RNAtargeted

RNAtargeted is a field of biomedical research and therapeutics that focuses on influencing RNA molecules to control gene expression and cellular behavior. By acting on transcripts rather than DNA, RNAtargeted approaches can alter splicing, translation, stability, or localization of RNA, with applications in disease treatment and biotechnology.

Key modalities include antisense oligonucleotides (ASOs) that modify RNA splicing or trigger degradation; RNA interference (RNAi) using siRNA or shRNA to silence genes; CRISPR-based RNA targeting such as Cas13 systems; small molecules that bind RNA structures; ribozymes and aptamers that affect RNA function.

Clinically, RNAtargeted therapies include nusinersen (Spinraza), an ASO for spinal muscular atrophy; patisiran (Onpattro), an siRNA drug for hereditary transthyretin amyloidosis; inotersen (Tegsedi), an ASO for transthyretin; eteplirsen (Exondys 51), an ASO intended to modify dystrophin splicing. Ongoing research explores broader indications in genetic diseases, cancer, and viral infections.

Delivery remains a major challenge, with lipid nanoparticles and conjugates improving tissue uptake. Other issues include safety, off-target effects, immunogenicity, manufacturing complexity, and regulatory considerations. Advances in chemistry and delivery platforms aim to broaden tissue distribution and duration of effect.

Looking ahead, RNAtargeted research seeks greater specificity, expanded targeting modalities, and personalized approaches that target patient-specific transcripts, alongside diagnostic tools that monitor RNA biology in real time.