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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.


  • Using artificial intelligence and promoter-level transcriptome analysis to identify a biomarker as a possible prognostic predictor of cardiac complications in male patients with Fabry disease Fri, 01 Nov 2024 06:00:00 -0400
    Fabry disease is the most frequently occurring form of lysosomal disease in Japan, and is characterized by a wide variety of conditions. Primarily, the three major types of concerns associated with Fabry disease observed during adulthood that must be prevented are central nervous system, renal, and cardiac complications. Cardiac complications, such as cardiomyopathy, cardiac muscle fibrosis, and severe arrhythmia, are the most common mortality causes in patients with Fabry disease. To predict...

  • Biomarkers to predict the benefits of immune-checkpoint blockade-based therapy in patients with malignant peritoneal mesothelioma (Review) Fri, 01 Nov 2024 06:00:00 -0400
    Malignant peritoneal mesothelioma (MPeM) is a type of rare and highly lethal tumor. Immune checkpoint blockade (ICB)-based therapy has shown encouraging clinical activity for MPeM. However, no definitive biomarkers have been identified for predicting which patients with MPeM will benefit from ICB-based therapy. At present, there are several novel potential biomarkers proposed for predicting the response to ICB-based therapy, and biomarkers available in MPeM cells and in the tumor...

  • Proinflammatory factors inhibition and fish oil treatment: A promising therapy for neonatal seizures Fri, 01 Nov 2024 06:00:00 -0400
    Brain injury is one of the most important causes of infant mortality and chronic neurological disabilities. Hypoxia is an acute brain injury which led to various cognitive, behavioral, and memory disorders throughout life. Previous studies reported neuroprotective possibilities for fish oil (FO) in brain-injured situations. In this study, we evaluated the effect of the FO diet during the lactation period on seizure activity, behavioral performance, histomorphometry, and inflammatory changes in...

  • Factor 3 regulates airway engraftment by human bronchial basal cells Fri, 01 Nov 2024 06:00:00 -0400
    Cystic fibrosis transmembrane conductance regulator (CFTR) gene editing and transplantation of CFTR-gene corrected airway basal cells has the potential to cure CF lung disease. Although mouse studies established that cell transplantation was feasible, the engraftment rate was typically low and frequently less than the estimated therapeutic threshold. The purpose of this study was to identify genes and culture conditions that regulate the therapeutic potential of human bronchial basal cells....

  • A dual role for PSIP1/LEDGF in T cell acute lymphoblastic leukemia Fri, 01 Nov 2024 06:00:00 -0400
    T cell acute lymphoblastic leukemia (T-ALL) is an aggressive hematological malignancy. Current intensified therapeutic protocols coincide with severe side effects, and no salvage therapy is available for primary therapy-resistant or relapsed patients. This highlights the need to identify new therapeutic targets in T-ALL. PSIP1, dispensable for normal hematopoiesis, is a dependency factor in KMT2A-rearranged myeloid leukemia. Nonetheless, loss-of-function mutations suggest a tumor suppressor role...

  • The effect of different interfaces during virtual game practice on motor performance of individuals with genetic ataxia: A cross-sectional study Fri, 01 Nov 2024 06:00:00 -0400
    CONCLUSION: The group of participants with genetic ataxia presented lower performance than the control group regardless of the interface (webcam or touchscreen). The most interesting observation was that although practicing with the webcam interface offers features that make the task more complex than the touchscreen interface, resulting in lower performance, this interface facilitated performance in a subsequent touchscreen task only in individuals with ataxia, suggesting that a virtual...

  • Effects of water-insoluble wheat bran-fraction powder on disease activity and caecal microbiota in dextran sodium sulphate-induced inflammatory bowel disease mouse model Fri, 01 Nov 2024 06:00:00 -0400
    CONCLUSION: WBI, rather than WBS, reduces inflammation and maintains the gut microbiota. However, further studies are warranted to elucidate the properties of the WBI active components and efficacy of WBI metabolites on gut microbiota, particularly on Faecalibaculum.

  • Prospective study of the real impact of fusion centered genomic assays in patient management in a national collaborative group: the GETHI-XX-16 study Fri, 01 Nov 2024 06:00:00 -0400
    CONCLUSION: Despite the growing knowledge of cancer biology and its translation to drug development, the overall impact of personalized treatments remains low. Access to comprehensive molecular tests covering properly all known actionable alterations and programs for a wide access to targeted therapies seem to be critical steps.

  • GUCA2A dysregulation as a promising biomarker for accurate diagnosis and prognosis of colorectal cancer Fri, 01 Nov 2024 06:00:00 -0400
    Colorectal cancer is a leading cause of global mortality and presents a significant barrier to improving life expectancy. The primary objective of this study was to discern a unique differentially expressed gene (DEG) that exhibits a strong association with colorectal cancer. By achieving this goal, the research aims to contribute valuable insights to the field of translational medicine. We performed analysis of colorectal cancer microarray and the TCGA colon adenoma carcinoma (COAD) datasets to...

  • Development of an optimized SEC method for characterization of genome DNA leakage from adeno-associated virus products Fri, 01 Nov 2024 06:00:00 -0400
    Adeno-associated virus (AAV) vectors are widely used to deliver therapeutic transgenes due to their superior safety, relatively low immunogenicity, and ability to target diverse tissues. AAV gene therapy products are typically formulated as frozen liquid and stored below - 60 °C, and therefore are subjected to multiple freeze/thaw cycles during manufacturing and administration. Recent studies have shown that genome DNA leakage could be induced by freeze/thaw stress. DNA leakage from AAV capsids...

  • Mechanisms of podocyte injury in genetic kidney disease Fri, 01 Nov 2024 06:00:00 -0400
    Glomerular diseases are a leading cause of chronic kidney disease worldwide. Both acquired and hereditary glomerulopathies frequently share a common final disease mechanism: disruption of the glomerular filtration barrier, podocyte injury, and ultimately podocyte death and detachment. Over 70 monogenic causes of proteinuric kidney disease have been identified, and most of these genes are highly expressed in podocytes, regulating key processes such as maintenance of the slit diaphragm, regulation...

  • The Pharmacogenomics Global Research Network Implementation Working Group: global collaboration to advance pharmacogenetic implementation Fri, 01 Nov 2024 06:00:00 -0400
    Pharmacogenetics promises to optimize treatment-related outcomes by informing optimal drug selection and dosing based on an individual's genotype in conjunction with other important clinical factors. Despite significant evidence of genetic associations with drug response, pharmacogenetic testing has not been widely implemented into clinical practice. Among the barriers to broad implementation are limited guidance for how to successfully integrate testing into clinical workflows and limited data...

  • Targeted therapies for Glioblastoma multiforme (GBM): State-of-the-art and future prospects Fri, 01 Nov 2024 06:00:00 -0400
    Glioblastoma multiforme (GBM) remains one of the most aggressive and lethal forms of brain cancer, characterized by rapid growth and resistance to conventional therapies. The present review explores the latest advancements in targeted therapies for GBM, emphasizing the critical role of the blood-brain barrier (BBB), blood-brain-tumor barrier, tumor microenvironment, and genetic mutations in influencing treatment outcomes. The impact of the key hallmarks of GBM, for example, chemoresistance,...

  • Targeting Noncoding cis-Regulatory Elements for Cancer Therapy in the Context of the 3D Genome Fri, 01 Nov 2024 06:00:00 -0400
    Significant efforts have been made to identify and validate oncoproteins and ncRNAs as therapeutic targets for cancer therapy; however, emerging observations suggest that noncoding cis-regulatory elements, which orchestrate the 3D organization of the genome and thus the transcriptional landscape, are potential therapeutic targets as well. In this commentary, we envisage that further efforts to decipher the noncoding cis-regulatory code and performing systematic surveys of functional noncoding...

  • Beneath the Surface: Neoantigens beyond Chromosomal DNA Mutations Fri, 01 Nov 2024 06:00:00 -0400
    The conventional wisdom is that the overwhelming majority of neoantigens arise from chromosomal DNA mutations; however, recent studies show that posttranscriptional and posttranslational events can also generate neoantigens. This commentary provides an overview of known and potential sources of nonchromosomal neoantigens, emerging technologies, and clinical trials that may move this field forward to redefine immunologically "hot/cold" tumors and develop next-generation immunotherapeutic...

  • A luminal non-coding RNA-based genomic classifier confirms favourable outcomes in patients with clinically organ-confined bladder cancer treated with radical cystectomy Fri, 01 Nov 2024 06:00:00 -0400
    CONCLUSIONS: This study validates the performance of the GC for identifying UCs with a luminal favourable subtype, harbouring less aggressive tumour biology.

  • The Effects and Mechanisms of Chrysosplenetin in Targeting RANKL-Induced NF-κB Signaling and NFATc1 Activation to Protect Bone Density in Osteolytic Diseases Fri, 01 Nov 2024 06:00:00 -0400
    Chrysosplenetin (CHR), an O-methylated flavonol from Chamomilla recutita and Laggera pterodonta, has previously demonstrated efficacy in enhancing osteoblast differentiation for treating postmenopausal osteoporosis. This study aims to evaluate CHR's potential to inhibit osteoclastogenesis and prevent bone deterioration in both in vitro and in vivo models. Using tartaric acid-resistant acid phosphatase staining and hydroxyapatite resorption assays, we examined the impact of CHR on RANKL-induced...

  • Early diagnosis of immunodeficient patients with partial albinism: The role of hair study and peripheral blood smear Fri, 01 Nov 2024 06:00:00 -0400
    CONCLUSION: Receiving hematopoietic stem cell transplantation (HSCT) in a timely manner is crucial in CHS and GS2 patients; therefore, screening tests may provide a vital clue for early diagnosis in these patients. However, the final confirmation of CHS, GS2, and HPS2 disorders is done by genetic assay.

  • Stem cell transplantation and allogeneic immunity: post treatment control or HIV cure? Fri, 01 Nov 2024 06:00:00 -0400
    PURPOSE OF REVIEW: Long-lasting HIV remission has been reported in a small group of people with HIV (PWH) following allogenic hematopoietic stem cell transplants (HSCT) for the treatment of hematologic malignancies. While the mechanisms of HIV remission following release from antiretroviral therapy (ART) were not initially known, subsequent findings from clinical cases and preclinical nonhuman primate studies have implicated mechanisms of clearance. Here, we review the six currently published...

  • Elucidating the Mechanisms of Astragalus Membranaceus in Colorectal Cancer Patients through Bioinformatics Analysis Fri, 01 Nov 2024 06:00:00 -0400
    CONCLUSION: This study identified the active components of Astragalus membranaceus and their corresponding targets in CRC. These findings provide robust evidence for precision drug therapy in patients with CRC.