Assessing the silencing effect of siRNA is crucial to confirm its effectiveness in downregulating target gene expression. Several methods can be used to evaluate the silencing efficiency of siRNA. Below are the most common approaches: 

Designing effective siRNA involves several important factors to ensure high silencing efficiency, specificity, and minimal off-target effects. Below are the key considerations when designing siRNA: 

Yes, several RNA-based therapeutics using GalNAc conjugation have been approved for clinical use, particularly for liver-targeted therapies. Some of the approved drugs that utilize GalNAc for targeted delivery include: 

GalNAc plays a crucial role in enhancing the targeted delivery of small nucleic acid drugs, particularly for liver-targeted therapies. Here's how it works: 

Various modifications are applied to small nucleic acids to enhance their stability, reduce immunogenicity, and improve targeting. Below are the most commonly used modifications and their primary functions: