Background: The lack of an outright treatment for Parkinson's disease (PD) is a pivotal concern in medicine and has driven the search for novel alternatives for treating the disease. Among the proposed approaches, small interfering RNA (siRNA)-based therapy is attracting significant attention as a potential method for the treatment of PD; however, siRNAs delivery possesses potential drawbacks, such as reduced stability in blood circulation and low capacity for reaching the target site.
Objective: This review aims to explore siRNA-based approaches to PD and the latest advances for designing nanoparticles that effectively target siRNAs to the action site and that protect these against degradation in blood circulation.
Results: siRNA-based approaches provide an interesting option for designing new strategies for treating PD through the silencing of genes, whose abnormal expressions contribute to the pathophysiology of the disease; however, siRNA delivery to the brain is a key issue that remains unsolved to date. Current research efforts are focused on designing vectors that effectively transport and protect siRNAs. In this regard, nanoparticles are being developed as carriers for siRNAs with controlled delivery efficiency and low toxicity profiles, and these represent an alternative to common vectors.
Conclusion: Identification of putative gene targets for siRNA therapy of PD has set the pace for researching non-viral vectors; however, the technological aspects for tackling the challenge that siRNAs targeting to the brain represents are essentials. In this respect, the formulation of siRNAs in nanoparticles would avoid harmful side effects, such as immunogenic and oncogenic drawbacks.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.2174/1389450118666170321130003 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Background: The hyperphosphorylation, mislocalization, and aggregation of the microtubule associated protein Tau (MAPT) is a driving force in tauopathies, a group of progressive, neurodegenerative disorders. These pathogenic intracellular aggregates, known as neurofibrillary tangles (NFTs), are a hallmark in several diseases such as frontotemporal dementia, progressive supranuclear palsy, and Alzheimer's Disease. While anti-Tau immunotherapies emphasize the clearance of extracellular Tau aggregates, they do not address the intracellular accumulation of NFTs.
View Article and Find Full Text PDFLight-activated nanocomposite thin sheet for high throughput contactless biomolecular delivery into hard-to-transfect cells.
Analyst
January 2025
Department of Engineering Design, Indian Institute of Technology Madras, India.
High throughput intracellular delivery of biological macromolecules is crucial for cell engineering, gene expression, therapeutics, diagnostics, and clinical studies; however, most existing techniques are either contact-based or have throughput limitations. Herein, we report a light-activated, contactless, high throughput photoporation method for highly efficient and viable cell transfection of more than a million cells within a minute. We fabricated reduced graphene oxide (rGO) nanoflakes that was mixed with a polydimethylsiloxane (PDMS) nanocomposite thin sheet with an area of 3 cm and a thickness of ∼600 μm.
View Article and Find Full Text PDFA Novel Cell-Penetrating Peptide-Vascular Endothelial Growth Factor Small Interfering Ribonucleic Acid Complex That Mediates the Inhibition of Angiogenesis by Human Umbilical Vein Endothelial Cells and in an Ex Vivo Mouse Aorta Ring Model.
Biomater Res
January 2025
Cellular Reprogramming and Embryo Biotechnology Laboratory, Dental Research Institute, Seoul National University School of Dentistry, Seoul 08826, Republic of Korea.
Angiogenesis is mediated by vascular endothelial growth factor (VEGF), a protein that plays a key role in wound healing, inflammatory diseases, cardiovascular processes, ocular diseases, and tumor growth. Indeed, modulation of angiogenesis represents a potential approach to treating cancer and, as such, therapeutic approaches targeting VEGF and its receptors have been widely investigated as part of the broader search for curative interventions. Equally, RNA interference is a powerful tool for treating diseases, but its application as a disease treatment has been limited in part because of a lack of efficient small interfering RNA (siRNA) delivery systems.
View Article and Find Full Text PDFEvaluation of anticancer efficacy of survivin si-RNA functionalized combined drug-loaded mesoporous silica nanoparticles in a lung cancer mouse model.
Naunyn Schmiedebergs Arch Pharmacol
January 2025
Institute of Life Sciences, Nalco Square, Bhubaneswar, Odisha, 751023, India.
Lung cancer continues to be the leading cause of mortality globally. Nanotechnology-mediated targeted drug delivery approach is one of the promising strategies for the treatment of lung cancer. Due to their multifactorial role, mesoporous silica nanoparticles (MSNs), have attracted a lot of attention for drug delivery.
View Article and Find Full Text PDFRNA nanotherapeutics for hepatocellular carcinoma treatment.
Theranostics
January 2025
Center for Nanomedicine and Department of Anesthesiology, Perioperative and Pain Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.
Hepatocellular carcinoma (HCC) remains a leading cause of cancer-related mortality worldwide, particularly due to the limited effectiveness of current therapeutic options for advanced-stage disease. The efficacy of traditional treatments is often compromised by the intricate liver microenvironment and the inherent heterogeneity. RNA-based therapeutics offer a promising alternative, utilizing the innovative approach of targeting aberrant molecular pathways and modulating the tumor microenvironment.
View Article and Find Full Text PDFWant 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!