Development of Non-Viral Targeted RNA Delivery Vehicles – A Key Factor in Success of Therapeutic RNA.

Development of Non-Viral Targeted RNA Delivery Vehicles – A Key Factor in Success of Therapeutic RNA.

Publication date: Oct 11, 2024

Decade-long efforts in medicinal biotechnology have enabled large-scale in-vitro production of optimized therapeutic RNA constructs for stable in-vivo delivery and modify the expression of disease-related genes. The success of lipid nanoparticle-formulated mRNA vaccines against Severe acute respiratory syndrome Coronavirus-2 (SARS-Cov2) has opened a new era of RNA therapeutics and non-viral drug delivery systems. The major limiting factor in the clinical translation of RNA-based drugs is the availability of suitable delivery vehicles that can protect RNA payloads from degradation, offer controlled release, and pose minimal inherent toxicity. Unwanted immune response, payload size constraints, genome integration, and non-specific tissue targeting limit the application of conventional viral drug-delivery vehicles. This review summarizes current research on nano-sized drug carriers, including lipid nanoparticles, polymer-based formulations, cationic nanoemulsion, and cell-penetrating peptides, for targeted therapeutic RNA delivery. Further, this paper highlights the biomimetic approaches (i. e., mimicking naturally occurring bio-compositions, molecular designs, and systems), including virus-like particles (VLPs), exosomes, and selective endogenous eNcapsidation (SEND) technology being explored as safer and more efficient alternatives.

Concepts Keywords
Biotechnology
Decade
Efficient
Nanoemulsion
Viral

Semantics

Type Source Name
drug DRUGBANK Spinosad
disease IDO production
pathway REACTOME Translation
pathway REACTOME Release
disease IDO immune response
disease IDO cell

Original Article

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