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.

---

• Identification of age-specific biomarkers of spinal cord injury: A bioinformatics analysis of young and aged mice models Thu, 30 Nov 2023 06:00:00 -0500
  CONCLUSION: Our study explored the RNA expression patterns and enriched signaling pathways in young and aged SCI mice. These findings may provide potential biomarkers for targeted SCI therapy.


• Pregnancy in Autosomal Dominant Polycystic Kidney Disease Thu, 30 Nov 2023 06:00:00 -0500
  Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disorder occurring in approximately 1:1000 individuals. ADPKD is characterized by gradual cyst expansion and kidney enlargement and is a slowly progressive disorder where patients typically initiate renal replacement therapy in the sixth decade of life. The vast majority of women with ADPKD become pregnant in the third or fourth decade, often before knowing that they have ADPKD, in the setting of normal...


• What are the considerations when initiating treatment for epilepsy in children? Thu, 30 Nov 2023 06:00:00 -0500
  INTRODUCTION: There is a very wide spectrum of epilepsies and developmental and epileptic encephalopathies that affect children, from self-limited forms, not necessarily requiring treatment, to severe drug-resistant ones.


• Augmentation of natriuretic peptide (NP) receptor a and B (NPR-A and NPR-B) and cyclic guanosine monophosphate (cGMP) signaling as a therapeutic strategy in heart failure Thu, 30 Nov 2023 06:00:00 -0500
  INTRODUCTION: Heart failure is a complex, debilitating condition and despite advances in treatment, it remains a significant cause of morbidity and mortality worldwide. Therefore, the need for alternative treatment strategies is essential. In this review, we explore the therapeutic strategies of augmenting natriuretic peptide receptors (NPR-A and NPR-B) and cyclic guanosine monophosphate (cGMP) in heart failure.


• Immunologic Signatures of Peripheral Blood T Cells Reveal the Outcome of p53MVA Vaccine and Pembrolizumab Treatment in Patients with Advanced Ovarian Cancer Thu, 30 Nov 2023 06:00:00 -0500
  CONCLUSIONS: p53MVA/pembrolizumab immunotherapy showed promising anti-tumor activity in patients who demonstrated functionally competent peripheral blood T cells. Detection of markers of terminally differentiated T cells before treatment may identify patients unlikely to respond to p53MVA/pembrolizumab.


• FBXW7-loss sensitizes cells to ATR inhibition through induced mitotic catastrophe Thu, 30 Nov 2023 06:00:00 -0500
  FBXW7 is a commonly mutated tumor suppressor gene that functions to regulate numerous oncogenes involved in cell cycle regulation. Genome-wide CRISPR fitness screens identified a signature of DNA repair and DNA damage response genes as required for the growth of FBXW7-knockout cells. Guided by these findings, we show that FBXW7 mutant cells have high levels of replication stress, which results in a genotype-specific vulnerability to inhibition of the ATR signaling pathway, as these mutant cells...


• Lipoprotein lipids and apolipoproteins in primary immune thrombocytopenia: Results from a clinical characteristics and causal relationship verification, potential drug target identification by Mendelian randomization analyses Thu, 30 Nov 2023 06:00:00 -0500
  Primary immune thrombocytopenia (ITP) is an acquired autoimmune disease. Cellular and systemic lipid metabolism plays a significant role in the regulation of immune cell activities. However, the role of lipoprotein lipids and apolipoproteins in ITP remains elusive. The automatic biochemistry analyser was used to measure the levels of serum total cholesterol (TC), triglyceride (TG), low-density lipoprotein cholesterol (LDL-C), high-density lipoprotein cholesterol (HDL-C), apolipoprotein A-I...


• DDX5 enhances HIF-1 activity by promoting the interaction of HIF-1α with HIF-1β and recruiting the resulting heterodimer to its target gene loci Thu, 30 Nov 2023 06:00:00 -0500
  Cancer cells acquire malignant characteristics and therapy resistance by employing the hypoxia-inducible factor 1 (HIF-1)-dependent adaptive response to hypoxic microenvironment in solid tumors. Since the underlying molecular mechanisms remain unclear, difficulties are associated with establishing effective therapeutic strategies. We herein identified DEAD-box helicase 5 (DDX5) as a novel activator of HIF-1 and found that it enhanced the heterodimer formation of HIF-1α and HIF-1β and facilitated...


• Mechanism of Weiwei granules in the treatment of chronic active Helicobacter pylori gastritis with atrophy based on the TLR4/NF-κB/COX-2 inflammatory signaling pathway Thu, 30 Nov 2023 06:00:00 -0500
  CONCLUSION: Weiwei granules can effectively improve Hp-positive CAG patients and reduce the expression levels of TLR4, NF-κB, and COX-2.


• Increased FH-Risk-Score and Diabetes Are Cardiovascular Risk Equivalents in Heterozygous Familial Hypercholesterolemia Thu, 30 Nov 2023 06:00:00 -0500
  CONCLUSIONS: This study strongly suggests that the observed risk of FH patients with diabetes, history of ASCVD, and FH-Risk-Score >20% is as high or higher than non-FH individuals with a history of ASCVD. More aggressive management should be recommended for these patients.


• Thrombosis in the Coronary Microvasculature Impairs Cardiac Relaxation and Induces Diastolic Dysfunction Thu, 30 Nov 2023 06:00:00 -0500
  CONCLUSIONS: Altogether, these results demonstrate that microvessel thrombosis may participate in the pathophysiology of heart failure with preserved ejection fraction.


• Type 1 diabetes - What's new in prevention and therapeutic strategies? Thu, 30 Nov 2023 06:00:00 -0500
  Type 1 diabetes (T1D) is an autoimmune disorder, and insulin deficiency is the result of b-cell dysfunction. Treatment of type 1 diabetes requires constant parenteral insulin administration, which can be very burdensome for the patient. Meticulous use of insulin therapy does not protect the patient against complications. Hence, the search for other methods of treatment as well as ways of preventing the onset of diabetes has been ongoing for a long time. The main obstacle in the implementation of...


• Anti-Gene IGF-I Vaccines in Cancer Gene Therapy: A Review of a Case of Glioblastoma Thu, 30 Nov 2023 06:00:00 -0500
  CONCLUSION: The anti-gene IGF-I vaccines continue to be applied in current GBM personalized medicine. Technical improvements in the preparation of AS and TH vaccines to increase MHC-1 and B7 immunogenicity have, in parallel, allowed to increase in the median survival of patients.


• Diazepam Binding Inhibitor Control of Eu- and Hypoglycemic Patterns of Ventromedial Hypothalamic Nucleus Glucose-Regulatory Signaling Thu, 30 Nov 2023 06:00:00 -0500
  Pharmacological stimulation/antagonism of astrocyte glio-peptide octadecaneuropeptide signaling alters ventromedial hypothalamic nucleus (VMN) counterregulatory γ-aminobutyric acid (GABA) and nitric oxide transmission. The current research used newly developed capillary zone electrophoresis-mass spectrometry methods to investigate hypoglycemia effects on VMN octadecaneuropeptide content, along with gene knockdown tools to determine if octadecaneuropeptide signaling regulates these transmitters...


• First-in-human trial of PCSK9 gene editing therapy for lowering cholesterol: a new frontier in cardiovascular pharmacotherapy? News from AHA Thu, 30 Nov 2023 06:00:00 -0500
  No abstract


• Hypoxia A Typical Target in Human Lung Cancer Therapy Thu, 30 Nov 2023 06:00:00 -0500
  Lung cancer (LC) is the leading cause of cancer-related death globally. Comprehensive knowledge of the cellular and molecular etiology of LC is perilous for the development of active treatment approaches. Hypoxia in cancer is linked with malignancy, and its phenotype is implicated in the hypoxic reaction, which is being studied as a prospective cancer treatment target. The hypervascularization of the tumor is the main feature of human LC, and hypoxia is a major stimulator of neo-angiogenesis. It...


• An Updated Review on Molecular Biomarkers in Diagnosis and Therapy of Colorectal Cancer Thu, 30 Nov 2023 06:00:00 -0500
  Colorectal cancer is one of the most common cancer types worldwide. Since colorectal cancer takes time to develop, its incidence and mortality can be treated effectively if it is detected in its early stages. As a result, non-invasive or invasive biomarkers play an essential role in the early diagnosis of colorectal cancer. Many experimental studies have been carried out to assess genetic, epigenetic, or protein markers in feces, serum, and tissue. It may be possible to find biomarkers that will...


• Combined RNAi of CTTN and FGF2 Modulates Cell Migration, Invasion and G1/S Transition of Hepatocellular Carcinoma through Ras/ERK Signaling Pathway Thu, 30 Nov 2023 06:00:00 -0500
  CONCLUSION: Combined RNAi of CTTN and FGF2 may be a novel and promising intervention strategy for HCC invasion and metastasis.


• The effect of TLR3 priming conditions on MSC immunosuppressive properties Thu, 30 Nov 2023 06:00:00 -0500
  CONCLUSIONS: TLR3 activation in MSCs is dependent on exposure time and poly(I:C) concentration. The maximum expression of immunosuppressive molecules was observed with 10 µg/mL poly(I:C) for 3-h preconditioning. This priming protocol for MSCs enhances the immunosuppressive effects of prMSCs on T cells.


• Visualization of microRNA therapy in cancers delivered by small extracellular vesicles Thu, 30 Nov 2023 06:00:00 -0500
  MicroRNA (miRNA) delivery by extracellular vesicles (EVs) has recently inspired tremendous developments in cancer treatments. However, hybridization between miRNA and its target mRNA is still difficult to be imaged in vivo to assess the therapeutic effects in time. Herein we design a nano-scale fluorescent "off-on" complex encapsulated by small extracellular vesicles (sEVs) for real-time visualization and evaluation of gene therapy efficiency in human gastric cancer cells and murine xenograft...