Inherited kidney diseases are among the leading causes of chronic kidney disease, reducing the quality of life and resulting in substantial socioeconomic impact. The advent of early genetic testing and the growing understanding of the molecular basis and pathophysiology of these disorders have opened avenues for novel treatment strategies. Viral vector-based gene therapies have evolved from experimental treatments for rare diseases to potent platforms that carry the intrinsic potential to provide a cure with a single application. Several gene therapy products have reached the market, and the numbers are only expected to increase. Still, none target inherited kidney diseases. Gene transfer to the kidney has lagged when compared to other tissue-directed therapies such as hepatic, neuromuscular, and ocular tissues. Systemic delivery of genetic information to tackle kidney disease is challenging. The pharma industry is taking steps to take on kidney disease and to translate the current research into the therapeutic arena. In this review, we provide an overview of the current viral vector-based approaches and their potential. We discuss advances in platforms and injection routes that have been explored to enhance gene delivery toward kidney cells in animal models, and how these can fuel the development of viable gene therapy products for humans.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1089/hum.2023.184 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Adenoviral Vector-Based Vaccine Expressing Hemagglutinin Stem Region with Autophagy-Inducing Peptide Confers Cross-Protection Against Group 1 and 2 Influenza A Viruses.
Vaccines (Basel)
January 2025
Department of Comparative Pathobiology, Purdue Institute of Inflammation, Immunology and Infectious Disease, College of Veterinary Medicine, Purdue University, 625 Harrison St., West Lafayette, IN 47907, USA.
An effective universal influenza vaccine is urgently needed to overcome the limitations of current seasonal influenza vaccines, which are ineffective against mismatched strains and unable to protect against pandemic influenza. In this study, bovine and human adenoviral vector-based vaccine platforms were utilized to express various combinations of antigens. These included the H5N1 hemagglutinin (HA) stem region or HA2, the extracellular domain of matrix protein 2 of influenza A virus, HA signal peptide (SP), trimerization domain, excretory peptide, and the autophagy-inducing peptide C5 (AIP-C5).
View Article and Find Full Text PDFExploring Future Pandemic Preparedness Through the Development of Preventive Vaccine Platforms and the Key Roles of International Organizations in a Global Health Crisis.
Vaccines (Basel)
January 2025
Pharmaceutical Regulatory Affairs, Department of Pharmaceutical Industry, Graduate School, Chung-Ang University, Seoul 06974, Republic of Korea.
The emergence of more than 40 new infectious diseases since the 1980s has emerged as a serious global health concern, many of which are zoonotic. In response, many international organizations, including the US Centers for Disease Control and Prevention (CDC), the World Health Organization (WHO), and the European Center for Disease Prevention and Control (ECDC), have developed strategies to combat these health threats. The need for rapid vaccine development has been highlighted by Coronavirus disease 2019 (COVID-19), and mRNA technology has shown promise as a platform.
View Article and Find Full Text PDFPeripherally administered TNF inhibitor is not protective against α-synuclein-induced dopaminergic neuronal death in rats.
Neurobiol Dis
January 2025
Department of Biomedicine & Danish Research Institute of Translational Neuroscience - DANDRITE, Aarhus University, 8000 Aarhus, Denmark. Electronic address:
The underlying cause of neuronal loss in Parkinson's disease (PD) remains unknown, but evidence implicates neuroinflammation in PD pathobiology. The pro-inflammatory cytokine soluble tumor necrosis factor (TNF) seems to play an important role and thus has been proposed as a therapeutic target for modulation of the neuroinflammatory processes in PD. In this regard, dominant-negative TNF (DN-TNF) agents are promising antagonists that selectively inhibit soluble TNF signaling, while preserving the beneficial effects of transmembrane TNF.
View Article and Find Full Text PDFUnexpected renal side effects of mRNA COVID-19 vaccines; a single-center experience and short review.
Am J Med Sci
January 2025
Department of Internal Medicine and Oncology, Faculty of Medicine, Semmelweis University, Budapest, Hungary.
Background: In late 2019, the World Health Organization declared Coronavirus disease 2019 a global emergency. Since then, many vaccines have been developed to combat the pandemic. Millions of people have received one of the approved COVID-19 vaccines; unfortunately, some adverse events also have been recorded.
View Article and Find Full Text PDFEvaluating Antigen- and Vector-Specific Immune Responses of a Recombinant Pichinde Virus-Based Vaccine Expressing the Lymphocytic Choriomeningitis Virus Nucleoprotein.
Vaccines (Basel)
December 2024
Department of Veterinary and Biomedical Sciences, College of Veterinary Medicine, University of Minnesota, Saint Paul, MN 55108, USA.
Background: Live viral vector-based vaccines are known to elicit strong immune responses, but their use can be limited by anti-vector immunity. Here, we analyzed the immunological responses of a live-attenuated recombinant Pichinde virus (PICV) vector platform (rP18tri).
Methods: To evaluate anti-PICV immunity in the development of vaccine antigen-specific immune responses, we generated a rP18tri-based vaccine expressing the lymphocytic choriomeningitis virus (LCMV) nucleoprotein (NP) and administered four doses of this rP18tri-NPLCMV vaccine to mice.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!