Advances in oligonucleotide therapeutics: recent bench-side progress in ASO, siRNA, and aptamer

Key Points

• Recent progress demonstrates the effectiveness of ASO and siRNA in manipulating gene expression, showing promise for various diseases.
• Key evidence shows that oligonucleotide therapeutics can achieve significant modulation of target RNA molecules, improving therapeutic outcomes.
• Assessment using molecular mechanisms across various models highlights the potential of aptamers and their unique properties in drug development.
• May enable a new era in targeted therapy, as oligonucleotides offer specific, effective interventions for previously challenging diseases.