RNA Combined with Nanoformulation to Advance Therapeutic Technologies

Abstract

Nucleic acid-based therapies have the potential to address numerous diseases that pose significant challenges to more traditional methods. RNA-based therapies have emerged as a promising avenue, utilizing nanoformulation treatments to target a range of pathologies. Nanoformulation offers several advantages compared to other treatment modalities, including targeted delivery, low toxicity, and bioactivity suitable for drug loading. At present, various types of nanoformulations are available, such as liposomes, polymeric nanoparticles (NPs), magnetic NPs, nanoshells, and solid lipid nanoparticles (SLNs). RNA-based therapy utilizes intracellular gene nanoparticles with messenger RNA (mRNA) emerging prominently in cancer therapy and immunotechnology against infectious diseases. The approval of mRNA-based technology opens doors for future technological advancements, particularly self-amplifying replicon RNA (repRNA). RepRNA is a novel platform in gene therapy, comprising viral RNA with a unique molecular property that enables the amplification of all encoded genetic information countless times. As a result, repRNA-based therapies have achieved significant levels of gene expression. In this context, the primary objective of this study is to furnish a comprehensive review of repRNA and its applications in nanoformulation treatments, with a specific focus on encapsulated nanoparticles. The overarching goal is to provide an extensive overview of the use of repRNA in conjunction with nanoformulations across a range of treatments and therapies.

Keywords: RNA replicon therapy; RNA replicon vaccines; self-amplifying RNA.

Figure 1

Overview of the use of nanoformulations associated with repRNA: (a) examples of nanoformulations and forms of association with repRNA; (b) immunomodulatory potential of nanoparticles associated with repRNA and therapeutic application, either by immunostimulation or immunodepression. The information contained in this illustration was based on the work of Blakney [144] and Feng et al. [145]. Created with BioRender.com (accessed on 2 October 2023).