| 초록 |
In recent years, RNA therapeutics have received tremendous attention as a tool to regulate gene expression in patients. These approaches include the regulation of abnormal gene expression by short interfering RNA (siRNA) and messenger RNA (mRNA). To fully realize the potential of RNA therapeutics, an efficient in vivo delivery system is of the utmost importance. Ionizable lipid nanoparticles (LNPs) have been widely utilized for the systemic delivery of RNA therapeutics. Here we report the engineered LNPs for the targeted delivery of RNA into hepatocytes and LSECs. Targeted delivery of RNA to LSECs was further investigated using active ligands. Incorporation of mannose allowed the selective delivery of RNA to LSECs, while minimizing the unwanted cellular uptake by hepatocytes. These results demonstrate that engineered LNPs have great potential for the cell type specific delivery of RNA into the liver and other tissues. |
