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. LNPs are mainly composed of ionizable lipid or lipid like materials with helper lipid, cholesterol, and polyethylene glycol (PEG)-lipid. Although LNPs are particularly advantageous for in vivo delivery, systemic delivery of RNA therapeutics other than liver hepatocytes remains highly challenging. Ionizable lipid nanoparticles (LNPs) have been widely utilized for in vivo delivery of RNA therapeutics into the liver. Here we report the engineered LNPs for the targeted delivery of RNA into hepatocytes and LSECs. |