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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.

  • Getting closer to hemophilia gene therapy for all? Mon, 14 Jul 2025 06:00:00 -0400
    No abstract

  • Inflammatory Pathways in Residual Asthma Exacerbations Among Mepolizumab-Treated Urban Children: A Secondary Analysis of a Randomized Clinical Trial Mon, 14 Jul 2025 06:00:00 -0400
    CONCLUSIONS AND RELEVANCE: The study's findings implicate multiple alternative inflammatory pathways associated with the epithelium and macrophages, as well as mucus hypersecretion, as mechanisms of residual acute exacerbations in children receiving mepolizumab. Further, they indicate that multiple distinct inflammatory axes can independently contribute to asthma exacerbations.

  • Whole genome sequencing analysis of Candida glabrata isolates collected from patients with selected drug-resistant candidiasis hospitalized in Eastern Poland Mon, 14 Jul 2025 06:00:00 -0400
    The epidemiology data for candidiasis indicate an increase in Candida glabrata infections. Moreover, several reports have shown an increasing number of drug-resistant cases of these infections. The source of drug resistance can often be traced to genetic mutations in genes related to a drug's mechanism of action. Therefore, we conducted whole genome sequencing of several drug-resistant isolates of Candida glabrata collected from patients hospitalized in Eastern Poland to assess whether mutations...

  • S100A9 promotes resistance to anti-PD-1 immunotherapy in hepatocellular carcinoma by degrading PARP1 and activating the STAT3/PD-L1 pathway Mon, 14 Jul 2025 06:00:00 -0400
    CONCLUSIONS: Our study reveals that S100A9 facilitates immune evasion in HCC by enhancing PARP1 ubiquitination, STAT3 phosphorylation, and PD-L1 expression. Furthermore, combining S100A9 inhibitors with anti-PD-1 antibodies markedly enhances the therapeutic efficacy of ICIs in HCC. These findings highlight S100A9 as a potential therapeutic target for overcoming resistance to immunotherapy in HCC.

  • Genetic variants linked to type 2 diabetes in CDKN1B and TCF7L2 influence survival outcomes in metastatic colorectal cancer Mon, 14 Jul 2025 06:00:00 -0400
    Evidence suggests that metastatic colorectal cancer patients with type 2 diabetes (T2D) experience a poorer prognosis in contrast to their non-diabetic counterparts. Considering the multifactorial genetic nature of colon cancer development, we examined whether gene polymorphisms associated with T2D could affect the clinical outcome of metastatic colon cancer. Using in silico analysis, we evaluated gene variants linked to both T2D and colon cancer utilizing data from The Cancer Genome Atlas...

  • The Role of Oxytocin and Oxytocin Gene Receptor Methylation During Withdrawal Therapy in Males With Alcohol Use Disorder Mon, 14 Jul 2025 06:00:00 -0400
    Oxytocin is a promising therapeutic target in the treatment of alcohol use disorder (AUD). However, many studies report contradicting evidence regarding its effect on drug craving, relapse risk and withdrawal symptoms. Epigenetic regulation of the oxytocin and oxytocin receptor (OXTR) gene is altered in several mental disorders and influences social behaviour, often depending on the underlying sex. Evidence suggests that altered promoter methylation could result in oxytocin and OXTR expression...

  • Genetics of short stature Mon, 14 Jul 2025 06:00:00 -0400
    PURPOSE OF REVIEW: This review highlights recent genetic discoveries and therapeutic advancements in evaluating and managing children with short stature. With an increasing diagnostic yield from genetic testing and the emergence of genotype-specific treatments, a comprehensive update is necessary for timely application in clinical practice.

  • Therapeutic liposomes synergize with active molecules to enhance targeted therapy Mon, 14 Jul 2025 06:00:00 -0400
    Liposomes composed of phospholipids (PLs) either alone or with an active molecule, can reveal a significant potential in the improvement of severe disorders such as Alzheimer's disease, osteoporosis, and inflammatory conditions. For instance, PLs exhibit anti-inflammatory, antioxidant, neuroprotective, and osteogenic properties in pathological conditions which accelerate the therapeutic effect of the drugs. These pharmacological properties can be modulated by the type and dose of PLs or liposome...

  • Amnioreduction as a therapeutic strategy for MAGED2-related Bartter syndrome: prolonging gestation and improving outcomes through genetic-guided prenatal management Mon, 14 Jul 2025 06:00:00 -0400
    CONCLUSION: Amnioreduction mitigates polyhydramnios-driven preterm birth in MAGED2-related Bartter syndrome, enabling safer gestational prolongation. Integration of WES for rapid genetic diagnosis and multidisciplinary care optimizes prenatal management. These findings support amnioreduction as a critical intervention for this high-risk population, emphasizing early genetic testing, and proactive fetal therapy.

  • Teaching an old vector new tricks: the surprising versatility of AAV vaccines Mon, 14 Jul 2025 06:00:00 -0400
    Adeno-associated virus (AAV) has proven its clinical efficacy in the realm of gene therapy, resulting in seven FDA-approved gene therapies. While AAV gene transfer research has predominantly focused on its utility in monogenic disorders, AAV vectors have been used as a platform for vaccines in over 50 preclinical studies over the last 25 years. Recombinant AAV-based vaccines have demonstrated induction and durability of antigen-specific adaptive immune responses in a variety of preclinical...

  • Mechanistic insights into JSS1_004-mediated antagonism of the DndBCDE-FGH restriction system and engineering applications Mon, 14 Jul 2025 06:00:00 -0400
    The bacterial DNA phosphorothioate (PT) modification system is orchestrated by the DndCDE enzymatic complex. This system collaborates with restriction components DndF, DndG, and DndH to establish a widespread prokaryotic anti-phage defense network. In the evolutionary arms race, phages such as JSS1 have evolved counter strategies, including the expression of the JSS1_004 protein, to subvert PT-mediated host immunity. Although the N-terminal kinase domain of JSS1_004 is known to inhibit DndFGH...

  • Bilateral Sequential Herpes Simplex Type 2 Panophthalmitis in an Adult with a Toll-Like Receptor 4 Mutation Mon, 14 Jul 2025 06:00:00 -0400
    CONCLUSION: HSV-2 panophthalmitis is rare and visually destructive, and we present the first reported case of bilateral disease. We hypothesise that TLR-4 mutation, not previously reported in association with HSV ocular inflammation, may have contributed to excessive inflammation and severe ocular damage. We suggest comprehensive immunological evaluation in all patients with severe viral intraocular inflammation. Prompt diagnosis with PCR and immediate high-dose antiviral therapy is critical and...

  • Identification of the important role of CA9 in immune infiltration and prognosis in cervical cancer Mon, 14 Jul 2025 06:00:00 -0400
    CONCLUSION: Our study indicated that CA9 was upregulated in CC and closely related to tumor immune microenvironment, thereby becoming an important prognostic factor for CC. This study highlights CA9 as a potential diagnostic and therapeutic target for the disease.

  • A Case of Idiopathic Follicular Mucinosis Treated Successfully with Cyclosporine Mon, 14 Jul 2025 06:00:00 -0400
    Follicular mucinosis (FM) is a disease histopathologically characterized by mucin deposition in the hair follicles; the main symptoms are papular erythema, papular pilaris, and hair loss in hairy areas (1). FM is classified as either idiopathic or secondary based on underlying diseases or complications. In recurrent or refractory cases, the disease can transform into mycosis fungoides. Treatment includes local or systemic corticosteroids, dapsone, indomethacin, interferon, hydroxychloroquine,...

  • Nanoplatform-Enabled Genetic Interventions for Central Nervous System Disorders: Advances in Delivery Strategies and Therapeutic Potential Mon, 14 Jul 2025 06:00:00 -0400
    Central nervous system (CNS) disorders are driven by complex genetic and epigenetic factors. While gene-based interventions (siRNA, mRNA, CRISPR systems, etc.) hold transformative potential, their clinical application is severely constrained by inefficient delivery, especially across the blood-brain barrier. Nanocarriers have emerged as transformative platforms that overcome these challenges by enabling efficient BBB penetration while ensuring precise biodistribution control and enhanced...

  • Concomitant Systemic Autoinflammatory Diseases: Diagnostic and therapeutic challenges Mon, 14 Jul 2025 06:00:00 -0400
    Neonatal-onset multisystem inflammatory disease (NOMID) and familial Mediterranean fever (FMF) are distinct entities within the expanding spectrum of systemic autoinflammatory diseases (SAIDs). We report a 3-month-old infant who presented with recurrent fever, urticarial rash, and polyarthritis. After excluding other causes, anakinra was initiated based on clinical suspicion of NOMID. Despite treatment optimisation, she continued to experience disease flares. An initial autoinflammatory panel...

  • Research Progress of Epigenetic Modifications in Myopia Mon, 14 Jul 2025 06:00:00 -0400
    Myopia, also known as nearsightedness, refers to a refractive error of the eye that causes parallel rays of light to focus in front of the retina, affecting distance vision. High myopia significantly increases the risk of pathological myopia, leading to severe complications and an increased likelihood of myopia-related eye diseases. In recent decades, the incidence of myopia has continued to rise, posing significant social and human health issues. The complex interplay between genetic and...

  • Cancer-Associated PIK3R1 Genetic Aberrations and Precision Medicine Mon, 14 Jul 2025 06:00:00 -0400
    The PIK3R1 gene encodes the class IA PI3K regulatory subunit p85α, which is frequently altered in cancer. PIK3R1 functions as a tumor suppressor by stabilizing and inhibiting the catalytic activity of p110, and it directly binds to and enhances the activity of the PTEN lipid phosphatase. Aberrations in the PIK3R1 gene are associated with poor prognosis in cancer; available data underscore the significant role of PIK3R1 mutations in mediating tumorigenesis by promoting the signaling of the...

  • Ginsenoside Rh7 affects β-catenin nuclear translocation by inhibiting SHCBP1 expression, thereby inhibiting epithelial-mesenchymal transition in gastric cancer cells Mon, 14 Jul 2025 06:00:00 -0400
    Background: Ginsenoside Rh7 is a bioactive compound with anticancer properties. This investigation was conducted to analyze the anticancer effects of ginsenoside Rh7 and its underlying molecular mechanisms in gastric cancer (GC) cells. Methods: The key gene module associated with GC was identified through weighted gene co-expression network analysis (WGCNA) of the GSE118897 dataset. Differentially expressed genes (DEGs) were examined in The Cancer Genome Atlas-Stomach Adenocarcinoma (TCGA-STAD)...

  • Actin-Like Protein 6A as an Oncogene and Therapeutic Target in Cancer Mon, 14 Jul 2025 06:00:00 -0400
    ACTL6A, a core subunit of the SWI/SNF chromatin remodeling complex, has emerged as a critical oncogenic driver across multiple malignancies. Recent studies reveal that aberrant ACTL6A overexpression promotes tumor initiation, progression, and metastasis by orchestrating chromatin remodeling, transcriptional reprogramming, and crosstalk with key signaling pathways (e.g., Hippo/YAP, Notch, and PI3K/AKT). This review systematically synthesizes evidence from in vitro, in vivo, and clinical studies...