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: 

Several delivery strategies are employed to overcome the challenges of nucleic acid stability, cellular uptake, and tissue targeting for small nucleic acid drugs. 

Despite their therapeutic potential, small nucleic acid drugs face several significant challenges in development: 

Small nucleic acid drugs exhibit distinct features that set them apart from small molecule drugs and antibody-based therapies.