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siRNA-based delivery systems: Technologies, carriers, applications, and approved products.
Eur J Pharmacol
February 2025
Pharmaceutical Sciences Research Center, Shiraz University of Medical Sciences, Shiraz, Iran; Department of Pharmaceutics, School of Pharmacy, Shiraz University of Medical Sciences, Shiraz, Iran. Electronic address:
Ribonucleic acid (RNA) therapeutics are a novel category of therapeutic agents that use different types of RNAs to regulate genes and modulate protein synthesis to treat a wide range of diseases. The main advantages of RNA therapeutics over conventional small molecule drugs would be the potential to target undruggable sites, ease of production and faster development process, and longer duration of action. Various types of RNA therapeutics including antisense oligonucleotides (ASO), RNA interference (RNAi), small interfering RNA (siRNA), microRNA (miRNA), and messenger RNA (mRNA), have been developed and used for various clinical applications, especially for gene and vaccine delivery purposes.
View Article and Find Full Text PDFViral vectors: design and delivery for small RNA.
J Med Microbiol
February 2025
UPM-MAKNA Cancer Research Laboratory, Institute of Bioscience, Universiti Putra Malaysia, 43400 UPM Serdang, Selangor, Malaysia.
RNA interference regulates gene expression by selectively silencing target genes through the introduction of small RNA molecules, such as microRNA, small interfering RNA and short hairpin RNA. These molecules offer significant therapeutic potential for diverse human ailments like cancer, viral infections and neurodegenerative disorders. Whilst non-viral vectors like nanoparticles have been extensively explored for delivering these RNAs, viral vectors, with superior specificity and delivery efficiency, remain less studied.
View Article and Find Full Text PDFPDGFR-α shRNA encoded nanoparticle with epithelial mesenchymal transformation interfering for corneal scarring treatment.
Int J Pharm
February 2025
National Engineering Research Center of Ophthalmology and Optometry, School of Biomedical Engineering, School of Ophthalmology and Optometry, Eye Hospital, Wenzhou Medical University, Wenzhou 325027 China. Electronic address:
Maintaining the clarity of the cornea is crucial for optimal vision. Corneal scarring (CS), resulting from corneal inflammation, trauma, or surgery, can lead to a reduction in corneal transparency and visual impairment. While corneal transplantation is the primary method for restoring vision, the limited availability of corneal donor presents a significant challenge on a global scale.
View Article and Find Full Text PDFNon-Viral Systems Based on PAMAM-Calix-Dendrimers for Regulatory siRNA Delivery into Cancer Cells.
Int J Mol Sci
November 2024
A.M. Butlerov Chemistry Institute, Kazan Federal University, 18 Kremlyovskaya Str., 420008 Kazan, Russia.
Cancer is one of the most common diseases in developed countries. Recently, gene therapy has emerged as a promising approach to cancer treatment and has already entered clinical practice worldwide. RNA interference-based therapy is a promising method for cancer treatment.
View Article and Find Full Text PDFRabies virus-mimicking liposomes for targeted gene therapy in Alzheimer's disease.
Int J Pharm
January 2025
Department of Biochemistry, Faculty of Pharmacy, Izmir Katip Celebi University, 35620 Izmir, Turkey.
Article Synopsis
- RNA interference (RNAi) shows promise for treating neurological disorders but faces challenges like barriers in the central nervous system and unstable nucleic acids.
- The study created a virus-like non-viral gene delivery system using rabies virus glycoprotein-decorated liposomes (f(Lipo)-RVG29) specifically for delivering small interfering RNAs (siRNAs) to the brain, targeting Alzheimer's disease.
- The liposomal system effectively protects siRNA, improves gene delivery rates in brain tissue, and significantly reduces BACE1 expression in vivo, indicating its potential as a novel RNAi therapeutic tool.
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