Key result
Advanced drug delivery systems enhance RNA therapeutics by preventing degradation and improving targeted cellular uptake.
Why the study?
Effective delivery of RNA-based therapeutics remains a major challenge due to their large size, negative charge, and susceptibility to degradation.
Innovations in viral and non-viral delivery vehicles, particularly lipid nanoparticles, have enabled significant clinical advancements and FDA approvals for RNA-based therapeutics.
May support RNA therapeutic optimization in select settings; leaves open need for prospective trials before practice change.
Background: RNA-based therapeutics, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and messenger RNAs (mRNAs), offer significant promise in treating genetic and acquired diseases by targeting specific RNA sequences, encoding therapeutic proteins, or facilitating genome editing. However, the effective delivery of these RNA therapeutics remains a major challenge due to their large size, negative charge, and susceptibility to degradation. Aim: This review aims to explore advanced drug delivery systems developed to enhance the efficacy of RNA-based therapeutics, focusing on both viral and non-viral methods, and to evaluate the progress and limitations of these systems in clinical applications. Methods: The review synthesizes recent advancements in RNA delivery technologies, including viral vectors, lipid nanoparticles (LNPs), polymer-based nanoparticles, and hybrid systems. It also examines various targeting strategies such as passive and active targeting to improve the specificity and efficiency of RNA delivery. Results: Significant progress has been made with both viral and non-viral delivery systems. Viral vectors, though effective, face challenges related to immunogenicity and production costs. Non-viral systems, particularly lipid nanoparticles and polymer-based carriers, have shown promising results, with several FDA-approved products demonstrating clinical efficacy. Advances in targeting strategies, including ligand-based and antibody-based methods, have improved the precision of RNA delivery.
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Alhabib et al. (2022) conducted a review in Genetic and acquired diseases. Advanced drug delivery systems (viral and non-viral) was evaluated. Advanced drug delivery systems, particularly lipid nanoparticles and polymer-based carriers, enhance the efficacy of RNA-based therapeutics by protecting RNA from degradation and improving targeted cellular uptake.
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