Non-viral gene delivery is currently a hot subject for its relative safety and simplicity of use; however, it is still far from being ideal enough to be clinically used for its comparatively lower efficiency than viral gene delivery. To improve the efficiency of non-viral gene delivery needs a comprehensive understanding of the uptake mechanisms. Macromolecules are internalized into cells by a variety of mechanisms, and their intracellular fates are usually relevant with the uptake pathways. The uptake pathways of non-viral gene complexes are usually determined by not only the gene/carrier interaction but also by the interaction between complexes and target cells. The best-characterized uptake pathway is the so-called clathrin-mediated endocytic pathway. However, there are numerous updates of knowledge about endocytic pathways and even non-endocytic pathways in recent years with the development of novel technologies for tracking and inhibiting. In this review, we will try to sort out our current understanding of the uptake mechanisms of non-viral gene delivery. In addition, factors for pathway selection are summarized in the third section. Finally, the useful inhibitors or tools for the study of these pathways will also be concluded in the last section.
Download full-text PDF |
Source |
---|---|
http://dx.doi.org/10.1016/j.jconrel.2011.09.093 | DOI Listing |
Mol Ther Nucleic Acids
March 2025
Department of Biology, Concordia University, Montreal, QC H4B 1R6, Canada.
Gene therapy targeting ischemic heart disease is a promising therapeutic avenue, but it is mostly restricted to viral-based delivery approaches which are limited due to off-target immunological responses. Focused ultrasound presents a non-viral, image-guided technique in which circulating intravascular microbubble contrast agents can reversibly enhance vascular permeability and gene penetration. Here, we explore the influence of flow rate on the microbubble-assisted delivery of miR-126, a potent pro-angiogenic biologic, using a custom acoustically coupled pressurized mesenteric artery model.
View Article and Find Full Text PDFInt J Biol Macromol
January 2025
Institute of Organic Chemistry and Macromolecular Chemistry (IOMC), Friedrich Schiller University Jena, Humboldtstr. 10, D-07743 Jena, Germany; Jena Center for Soft Matters (JCSM), Friedrich Schiller University Jena, Philosophenweg 7, D-07743 Jena, Germany. Electronic address:
Nanomedicine, particularly gene delivery, holds immense potential and offers promising therapeutic options. Non-viral systems gained attention due to their binding capacity, stability and scalability. Among these, natural polysaccharides, such as pullulan, are advantageous in terms of sustainability, biocompatibility and potential degradability.
View Article and Find Full Text PDFBiotechnol Prog
January 2025
Department of Life Sciences, Albany College of Pharmacy and Health Sciences, Albany, New York, USA.
Cell and gene therapy (CGT) products are emerging and innovative biopharmaceuticals that hold promise for treating diseases that are otherwise beyond the scope of conventional medicines. The evolution of CGT from a research idea to a promising therapeutic product is due to the complementary advancements across various scientific disciplines. First, the innovations and advancements in gene editing and delivery technology have provided fundamental tools to manipulate genes and cells for therapeutic pursuits.
View Article and Find Full Text PDFRegen Ther
March 2025
Pediatric Cell and Gene Therapy Research Center, Gene, Cell & Tissue Research Institute, Tehran University of Medical Science, Tehran, Iran.
Gene therapy (GT) as a groundbreaking approach holds promise for treating many diseases including immune deficiencies and blood disorders. GT can benefit patients suffering from these diseases, especially those without matched donors or who are at risk after hematopoietic stem cell transplantation (HSCT). Due to all the advances in the field of GT, its main challenge is still gene delivery.
View Article and Find Full Text PDFInt J Pharm
January 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 PDFEnter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!