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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.

  • High clinical utility of long-read sequencing for precise diagnosis of congenital adrenal hyperplasia in 322 probands Tue, 14 Jan 2025 06:00:00 -0500
    CONCLUSION: T-LRS has higher resolution and reduced cost than control method with accurate diagnosis. The clinical utility of L-LRS could help to provide precision therapy to CAH patients, advance the life-long management of this complex disease and promote our understanding of CAH.

  • Targeting of the G9a, DNMT1 and UHRF1 epigenetic complex as an effective strategy against pancreatic ductal adenocarcinoma Tue, 14 Jan 2025 06:00:00 -0500
    CONCLUSIONS: The combined overexpression of DNMT1, G9a, and UHRF1 in PDAC is a strong predictor of poor prognosis. CM272, by targeting this epigenetic complex, shows promising therapeutic potential by inducing apoptosis, reprogramming metabolic pathways, and enhancing immune responses. The combination of CM272 with immunotherapy offers a novel, effective treatment strategy for PDAC.

  • Crosstalk between non-coding RNAs and programmed cell death in colorectal cancer: implications for targeted therapy Tue, 14 Jan 2025 06:00:00 -0500
    CONCLUSION: Understanding the involvement of ncRNAs in PCD regulation offers new insights into CRC biology. The targeted modulation of ncRNA-PCD interactions presents promising avenues for personalized cancer treatment, which may improve patient outcomes by enhancing therapeutic effectiveness and reducing resistance.

  • Construction of a prognostic model for gastric cancer based on immune infiltration and microenvironment, and exploration of MEF2C gene function Tue, 14 Jan 2025 06:00:00 -0500
    CONCLUSIONS: Our prognostic model can effectively predict outcomes and facilitate stratification GC patients, offering valuable insights for clinical decision-making. The identified transcription factor MEF2C can serve as a biomarker for assessing the efficacy of immunotherapy for GC.

  • Predictive value of dendritic cell-related genes for prognosis and immunotherapy response in lung adenocarcinoma Tue, 14 Jan 2025 06:00:00 -0500
    CONCLUSION: We have innovatively established a deep learning-based prediction model, DCRGS, for the prediction of the prognosis of patients with LUAD. The model possesses a strong prognostic prediction performance with high accuracy and sensitivity and could be clinically useful to guide the management of LUAD. Furthermore, the findings of this study could provide an important reference for individualized clinical treatment and prognostic prediction of patients with LUAD.

  • Identifying effective immune biomarkers in alopecia areata diagnosis based on machine learning methods Tue, 14 Jan 2025 06:00:00 -0500
    CONCLUSION: Overall, the study provides a novel approach for the diagnosis, prevention, and treatment of AA. The findings could potentially lead to the development of targeted therapies for AA based on the identified hub genes. The study also highlights the potential of machine learning and bioinformatics analysis in identifying new biomarkers for autoimmune diseases.

  • Synergy of zinc oxide nanoparticles to losartan attenuates kidney injury induced by unilateral ureteral obstruction through modulation of the TNF-α/IL6 and BAX/BCL2 signaling pathways Tue, 14 Jan 2025 06:00:00 -0500
    AIM: Although the relief of ureteral obstruction seems to be a radical treatment for obstructive uropathy (OU), progressive kidney damage is the result because of the associated increased apoptosis and fibrosis. Therefore, it is urgent to find a complementary renoprotective therapy against partially obstructed uropathy cascades. Thus, this study investigated the renoprotective effects of both losartan (LOS) and zinc oxide nanoparticles (ZnONPs) in partial unilateral ureteral obstruction (PUUO).

  • In vivo base editing extends lifespan of a humanized mouse model of prion disease Tue, 14 Jan 2025 06:00:00 -0500
    Prion disease is a fatal neurodegenerative disease caused by the misfolding of prion protein (PrP) encoded by the PRNP gene. While there is currently no cure for the disease, depleting PrP in the brain is an established strategy to prevent or stall templated misfolding of PrP. Here we developed in vivo cytosine and adenine base strategies delivered by adeno-associated viruses to permanently modify the PRNP locus to achieve PrP knockdown in the mouse brain. Systemic injection of...

  • Brain-derived tau oligomer polymorphs: distinct aggregations, stability profiles, and biological activities Tue, 14 Jan 2025 06:00:00 -0500
    Aggregation of microtubule-associated tau protein is a distinct hallmark of several neurodegenerative disorders such as Alzheimer's disease (AD), dementia with Lewy bodies (DLB), and progressive supranuclear palsy (PSP). Tau oligomers are suggested to be the primary neurotoxic species that initiate aggregation and propagate prion-like structures. Furthermore, different diseases are shown to have distinct structural characteristics of aggregated tau, denoted as polymorphs. Here, we investigate...

  • Catalytic-independent functions of the Integrator-PP2A complex (INTAC) confer sensitivity to BET inhibition Tue, 14 Jan 2025 06:00:00 -0500
    Chromatin and transcription regulators are critical to defining cell identity through shaping epigenetic and transcriptional landscapes, with their misregulation being closely linked to oncogenesis. Pharmacologically targeting these regulators, particularly the transcription-activating BET proteins, has emerged as a promising approach in cancer therapy, yet intrinsic or acquired resistance frequently occurs, with poorly understood mechanisms. Here, using genome-wide CRISPR screens, we find that...

  • A targetable OSGIN1 - AMPK - SLC2A3 axis controls the vulnerability of ovarian cancer to ferroptosis Tue, 14 Jan 2025 06:00:00 -0500
    Despite advances in various chemotherapy regimens, current therapeutic options are limited for ovarian cancer patients. Oxidative stress-induced growth inhibitor 1 (OSGIN1), which is a tumor suppressor gene known to regulate the cellular stress response and apoptosis, is associated with ovarian cancer development. However, the underlying mechanisms involved in ferroptosis regulation have not been elucidated. Thus, this study aimed to investigate the effect and underlying regulatory mechanism of...

  • Genomic landscape and comparative analysis of tissue and liquid-based NGS in Taiwanese anaplastic thyroid carcinoma Tue, 14 Jan 2025 06:00:00 -0500
    Anaplastic thyroid carcinoma (ATC) is an aggressive cancer that requirements rapid diagnosis and multimodal treatment. Next-generation sequencing (NGS) aids in personalized therapies and improved trial enrollment. The role of liquid-based NGS in ATC remains unclear. This study analyzed ATC samples using tissue-based NGS, liquid-based NGS, or both platforms. Genetic alterations showed highly heterogeneity, including mutations in RAS/RAF/MEK/ERK, PI3K/AKT/mTOR, cell cycle regulation, other...

  • MicroRNA-150-3p enhances the antitumour effects of CGP57380 and is associated with a favourable prognosis in non-small cell lung cancer Tue, 14 Jan 2025 06:00:00 -0500
    MicroRNA (miRNA) dysregulation has been identified in several carcinomas, including non-small cell lung cancer (NSCLC), and is known to play a role in the development and progression of this disease. We initially conducted a miRNA microarray analysis, which revealed that the MNK inhibitor CGP57380 increased the expression of miR-150-3p. A similar analysis was performed using data from The Cancer Genome Atlas (TCGA). Cell proliferation, colony formation and migration assays were validated in A549...

  • Expression of some circulating microRNAs as predictive biomarkers for prognosis and treatment response in glioblastoma Tue, 14 Jan 2025 06:00:00 -0500
    Glioblastoma multiforme (GBM) is the most prevalent, treatment-resistant, and fatal form of brain malignancy. It is characterized by genetic heterogeneity, and an infiltrative nature, and GBM treatment is highly challenging. Despite multimodal therapies, clinicians lack efficient prognostic and predictive markers. Therefore, new insights into GBM management are urgently needed to increase the chance of therapeutic success. Circulating miRNAs (miRs) are important regulators of cancer progression...

  • Network pharmacology combined with experimental verification for exploring the potential mechanism of phellodendrine against depression Tue, 14 Jan 2025 06:00:00 -0500
    The anti-inflammatory effect of phellodendrine (PHE), derived from Phellodendri Chinensis Cortex, has been verified in previous studies. Major depressive disorder (MDD) is associated with immune dysregulation and inflammatory processes. This study aimed to explore the therapeutic effects of PHE on MDD through network pharmacology and experimental validation. Multiple databases were used to predict the targets of PHE and MDD. The intersection targets between PHE and MDD were obtained to identify...

  • Robust genome editing activity and the applications of enhanced miniature CRISPR-Cas12f1 Tue, 14 Jan 2025 06:00:00 -0500
    With recent advancements in gene editing technology using the CRISPR/Cas system, there is a demand for more effective gene editors. A key factor facilitating efficient gene editing is effective CRISPR delivery into cells, which is known to be associated with the size of the CRISPR system. Accordingly, compact CRISPR-Cas systems derived from various strains are discovered, among which Un1Cas12f1 is 2.6 times smaller than SpCas9, providing advantages for gene therapy research. Despite extensive...

  • Enabling next-generation engineered TCR-T therapies based on high-throughput TCR discovery from diagnostic tumor biopsies Tue, 14 Jan 2025 06:00:00 -0500
    Adoptive cell therapy with tumor-infiltrating lymphocytes (TIL) can mediate tumor regression, including complete and durable responses, in a range of solid cancers, most notably in melanoma. However, its wider application and efficacy has been restricted by the limited accessibility, proliferative capacity and effector function of tumor-specific TIL. Here, we develop a platform for the efficient identification of tumor-specific TCR genes from diagnostic tumor biopsies, including core-needle...

  • Digestive cancers: mechanisms, therapeutics and management Tue, 14 Jan 2025 06:00:00 -0500
    Cancers of the digestive system are major contributors to global cancer-associated morbidity and mortality, accounting for 35% of annual cases of cancer deaths. The etiologies, molecular features, and therapeutic management of these cancer entities are highly heterogeneous and complex. Over the last decade, genomic and functional studies have provided unprecedented insights into the biology of digestive cancers, identifying genetic drivers of tumor progression and key interaction points of tumor...

  • A diagnostic host-specific transcriptome response for Mycoplasma pneumoniae pneumonia to guide pediatric patient treatment Tue, 14 Jan 2025 06:00:00 -0500
    Mycoplasma pneumoniae causes atypical pneumonia in children and young adults. Its lack of a cell wall makes it resistant to beta-lactams, which are the first-line treatment for typical pneumonia. Current diagnostic tests are time-consuming and have low specificity, leading clinicians to administer empirical antibiotics. Using a LASSO regression simulation approach and blood microarray data from 107 children with pneumonia (including 30 M. pneumoniae) we identify eight different transcriptomic...

  • Behavioral and Electrophysiological Assessment of Central Auditory Processing in Individuals with Sickle Cell Disease Tue, 14 Jan 2025 06:00:00 -0500
    CONCLUSION: Individuals with sickle cell disease have central auditory processing disorder, identified primarily by behavioral assessment.