AntiRNAbased
AntiRNA-based, or anti-RNA-based, therapies are approaches that target RNA molecules to modulate gene expression or RNA processing. By binding complementary RNA sequences or using RNA-targeting enzymes, these strategies aim to reduce or alter the production of disease-causing proteins without changing the DNA itself. The main modalities include antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and RNA-targeting systems such as CRISPR-Cas13. ASOs bind target RNA to block translation, modify splicing, or recruit RNase H to degrade the transcript. siRNAs guide RNA-induced silencing complexes to degrade specific mRNAs, thereby lowering protein output. AntimiRs inhibit endogenous microRNAs, impacting post-transcriptional regulation. Some approaches employ chemical modifications and delivery systems to improve stability and cellular uptake.
Applications span basic research and medicine. In therapeutics, several anti-RNA–based drugs have been approved for rare
Delivery remains a central challenge, with lipid nanoparticles and tissue-targeting conjugates (such as GalNAc) improving pharmacokinetics