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.

Mor Sela, Doctor of Philosophy, Chemical Engineering – Postdoctoral scholar, Bioengineering, Rice University; Dganit Danino, Doctor of Philosophy, Chemical Engineering – Full Professor, 1CryoEM Laboratory of Soft Matter, Department of Biotechnology and Food Engineering 2Guangdong Technion - Israel Institute of Technology, Technion - Israel Institute of Technology; Peleg Hasson, Doctor of Philosophy, Genetics – Full Professor, 5Department of Genetics and Developmental Biology, The Rappaport Faculty of Medicine and Research Institute, Technion – Israel Institute of Technology; Amit Zeisel, Doctor of Philosophy, Systems biology/Data analysis – Assistance Professor, Faculty of Biotechnology and Food Engineering, Technion - Israel Institute of Technology; Kira Radinsky, Doctor of Philosophy, Computer Sciences – Visiting Professor, Computer Sciences, Technion - Israel Institute of Technology; Avi Schroeder, Doctor of Philosophy, Chemical Engineering – Full Professor, Chemical Engineering, Technion - Israel Institute of Technology