AI-Validated Brain Targeting mRNA Lipid Nanoparticles with High Neuronal Specificity

Introduction: Nucleic acid therapeutics like mRNA and siRNA offer promising, non-invasive alternatives for treating brain disorders by modulating gene expression. While carrier systems like viral vectors and exosomes show potential, lipid nanoparticles (LNPs) have emerged as a safer and more versatile platform due to their biocompatibility and ability to encapsulate RNA. Incorporating targeting moieties on LNP surfaces can enhance their brain delivery, and AI-based models offer a powerful tool for optimizing targeting strategies to improve precision and efficacy.