What Are the Challenges in Implementing RNA Silencing Therapies in Pediatrics?
While RNA silencing therapies hold promise, there are several challenges:
- [Delivery Systems](https://): Efficiently delivering RNA molecules to specific tissues or cells in the body is challenging. Nanoparticles and viral vectors are being explored as potential delivery systems. - [Off-Target Effects](https://): Ensuring specificity to avoid off-target effects is crucial. Off-target effects can lead to unintended gene silencing and potential side effects. - [Long-term Efficacy](https://): Assessing the long-term efficacy and safety of RNA-based therapies is essential, especially in a pediatric population.
