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Abstract
Small interfering RNA (siRNA) has transitioned from an experimental tool to an established therapeutic modality.
- Chemical modifications and improved delivery methods have enhanced siRNA's clinical applicability.
- Lipid nanoparticles (LNP) and N-acetylgalactosamine (GalNAc)-siRNA conjugates are significant contributors to the success of liver-targeted therapies.
- Achieving similar efficiency and selectivity in delivering siRNA to non-liver cells and other tissues is still a major challenge.
- The advancements in siRNA delivery may allow for broader therapeutic applications beyond liver-directed treatments.
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