A Vision of Future Gene Therapy: Non-Viral Nanovectors
Recent developments in research have highlighted the potential of non-viral nanoparticles as vectors in gene therapy. Gene therapy typically refers to the use of nucleic acids, such as plasmids and siRNA, to genetically modify patient’s cells.¹ Traditionally, viral vectors have been pursued as gene delivery vehicles, but a variety of risks and problems associated with them have limited their large-scale use clinically.
There are two main approaches to gene therapy:
The first approach, gene augmentation therapy, involves gene replacement or addition to enhance the expression of a specific missing or malfunctioning gene through the administration of a suitable pDNA.¹ The second approach, gene silencing, is conducted by RNA interference using an siRNA molecule derived from DNA plasmids to inhibit the expression of undesirable genes.¹
Non-viral nanovectors include two main categories: physical transmission and chemical transfection:
Physical transmission involves the injection of naked DNA alone or in combination with other physical methods. Chemical transfection involves the electrostatic combination of liposomes or cationic polymers with a nucleic acid molecule followed by the transfer of the nucleic acid molecule into the cells.¹
Some advantages of non-viral nanovectors include the lower biosafety risk, more controllable chemical structure, wider material source, and ease for mass production. However, despite great potential for gene therapy, the clinical applications and in vivo of non-viral nanovectors still face a few challenges. In face of potential challenges, non-viral nanovectors are “one of the foundational pillars of gene therapy,” and hold great promise for the future.¹
Purilogics develops ion exchange and HIC membrane adsorbers which can help with non-viral gene therapy nanocarrier purification. Purilogics can also develop a custom affinity membrane to fit your needs. Send an email to bd@purilogics.com to learn more!
Check out our previous posts to read more about our membrane adsorbers:
How Does Flow Rate Affect the Binding Capacity of Purexa™ Membranes?
What Differentiates Us from Other Membrane Chromatography Products
References
1. Lin, G., Li, L., Panwar, N., Wang, J., Tjin, S. C., Wang, X., & Yong, K. T. (2018). Non-viral gene therapy using multifunctional nanoparticles: status, challenges, and opportunities. Coordination Chemistry Reviews, 374, 133-152.