...

  • Drosophila modeling to identify causative genes and reveal the underlying molecular mechanisms for primary ovarian insufficiency Fri, 24 Jan 2025 06:00:00 -0500
    Primary ovarian insufficiency (POI) is a disease defined as a reduction in ovarian function under the age of 40 and represents the main cause of female infertility. In recent years, many genetic mutations associated with POI have been identified using high-throughput sequencing technology. However, one big challenge today is to determine the disease-causing gene associations through functional assessment. Here, we develop a Drosophila model to study the POI-associated genes and provide in vivo...

  • Nivolumab and rituximab in treatment naive follicular lymphoma: the phase II '1st FLOR' study Fri, 24 Jan 2025 06:00:00 -0500
    Follicular lymphoma (FL) outcomes are heavily influenced by host immune activity with immune anti-tumor activity mitigated by PD-1/PD-L1 pathway engagement. Combination CD20-directed therapy plus PD-1 inhibition (PD-1i) increases T-cell tumor killing and NK-cell antibody-dependent cell cytotoxicity (ADCC). Mounting evidence supports immune-priming using PD-1i before cancer-directed agents. Our multicentre, open-label, phase II 1st FLOR study (NCT03245021) enrolled 39 previously-untreated...