RNA Therapeutics Beyond mRNA- siRNA, Antisense Oligonucleotide and microRNA as next generation pharmaceutical modalities- formulation, delivery and clinical progress

Abstract

With highly targeted methods for controlling gene expression and treating a wide range of genetic, metabolic, viral, and cancerous disorders, RNA therapies have become a revolutionary technique in contemporary medicine. The main kinds of RNA (ribonucleic acid) treatments, such as antisense oligonucleotides (ASOs), small interfering RNA (siRNA), and microRNA (miRNA), are thoroughly reviewed in this study along with their methods of action, therapeutic uses, and most recent clinical developments. With a focus on viral and non-viral delivery methods such as lipid nanoparticles, liposomes, polymeric carriers, and viral vectors, this review also covers the formulation and delivery strategies used to enhance the stability, cellular uptake, and target specificity of these compounds. A comprehensive analysis is conducted of current issues, such as nuclease degradation, immune recognition, off-target effects, restricted extrahepatic distribution, and formulation-related hurdles. Furthermore, the expanding clinical value of these technologies is demonstrated by current clinical trials and recent advancements in FDA-approved RNA-based therapies. Promising approaches to circumvent current constraints are also highlighted, such as chemical changes, ligand-targeted delivery systems, formulations based on nanotechnology, and artificial intelligence-assisted therapeutic design. All things considered, RNA therapeutics are a quickly developing discipline with enormous promise to improve precision medicine and offer efficient treatment choices for illnesses that are still challenging to treat with traditional medicines.