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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.


  • Lung Progenitor and Stem Cell Transplantation as a Potential Regenerative Therapy for Lung Diseases Wed, 28 Feb 2024 06:00:00 -0500
    Chronic lung diseases are debilitating illnesses ranking among the top causes of death globally. Currently, clinically available therapeutic options capable of curing chronic lung diseases are limited to lung transplantation, which is hindered by donor organ shortage. This highlights the urgent need for alternative strategies to repair damaged lung tissues. Stem cell transplantation has emerged as a promising avenue for regenerative treatment of the lung, which involves delivery of healthy lung...

  • Durvalumab Plus Gemcitabine and Cisplatin in Patients with Advanced Biliary Tract Cancer: An Exploratory Analysis of Real-World Data Wed, 28 Feb 2024 06:00:00 -0500
    CONCLUSIONS: The treatment of advanced patients with BTC with durvalumab and gem/cis, even beyond the inclusion criteria of the TOPAZ-1 trial, shows promising safety.

  • Current challenges in cell and gene therapy: a joint view from the European Committee of the International Society for Cell & Gene Therapy (ISCT) and the European Society for Blood and Marrow Transplantation (EBMT) Wed, 28 Feb 2024 06:00:00 -0500
    Cell and gene therapy poses evolving challenges. The current article summarizes the discussions held by European Regional Committee of the International Society for Cell & Gene Therapy and the European Society for Blood and Marrow Transplantation (EBMT) on the current challenges in this field, focusing on the European setting. This article emphasizes the imperative assessment of real-world cell and gene therapy activity, advocating for expanded registries beyond hematopoietic transplantation and...

  • Cone cell dysfunction attenuates retinal neovascularization in oxygen-induced retinopathy mouse model Wed, 28 Feb 2024 06:00:00 -0500
    Aberrant neovascularization is the most common feature in retinopathy of prematurity (ROP), which leads to the retinal detachment and visual defects in neonates with a low gestational age eventually. Understanding the regulation of inappropriate angiogenic signaling benefits individuals at-risk. Recently, neural activity originating from the specific neural activity has been considered to contribute to retinal angiogenesis. Here, we explored the impact of cone cell dysfunction on oxygen-induced...

  • Effect of CHRDL1 on angiogenesis and metastasis of colorectal cancer cells via TGF-β/VEGF pathway Wed, 28 Feb 2024 06:00:00 -0500
    Colorectal cancer (CRC) is a common digestive tract tumor with the third incidence and death in the world. There is still an urgent need for effective therapeutic targets and prognostic markers for CRC. Herein, we report a novel potential target and marker, Chordin like-1 (CHRDL1). The function of CHRDL1 has been reported in gastric cancer, breast cancer, and oral squamous cell carcinoma. However, the biological effect of CHRDL1 in CRC remains unrevealed. Transwell and tube formation experiments...

  • Recent Developments in Nanotechnology and Immunotherapy for the Diagnosis and Treatment of Pancreatic Cancer Wed, 28 Feb 2024 06:00:00 -0500
    Pancreatic cancer kills millions of people worldwide each year and is one of the most prevalent causes of mortality that requires prompt therapy. A large number of people who have pancreatic cancer are detected at an advanced stage, with incurable and drug-resistant tumors. Hence the overall survival rate of pancreatic cancer is less. The advance phase of this cancer is generated because of the expression of the cancer-causing gene, inactivation of the tumorsuppressing gene, and deregulation of...

  • Analysis and Validation of Tyrosine Metabolism-related Prognostic Features for Liver Hepatocellular Carcinoma Therapy Wed, 28 Feb 2024 06:00:00 -0500
    CONCLUSION: Our study developed a tyrosine metabolism-related risk model that performed well in survival prediction, showing the potential to serve as an independent prognostic predictor for LIHC treatment.

  • Advanced Targeted Drug Delivery of Bioactive Agents Fortified with Graft Chitosan in Management of Cancer: A Review Wed, 28 Feb 2024 06:00:00 -0500
    Cancer is characterized by the uncontrolled proliferation and spread of abnormal cells in the body, resulting in the development of tumors or clusters of irregular cells. The factors contributing to cancer are intricate, involving a combination of genetic, environmental, and lifestyle elements. Risk factors for cancer include the use of nicotine, excessive alcohol consumption, exposure to radiation or specific chemicals, and a family history of the disease. Common treatment methods for cancer...

  • A classification-based generative approach to selective targeting of global slow oscillations during sleep Wed, 28 Feb 2024 06:00:00 -0500
    CONCLUSION: Our research presents a novel approach to optimize cl-tACS during sleep, with a focus on targeting global SOs. This approach holds promise for improving cl-tACS not only for global SOs but also for other physiological events, benefiting both research and clinical applications in sleep and cognition.

  • Primary Malignant Melanoma of the Endometrium: A Case Report Wed, 28 Feb 2024 06:00:00 -0500
    CONCLUSION: Mucosal melanoma is infrequent, and primary malignant melanoma of endometrial is a rare subtype. To the best of our knowledge, malignant melanoma originating from endometrium has never been reported before. It has a high degree of malignancy and is prone to early metastasis. Further investigations are warranted to explore its underlying pathogenesis, management, and outcomes.

  • Case Report: A case report and literature review of hemoglobin variation associated with neonatal cyanosis Wed, 28 Feb 2024 06:00:00 -0500
    We will discuss a recent case of unexplained neonatal cyanosis, evaluate its origin, clinical presentation, diagnosis, and treatment, and share with you some of our clinical insights. We report a transient cyanosis in a newborn due to a mutation in the globulin gene (HBG2), as well as diagnosis and treatment. Clinically, the infant was in good overall health, and despite low oxygen saturation, the arterial oxygen partial pressure was always normal. Early respiratory support includes mechanical...

  • Grand Challenges at the Interface of Engineering and Medicine Wed, 28 Feb 2024 06:00:00 -0500
    Over the past two decades Biomedical Engineering has emerged as a major discipline that bridges societal needs of human health care with the development of novel technologies. Every medical institution is now equipped at varying degrees of sophistication with the ability to monitor human health in both non-invasive and invasive modes. The multiple scales at which human physiology can be interrogated provide a profound perspective on health and disease. We are at the nexus of creating "avatars"...

  • A comprehensive literature scoping review of infection prevention and control methods for viral-mediated gene therapies Wed, 28 Feb 2024 06:00:00 -0500
    CONCLUSIONS: The need to define IPC methods is high due to the rapid emergence of viral-mediated gene therapies to treat rare diseases, but published clinical guidance remains scarce. In the absence of these data, our center recommends a 1:10 sodium hypochlorite solution in clinical and academic environments to ensure complete germicidal activity of viral-mediated gene therapies.

  • Clinical Experience with Berotralstat in Patients with Hereditary Angioedema with Normal C1-Esterase Inhibitor: A Commented Case Series Wed, 28 Feb 2024 06:00:00 -0500
    Hereditary angioedema (HAE) is a rare genetic disorder characterized by potentially life-threatening episodes of swelling. Most HAE cases are caused by deficient (type I) or dysfunctional (type II) C1-esterase inhibitor (C1-INH) protein. However, some patients present with a subtype of HAE that is associated with normal plasma levels of functional C1-INH protein and complement component 4 (HAE-nC1INH). Treatment of HAE-nC1INH is driven by clinical experience as robust clinical trial data to...

  • Exploring the relationship between intestinal microbiota and immune checkpoint inhibitors in the treatment of non-small cell lung cancer: insights from the "lung and large intestine stand in exterior-interior relationship" theory Wed, 28 Feb 2024 06:00:00 -0500
    CONCLUSION: The strains Clostridia, Lachnospiraceae, and Lachnospirales emerge as potential biomarkers denoting the composition of the intestinal microbiota in the NSCLC therapy. The immunotherapy efficacy of ICIs markedly accentuates in patients displaying durable treatment benefits and those expressing positive PD-L1.

  • Expert Consensus on the Diagnosis and Treatment of NRG1/2 Gene Fusion Solid Tumors Wed, 28 Feb 2024 06:00:00 -0500
    The fusion genes NRG1 and NRG2 , members of the epidermal growth factor (EGF) receptor family, have emerged as key drivers in cancer. Upon fusion, NRG1 retains its EGF-like active domain, binds to the ERBB ligand family, and triggers intracellular signaling cascades, promoting uncontrolled cell proliferation. The incidence of NRG1 gene fusion varies across cancer types, with lung cancer being the most prevalent at 0.19 to 0.27%. CD74 and SLC3A2 are the most frequently observed fusion partners....

  • Myoglobin modulates the Hippo pathway to promote cardiomyocyte differentiation Wed, 28 Feb 2024 06:00:00 -0500
    The endogenous mechanisms that propagate cardiomyocyte differentiation and prevent de-differentiation remain unclear. While the expression of the heme protein myoglobin increases by over 50% during cardiomyocyte differentiation, a role for myoglobin in regulating cardiomyocyte differentiation has not been tested. Here, we show that deletion of myoglobin in cardiomyocyte models decreases the gene expression of differentiation markers and stimulates cellular proliferation, consistent with...

  • Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome Wed, 28 Feb 2024 06:00:00 -0500
    Wiskott-Aldrich syndrome (WAS) is a severe X-linked primary immunodeficiency resulting from a diversity of mutations distributed across all 12 exons of the WAS gene. WAS encodes a hematopoietic-specific and developmentally regulated cytoplasmic protein (WASp). The objective of this study was to develop a gene correction strategy potentially applicable to most WAS patients by employing nuclease-mediated, site-specific integration of a corrective WAS gene sequence into the endogenous WAS...

  • Anisotropic microtopography surface of chitosan scaffold regulating skin precursor-derived Schwann cells towards repair phenotype promotes neural regeneration Wed, 28 Feb 2024 06:00:00 -0500
    For repairing peripheral nerve and spinal cord defects, biomaterial scaffold-based cell-therapy was emerged as an effective strategy, requiring the positive response of seed cells to biomaterial substrate and environment signals. Previous work highlighted that the imposed surface properties of scaffold could provide important guidance cues to adhered cells for polarization. However, the insufficiency of native Schwann cells and unclear cellular response mechanisms remained to be addressed. Given...

  • Exploring the efficacy and safety of Ambroxol in Gaucher disease: an overview of clinical studies Wed, 28 Feb 2024 06:00:00 -0500
    Gaucher disease (GD) is mainly caused by glucocerebrosidase (GCase) enzyme deficiency due to genetic variations in the GBA1 gene leading to the toxic accumulation of sphingolipids in various organs, which causes symptoms such as anemia, thrombocytopenia, hepatosplenomegaly, and neurological manifestations. GD is clinically classified into the non-neuronopathic type 1, and the acute and chronic neuronopathic forms, types 2 and 3, respectively. In addition to the current approved GD medications,...