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Progress in RNA‐Targeted Therapeutics for Human Diseases
Advances in RNA-Based Treatments for Human Diseases
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Abstract
RNA-targeted therapy is reshaping molecular medicine by shifting from a traditional 'protein-centric' view to an 'RNA-regulatory network' paradigm.
- RNA plays essential roles in regulating gene expression, signaling pathways, and modifying gene activity beyond just carrying genetic information.
- The development of RNA therapeutics has accelerated due to advances in high-throughput sequencing and delivery technologies.
- Various RNA modalities, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA), are being explored for applications in gene silencing, editing, and targeted drug delivery.
- Key challenges that may limit broader clinical application include in vivo stability, delivery efficiency, and immune activation.
- Chemical modifications and nanotechnology are improving the specificity and stability of RNA therapeutics.
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