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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.

  • Pancreatic carcinosarcoma: a rare type of pancreatic neoplasia with long-term survival Wed, 19 Mar 2025 06:00:00 -0400
    A woman in her 60s presented with persistent abdominal pain and jaundice. She had a history of BRCA1 gene mutation and was previously treated for triple-negative early breast cancer. Abdominal ultrasound and CT scan of the abdomen revealed a mass around the pancreatic head. Magnetic resonance cholangiopancreatography showed a dilated common bile duct and a normal pancreatic duct. Liver function improved after endoscopic retrograde cholangiopancreatography and biliary stent placement. Staging CT...

  • Cardiac MRI study of adverse events in patients treated with immune checkpoint inhibitors: a prospective cohort study of cardiac adverse events Wed, 19 Mar 2025 06:00:00 -0400
    Immune checkpoint inhibitors (ICIs) revolutionized cancer therapy, yet require management of immune-related adverse events (irAEs). Fulminant myocarditis is a rare irAE, but lower-severity cardiac events are being reported more frequently, leading to an unmet need for irAE prevention, early diagnosis, and treatment, especially for long-life-expectancy patients. We recruited 57 patients, stratified according to therapy regime (monotherapy (30%) or combination (33%) cohort) or history of cardiac...

  • Comparative Uptake of Statins by Hepatic Organic Anion Transporting Polypeptides Wed, 19 Mar 2025 06:00:00 -0400
    Uptake into hepatocytes by the organic anion transporting polypeptides (OATP) 1B1, 1B3, and 2B1 can form a rate-determining step in drug clearance. While several lipid-lowering statin drugs are substrates of the hepatic OATPs, the literature statin uptake profiles form a wide range with values obtained from different laboratories and systems. Therefore, this study aimed to characterize hepatic OATP-mediated transport of statins in a single-laboratory experimental setup, allowing comparison...

  • Codon usage bias and nucleotide bias are not influenced by the 5' flanking but by 3' and intronic region composition in SCID-associated genes Wed, 19 Mar 2025 06:00:00 -0400
    Compositional constraints, selectional and mutational forces, nucleotide skews, RNA folding free energy, gene expression, protein properties, and differential expression are a few attributes that define the molecular signatures of any gene. The absence of information regarding these attributes for genes associated with severe combined immunodeficiency disorder (SCID) prompted us to take up this study. The compositional bias influenced codon bias. Overall percent T composition is the lowest among...

  • Unravelling the epigenetic based mechanism in discovery of anticancer phytomedicine: Evidence based studies Wed, 19 Mar 2025 06:00:00 -0400
    Epigenetic mechanisms play a crucial role in the normal development and maintenance of tissue-specific gene expression patterns in mammals. Disruption of these processes can result in changes to gene function and the transformation of cells into a malignant state. Cancer is characterized by widespread alterations in the epigenetic landscape, revealing that it involves not only genetic mutations but also epigenetic abnormalities. Recent progress in the field of cancer epigenetics has demonstrated...

  • Safety and efficacy of gene therapy for RAG1-deficient SCID Wed, 19 Mar 2025 06:00:00 -0400
    No abstract

  • Safety and efficacy of combined treatment with tumor-infiltrating lymphocytes and oncolytic adenovirus TILT-123 in metastatic melanoma Wed, 19 Mar 2025 06:00:00 -0400
    Tumor-infiltrating lymphocytes (TILs) are effective in the treatment of metastatic melanoma (MM), but toxicity limits its application. TILT-123 (igrelimogene litadenorepvec) is an oncolytic adenovirus producing interleukin-2 (IL-2) and tumor necrosis factor (TNF) upon replication. In this phase 1 trial, 17 patients with metastatic checkpoint inhibitor-resistant melanoma are treated with TILT-123 and TILs without preconditioning chemotherapy or postconditioning IL-2. The treatment is safe and...

  • Targeting pathogenic interferon-stimulated gene RSAD2 improves pregnancy outcomes in systemic lupus erythematosus models Wed, 19 Mar 2025 06:00:00 -0400
    Excessive type I interferons during pregnancy are associated with poor pregnancy outcomes. Ding et al.¹ demonstrate the pathogenic effects of the interferon-stimulated gene RSAD2 that drives adverse pregnancy outcomes through placental lipid accumulation, and further identify a promising therapeutic candidate.

  • ALK-EML4 Fusion in Small Cell Lung Cancer: Clinical and Molecular Insights From a Rare Case Wed, 19 Mar 2025 06:00:00 -0400
    CONCLUSIONS: This case highlights the rare occurrence of ALK fusion in SCLC, which may offer insights into potential targeted treatments. The patient's progression despite standard therapies suggests the need for further investigation into ALK inhibitors for SCLC patients with such mutations. Clinicians should consider NGS testing as a valuable diagnostic tool for identifying genetic alterations in SCLC, which could guide personalized treatment strategies and improve outcomes.

  • POLR3-Related Leukodystrophy: A Qualitative Study on Parents' Experiences With the Health Care System Wed, 19 Mar 2025 06:00:00 -0400
    CONCLUSIONS: This study will help better inform health care providers, administrators, and policymakers to expand and improve access to quality care for patients with POLR3-HLD and their families. These conclusions may also be generalizable to other rare diseases.

  • Diverse Acinetobacter species and Plasmid-Driven spread of carbapenem resistance in pharmaceutical settings in China Wed, 19 Mar 2025 06:00:00 -0400
    Acinetobacter species have emerged as a significant public health concern due to their remarkable capacity to acquire antimicrobial resistance. Environmental reservoirs play a crucial role in spreading antimicrobial resistance genes and potentially pathogenic bacteria to clinical settings. However, most studies on nonhuman isolates have focused on a limited sample size. Comprehensive population sampling on One Health principles is essential to monitor the antibiotic resistome and virulome in...

  • Role of allo-HCT in "nonclassical" MPNs and MDS/MPNs: recommendations from the PH&G Committee and the CMWP of the EBMT Wed, 19 Mar 2025 06:00:00 -0400
    'Non-classical' Myeloproliferative Neoplasms (MPNs) and Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPNs) represent a heterogeneous group of malignancies characterized by a wide range of clinical manifestations. Unlike classical MPNs, there is no standardized management approach for these conditions, particularly concerning the indications for and management of allogeneic hematopoietic cell transplantation (HCT). To address this gap, the EBMT Practice Harmonization and Guidelines (PH&G)...

  • Fertility Preservation in People With Cancer: ASCO Guideline Update Wed, 19 Mar 2025 06:00:00 -0400
    PURPOSE: To provide updated fertility preservation (FP) recommendations for people with cancer.

  • Interneuron-specific dual-AAV SCN1A gene replacement corrects epileptic phenotypes in mouse models of Dravet syndrome Wed, 19 Mar 2025 06:00:00 -0400
    Dravet syndrome (DS) is a severe developmental epileptic encephalopathy marked by treatment-resistant seizures, developmental delay, intellectual disability, motor deficits, and a 10 to 20% rate of premature death. Most patients with DS harbor loss-of-function mutations in one copy of SCN1A, which encodes the voltage-gated sodium channel (Na(V))1.1 alpha subunit and has been associated with inhibitory neuron dysfunction. Here, we generated a split-intein form of SCN1A and used a dual-vector...

  • T cell receptor precision editing of regulatory T cells for celiac disease Wed, 19 Mar 2025 06:00:00 -0400
    Celiac disease, a gluten-sensitive enteropathy, demonstrates a strong human leukocyte antigen (HLA) association, with more than 90% of patients carrying the HLA-DQ2.5 allotype. No therapy is available for the condition except for a lifelong gluten-free diet. To address this gap, we explored the therapeutic potential of regulatory T cells (T(regs)). By orthotopic replacement of T cell receptors (TCRs) through homology-directed repair, we generated gluten-reactive HLA-DQ2.5-restricted CD4^(+)...

  • Palmoplantar epidermal differentiation disorders: a new classification towards pathogenesis-based therapy Wed, 19 Mar 2025 06:00:00 -0400
    More than 50 palmoplantar epidermal differentiation disorders (pEDDs) have been reported in the literature. Various descriptors have been used to name these conditions including clinical features often designated as acronyms or evocative terms, names of clinicians and of geographical locations. Not only has this nomenclature been criticized for its lack of methodological consistency, it has progressively lost its clinical relevance. Indeed, the advent of a wide range of pathogenesis-targeting...

  • PPDPF preserves integrity of proximal tubule by modulating NMNAT activity in chronic kidney diseases Wed, 19 Mar 2025 06:00:00 -0400
    Genome-wide association studies (GWAS) have identified loci associated with kidney diseases, but the causal variants, genes, and pathways involved remain elusive. Here, we identified a kidney disease gene called pancreatic progenitor cell differentiation and proliferation factor (PPDPF) through integrating GWAS on kidney function and multiomic analysis. PPDPF was predominantly expressed in healthy proximal tubules of human and mouse kidneys via single-cell analysis. Further investigations...

  • Differential gene expression patterns in Niemann-Pick Type C and Tay-Sachs diseases: Implications for neurodegenerative mechanisms Wed, 19 Mar 2025 06:00:00 -0400
    Lysosomal storage disorders (LSDs) are a group of rare genetic conditions characterized by the impaired function of enzymes responsible for lipid digestion. Among these LSDs, Tay-Sachs disease (TSD) and Niemann-Pick type C (NPC) may share a common gene expression profile. In this study, we conducted a bioinformatics analysis to explore the gene expression profile overlap between TSD and NPC. Analyses were performed on RNA-seq datasets for both TSD and NPC from the Gene Expression Omnibus (GEO)...

  • The IBEX Knowledge-Base: A central resource for multiplexed imaging techniques Wed, 19 Mar 2025 06:00:00 -0400
    Multiplexed imaging is a powerful approach in spatial biology, although it is complex, expensive and labor-intensive. Here, we present the IBEX Knowledge-Base, a central resource for reagents, protocols and more, to enhance knowledge sharing, optimization and innovation of spatial proteomics techniques.

  • AGO2 mediates immunotherapy failure via suppressing tumor IFN-gamma response-dependent CD8+ T cell immunity Wed, 19 Mar 2025 06:00:00 -0400
    Interferon-gamma (IFN-γ), a cytokine essential for activating cellular immune responses, plays a crucial role in cancer immunosurveillance and the clinical success of immune checkpoint blockade therapy. In this study, we show that Argonaute 2 (AGO2), a key mediator in small RNA-guided gene regulation, inversely correlates with tumor responsiveness to IFN-γ and the efficacy of immunotherapy. Mechanistically, IFN-γ upregulates miR-1246 expression in tumor cells, enhancing its interaction with...