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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.


  • Prognostic Significance of Iron Metabolism and Immune-Related Genes as Risk Markers in Hepatocellular Carcinoma Fri, 12 Apr 2024 06:00:00 -0400
    Hepatocellular carcinoma (HCC) is a highly lethal liver cancer with significant heterogeneity, which poses challenges in predicting prognosis and treatment outcomes. The impact of iron metabolism and immune-related genes (IMRGs) on HCC patient prognoses remains elusive. We utilized The Cancer Genome Atlas (TCGA) dataset to obtain mRNA expression data and clinical information from HCC patients. Through the application of LASSO regression and univariate/multivariate Cox regression analyses, we...

  • Molecular Pathogenesis, Organ Metastasis, and Targeted Therapy for Non-Small-Cell Lung Cancer Fri, 12 Apr 2024 06:00:00 -0400
    Around 2 million people are diagnosed with lung cancer annually, causing 20,000 deaths. Non-small cell carcinomas account for 80-85% of lung cancer cases. Over the last few decades, there has been an improved understanding of the chromosomal makeup of lung cancer. As a result, the clinical care and treatment of patients with advanced or metastatic non-small-cell lung cancer (NSCLC) have changed. This is possible due to advanced molecular techniques and chromosomal analysis, which have revealed...

  • Charge-Reversed Exosomes for Targeted Gene Delivery to Cartilage for Osteoarthritis Treatment Fri, 12 Apr 2024 06:00:00 -0400
    Gene therapy has the potential to facilitate targeted expression of therapeutic proteins to promote cartilage regeneration in osteoarthritis (OA). The dense, avascular, aggrecan-glycosaminoglycan (GAG) rich negatively charged cartilage, however, hinders their transport to reach chondrocytes in effective doses. While viral vector mediated gene delivery has shown promise, concerns over immunogenicity and tumorigenic side-effects persist. To address these issues, this study develops...

  • Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy Fri, 12 Apr 2024 06:00:00 -0400
    CONCLUSIONS: The Delphi Panel established consensus considerations for the evaluation and management of potential TRAEs for patients receiving delandistrogene moxeparvovec, including vomiting, acute liver injury, myocarditis, and immune-mediated myositis.

  • Channelopathies in epilepsy: an overview of clinical presentations, pathogenic mechanisms, and therapeutic insights Fri, 12 Apr 2024 06:00:00 -0400
    Pathogenic variants in genes encoding ion channels are causal for various pediatric and adult neurological conditions. In particular, several epilepsy syndromes have been identified to be caused by specific channelopathies. These encompass a spectrum from self-limited epilepsies to developmental and epileptic encephalopathies spanning genetic and acquired causes. Several of these channelopathies have exquisite responses to specific antiseizure medications (ASMs), while others ASMs may prove...

  • Treatment of Netherton Syndrome in Pediatrics with Upadacitinib Fri, 12 Apr 2024 06:00:00 -0400
    BACKGROUND: Netherton syndrome, a rare autosomal recessive genetic disease, lacks effective treatment options. This article presents a novel case of successful Upadacitinib therapy in a 14-year-old boy with Netherton syndrome.

  • Gentiopicrin-Loaded Chitosan Nanoparticles as a Topical Agent for the Treatment of Psoriasis Fri, 12 Apr 2024 06:00:00 -0400
    Psoriasis, a chronic inflammatory skin disease induced by various factors, including genetic factors, immune factors, environmental factors, and psychological factors, is characterized by thickening of the epidermis, excessive proliferation of keratinocytes, abnormal differentiation, and an excessive inflammatory response. Traditional treatments for psoriasis still face challenges because of limited curative effects, notable side effects, and a tendency for recurrence. In contrast, topical...

  • Nanoscale Structure of Lipid-Gemini Surfactant Mixed Monolayers Resolved with AFM and KPFM Microscopy Fri, 12 Apr 2024 06:00:00 -0400
    Drug delivery vehicles composed of lipids and gemini surfactants (GS) are promising in gene therapy. Tuning the composition and properties of the delivery vehicle is important for the efficient load and delivery of DNA fragments (genes). In this paper, we studied novel gene delivery systems composed of 1,2-dioleoyl-sn-glycero-3-phosphocholine (DOPC), 1,2-dipalmitoyl-sn-3-phosphocholine (DPPC), and GS of the type N,N-bis(dimethylalkyl)-α,ω-alkanediammonium dibromide at different ratios. The...

  • Residual Cystine Transport Activity for Specific Infantile and Juvenile CTNS Mutations in a PTEC-Based Addback Model Fri, 12 Apr 2024 06:00:00 -0400
    Cystinosis is a rare, autosomal recessive, lysosomal storage disease caused by mutations in the gene CTNS, leading to cystine accumulation in the lysosomes. While cysteamine lowers the cystine levels, it does not cure the disease, suggesting that CTNS exerts additional functions besides cystine transport. This study investigated the impact of infantile and juvenile CTNS mutations with discrepant genotype/phenotype correlations on CTNS expression, and subcellular localisation and function in...

  • Unlocking the Future: Pluripotent Stem Cell-Based Lung Repair Fri, 12 Apr 2024 06:00:00 -0400
    The human respiratory system is susceptible to a variety of diseases, ranging from chronic obstructive pulmonary disease (COPD) and pulmonary fibrosis to acute respiratory distress syndrome (ARDS). Today, lung diseases represent one of the major challenges to the health care sector and represent one of the leading causes of death worldwide. Current treatment options often focus on managing symptoms rather than addressing the underlying cause of the disease. The limitations of conventional...

  • Synthetic Lethality between Cohesin and WNT Signaling Pathways in Diverse Cancer Contexts Fri, 12 Apr 2024 06:00:00 -0400
    Cohesin is a highly conserved ring-shaped complex involved in topologically embracing chromatids, gene expression regulation, genome compartmentalization, and genome stability maintenance. Genomic analyses have detected mutations in the cohesin complex in a wide array of human tumors. These findings have led to increased interest in cohesin as a potential target in cancer therapy. Synthetic lethality has been suggested as an approach to exploit genetic differences in cancer cells to influence...

  • Genetic Signature of Human Pancreatic Cancer and Personalized Targeting Fri, 12 Apr 2024 06:00:00 -0400
    Pancreatic cancer is a highly lethal disease with a 5-year survival rate of around 11-12%. Surgery, being the treatment of choice, is only possible in 20% of symptomatic patients. The main reason is that when it becomes symptomatic, IT IS the tumor is usually locally advanced and/or has metastasized to distant organs; thus, early diagnosis is infrequent. The lack of specific early symptoms is an important cause of late diagnosis. Unfortunately, diagnostic tumor markers become positive at a late...

  • Preclinical Development of Antisense Oligonucleotides to Rescue Aberrant Splicing Caused by an Ultrarare ABCA4 Variant in a Child with Early-Onset Stargardt Disease Fri, 12 Apr 2024 06:00:00 -0400
    Precision medicine is rapidly gaining recognition in the field of (ultra)rare conditions, where only a few individuals in the world are affected. Clinical trial design for a small number of patients is extremely challenging, and for this reason, the development of N-of-1 strategies is explored to accelerate customized therapy design for rare cases. A strong candidate for this approach is Stargardt disease (STGD1), an autosomal recessive macular degeneration characterized by high genetic and...

  • Challenges and Considerations of Preclinical Development for iPSC-Based Myogenic Cell Therapy Fri, 12 Apr 2024 06:00:00 -0400
    Cell therapies derived from induced pluripotent stem cells (iPSCs) offer a promising avenue in the field of regenerative medicine due to iPSCs' expandability, immune compatibility, and pluripotent potential. An increasing number of preclinical and clinical trials have been carried out, exploring the application of iPSC-based therapies for challenging diseases, such as muscular dystrophies. The unique syncytial nature of skeletal muscle allows stem/progenitor cells to integrate, forming new...

  • Controlling Recombination to Evolve Bacteriophages Fri, 12 Apr 2024 06:00:00 -0400
    Recombination among different phages sometimes facilitates their ability to grow on new hosts. Protocols to direct the evolution of phage host range, as might be used in the application of phage therapy, would then benefit from including steps to enable recombination. Applying mathematical and computational models, in addition to experiments using phages T3 and T7, we consider ways that a protocol may influence recombination levels. We first address coinfection, which is the first step to...

  • Preclinical Advances in LNP-CRISPR Therapeutics for Solid Tumor Treatment Fri, 12 Apr 2024 06:00:00 -0400
    Solid tumors, with their intricate cellular architecture and genetic heterogeneity, have long posed therapeutic challenges. The advent of the CRISPR genome editing system offers a promising, precise genetic intervention. However, the journey from bench to bedside is fraught with hurdles, chief among them being the efficient delivery of CRISPR components to tumor cells. Lipid nanoparticles (LNPs) have emerged as a potential solution. This biocompatible nanomaterial can encapsulate the CRISPR/Cas9...

  • Nasopharyngeal Carcinoma Cell Lines: Reliable Alternatives to Primary Nasopharyngeal Cells? Fri, 12 Apr 2024 06:00:00 -0400
    Nasopharyngeal carcinoma (NPC) is a type of cancer that originates from the mucosal lining of the nasopharynx and can invade and spread. Although contemporary chemoradiotherapy effectively manages the disease locally, there are still challenges with locoregional recurrence and distant failure. Therefore, it is crucial to have a deeper understanding of the molecular basis of NPC cell movement in order to develop a more effective treatment and to improve patient survival rates. Cancer cell line...

  • Monitoring for liver cancer post-gene therapy-How much and how often? Fri, 12 Apr 2024 06:00:00 -0400
    Hepatocellular carcinoma (HCC) has long been recognized as a complication in people with chronic liver disease, particularly those with cirrhosis. Two gene therapies for haemophilia A and B recently approved in Europe and the US utilize adeno-associated virus (AAV) vectors designed to target hepatocytes. A number of other AAV gene therapies are undergoing clinical investigation for both liver and extrahepatic diseases, many of which likely transduce hepatocytes as well. Although AAV vectors...

  • Hepatitis after gene therapy, what are the possible causes? Fri, 12 Apr 2024 06:00:00 -0400
    Hepatitis is a common adverse event following gene therapy for haemophilia, often associated with a loss of transgene expression. Investigating the potential causes and implications of this is crucial for the overall success of treatment. Gene therapy trials using adeno-associated virus (AAV) vectors have demonstrated promising results marked by increases in factor FVIII and FIX levels and reductions in episodes of bleeding. However, hepatocellular injury characterised by elevations in alanine...

  • The benefits of gene therapy in people with haemophilia Fri, 12 Apr 2024 06:00:00 -0400
    Haemophilia is an inherited bleeding disorder which causes significant morbidity and mortality, especially in the severe form. Prophylaxis with factor replacement has high efficacy in reducing bleeding but is limited by the need for frequent intravenous infusion and fluctuations in haemostasis between doses. Additional prophylaxis therapies are being developed which may overcome some of the current treatment barriers. Gene therapy (GT) is being developed to provide a functional cure such that...