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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 multifunction murine Col4a1 allele reveals potential gene therapy parameters for Gould syndrome Fri, 25 Apr 2025 06:00:00 -0400
    Basement membranes (BMs) are specialized extracellular matrix (ECM) structures essential for organ morphogenesis, architecture, and function. BM composition and properties vary between tissues, developmental stages, and disease states, and there is only a rudimentary understanding of BM dynamics. Here, we introduce a versatile mouse model carrying a multifunctional dual-color fluorescence tagged allele with knockout potential for the fundamental BM component type IV collagen alpha 1 (COL4A1)....

  • Lower Expression of SARS-CoV-2 Host Cell Entry Genes in the Intestinal Mucosa of IBD Patients With Quiescent or Mildly Active Disease Fri, 25 Apr 2025 06:00:00 -0400
    CONCLUSIONS: Overall, our study indicates that the presence of higher basal circulating levels of MBL in CD patients and the decreased intestinal mucosa expression of the SARS-CoV-2 receptor ACE2 and the host cell priming proteases TMPRSS2 and TMPRSS4 in both CD and UC patients may reduce COVID-19 risk, underscoring the potential protective role of these biomarkers in IBD populations against SARS-CoV-2 infection.

  • Effects of two different peptides on pentylenetetrazole-induced seizures in larval zebrafish Fri, 25 Apr 2025 06:00:00 -0400
    Epilepsy is a common and severe neurological disease characterized by spontaneous and recurrent seizures. Although anti-seizure treatments are effective for most patients, approximately 30% remain pharmacoresistant. Moreover, uncontrolled seizures are associated with increased health risks and shortened life expectancy in individuals with refractory epilepsy. Preclinical studies play a crucial role in drug discovery, and the zebrafish (Danio rerio) have been successfully employed for this...

  • Prognostic significance of KRAS, NRAS, BRAF, and PIK3CA mutations in stage II/III colorectal cancer: A retrospective study and meta-analysis Fri, 25 Apr 2025 06:00:00 -0400
    The prognostic significance of KRAS and BRAF mutations is well-established in metastatic colorectal cancer (CRC) but remains uncertain in early-stage tumors. This study retrospectively analyzed 47 stage II/III CRC patients undergoing curative surgery to assess the association of mutations in KRAS, NRAS, BRAF, and PIK3CA with overall survival (OS) and disease-free survival (DFS). Additionally, a meta-analysis was conducted to validate the prognostic relevance of these gene mutations. We included...

  • Decorin the anti-fibrotic proteoglycan and its progression in therapy Fri, 25 Apr 2025 06:00:00 -0400
    Fibrosis, which underlies numerous chronic diseases, is characterized by excessive extracellular matrix (ECM) accumulation, resulting in disrupted tissue architecture. Decorin, a small leucine-rich proteoglycan synthesized primarily by fibroblasts and myoblasts, has emerged as a potent antifibrotic agent mainly by inhibiting TGF-β, which is a major driver of fibrosis in various tissues and organs such as the heart, eyes, skin, liver, muscle, etc. Numerous therapeutic applications of decorin...

  • A novel cancer-associated fibroblast-related gene signature for predicting diffuse large B cell lymphoma prognosis using weighted gene co-expression network analysis and machine learning Fri, 25 Apr 2025 06:00:00 -0400
    ObjectiveOur objective was to investigate a novel cancer-associated fibroblast-related gene signature for predicting clinical outcomes in patients with diffuse large B cell lymphoma.MethodsThe cancer-associated fibroblast-related module genes were identified from Gene Expression Omnibus datasets using weighted gene co-expression network analysis in our retrospective study. Least Absolute Shrinkage and Selection Operator Cox regression was applied to screen a minimal set of genes and construct a...

  • Recessively inherited protein C deficiency in two sisters: intracerebral hemorrhage and coagulopathy complications Fri, 25 Apr 2025 06:00:00 -0400
    CONCLUSION: Homozygous protein C deficiency can lead to severe neonatal coagulopathy, necessitating early diagnosis and aggressive anticoagulant therapy. This case underscores the importance of multidisciplinary management and genetic screening in affected families.

  • A Dual-Interaction Supramolecular Hydrogel System for siRNA Delivery to Enhance Endometrial Receptivity in Stem Cell Therapy Fri, 25 Apr 2025 06:00:00 -0400
    Endometrial receptivity is crucial for embryo implantation success. Stem cell therapy shows promise for improving endometrial health. We developed a supramolecular hydrogel scaffold based on stereocomplexed triblock copolymers (MPEG-(sc-PLA)-PEI) and α-cyclodextrin for codelivering menstrual blood-derived endometrial stem cells (MenSCs) and siRNA targeting DNA methyl transferase 1 (DNMT1), enabling RNAi-mediated gene silencing to improve endometrial receptivity. The resulting...

  • From non-coding RNAs to cancer regulators: The fascinating world of micropeptides Fri, 25 Apr 2025 06:00:00 -0400
    Micropeptides are commonly identified as peptides encoded by non-coding RNAs (ncRNAs). In the short open reading frame (sORF) of ncRNAs, there is a base sequence encoding functional micropeptides, which is of great significance in the biological field. Recently, micropeptides regulate diverse processes, including mitochondrial metabolism, calcium transport, mRNA splicing, signal transduction, myocyte fusion, and cellular senescence, regulating the homeostasis of the internal environment and...

  • Combination Immunotherapy for Mucosal Melanoma: Molecular Mechanism, Research Status, and Future Directions Fri, 25 Apr 2025 06:00:00 -0400
    Mucosal melanoma is a rare and aggressive subtype of melanoma, accounting for 1%-2% of new cases in the United States in 2023, and 20%-30% in China and other Asian countries. Its origin is often occult, with the lack of early clinical features, the absence of actionable driver mutations, and poor response to immunotherapy, all contributing to its poor prognosis. The rarity of this subtype leads to limited awareness and interventions. Furthermore, due to its immune evasion mechanisms, mucosal...

  • Results of therapy in children diagnosed with severe traumatic brain injury Fri, 25 Apr 2025 06:00:00 -0400
    The aim of this research is to enhance therapeutic outcomes in children diagnosed with severe craniocerebral trauma by evaluating the contributing factors involved in cerebral injury. The investigation focused on evaluating the impact of infusion therapy on the improvement of pediatric patients' conditions with SCCT, particularly by assessing hydrobalance indicators. Adaptive changes associated with the humoral stress response were evaluated through alterations in hormone concentrations...

  • Obesity: pathophysiology and therapeutic interventions Fri, 25 Apr 2025 06:00:00 -0400
    Over the past few decades, obesity has transitioned from a localized health concern to a pressing global public health crisis affecting over 650 million adults globally, as documented by WHO epidemiological surveys. As a chronic metabolic disorder characterized by pathological adipose tissue expansion, chronic inflammation, and neuroendocrine dysregulation that disrupts systemic homeostasis and impairs physiological functions, obesity is rarely an isolated condition; rather, it is frequently...

  • CA1 induced dental follicle stem cells co-culture with dental pulp stem cells and loaded three-dimensional printed PCL/β-TCP scaffold: a novel strategy for alveolar cleft bone regeneration Fri, 25 Apr 2025 06:00:00 -0400
    CONCLUSION: A strategy based on a novel osteogenic gene CA1 and dental-derived mesenchymal stem cells co-culture is applied to the alveolar cleft, providing a novel idea for the application of bone tissue engineering in alveolar cleft bone grafting.

  • Anodal direct current stimulation of the auditory cortex at the onset of presbycusis delays cortical aging Fri, 25 Apr 2025 06:00:00 -0400
    Presbycusis or age-related hearing loss (ARHL) affects millions of people worldwide, increasing their risk of cognitive decline and poor quality of life. However, ARHL remains an irreversible condition due to our inability to induce inner-ear hair cell regeneration. Nevertheless, multisession epidural stimulation of the auditory cortex (AC) at the onset of ARHL prevents hearing threshold elevation in naturally aging Wistar rats. Accordingly, we hypothesized that anodal direct current (DC)...

  • Treatment outcomes of BTK inhibitors and venetoclax with or without anti-CD20 monoclonal antibody in relapsed or refractory mantle cell lymphoma Fri, 25 Apr 2025 06:00:00 -0400
    To characterise the effectiveness of Bruton tyrosine kinase inhibitors with venetoclax in patients with refractory or relapsed mantle cell lymphoma, with or without the addition of an anti-CD20 antibody. Progression-free and overall survival were estimated for forty-nine patients treated with Bruton tyrosine kinase inhibitors and venetoclax (200 mg, daily) or in combination with an anti-CD20 monoclonal antibody between June 2018 and February 2022 in China. The median number of treatment lines...

  • CHD1 dysregulation in cancer: bridging chromatin instability, therapy resistance, and immune evasion Fri, 25 Apr 2025 06:00:00 -0400
    Chromodomain-Helicase-DNA-binding protein 1 (CHD1) is a central regulator of chromatin dynamics, profoundly influencing gene expression, DNA repair, and genomic stability. This review critically explores CHD1's role in cancer biology, emphasizing its complex, context-dependent functions. In prostate cancer, CHD1 acts as both a tumour suppressor and a facilitator of neuroendocrine differentiation, with its loss linked to aggressive phenotypes, resistance to androgen receptor therapies, and...

  • Applying nanotechnology to glaucoma therapy: past, present, and future Fri, 25 Apr 2025 06:00:00 -0400
    No abstract

  • Magnetic-Guided Delivery of Antisense Oligonucleotides for Targeted Transduction in Multiple Retinal Explant and Organoid Models Fri, 25 Apr 2025 06:00:00 -0400
    Antisense oligonucleotide (ASO) therapy holds promise in gene therapy but faces challenges due to poor delivery efficiency and limited evaluation models. This investigation employs magnetic nanoparticles (MNPs) to augment the delivery efficiency of ASOs. It assesses their distribution and therapeutic efficacy across various models, including retinal explants from mice and macaques or human retinal and inner ear organoids. Retinal explants from both mice and monkeys are methodically arranged to...

  • Analysis of Processing, Post-Maturation, and By-Products of shRNA in Gene and Cell Therapy Applications Fri, 25 Apr 2025 06:00:00 -0400
    Short hairpin RNAs (shRNAs) are potent tools for gene silencing, offering therapeutic potential for gene and cell therapy applications. However, their efficacy and safety depend on precise processing by the RNA interference machinery and the generation of minimal by-products. In this protocol, we describe how to systematically analyze the processing of therapeutic small RNAs by DROSHA and DICER1 and their incorporation into functional AGO complexes. Using standard small RNA sequencing and...

  • Genetic Predictors of Paxlovid Treatment Response: The Role of IFNAR2, OAS1, OAS3, and ACE2 in COVID-19 Clinical Course Fri, 25 Apr 2025 06:00:00 -0400
    Background: This study investigated the role of genetic polymorphisms in IFNAR2, OAS1, OAS3, and ACE2 as predictors of Paxlovid treatment response, specifically examining their influence on the clinical course and laboratory parameters of COVID-19 patients. Methods: We analyzed the impact of polymorphisms in genes associated with the interferon pathway (IFNAR2 rs2236757), antiviral response (OAS1 rs10774671, OAS3 rs10735079), and viral entry (ACE2 rs2074192) in individuals treated with Paxlovid....