siRNA (small interfering RNA) is a powerful tool for gene silencing, and it has a wide range of applications in both research and therapeutic contexts. Here are the key applications:
siRNA (small interfering RNA) and sgRNA (single guide RNA) are both short
RNA molecules involved in RNA-mediated gene silencing, but they function
in different RNA interference pathways and have distinct roles. Here are
the key differences:
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: