Intracranial delivery of neuron-preferential lipid nanoparticles for gene-editing activity in mouse brain
Lipid nanoparticles (LNPs) are promising non-viral vectors for central nervous system (CNS) gene therapy, but effective delivery is limited by the blood-brain barrier and cell-type specificity. Here, we combined intracranial delivery with high-throughput, cluster-based screening and machine learning to identify LNP for...
S. S. Cai, Cody Slater, Veronica E. Farag et al.
· Cell Reports Physical Scienc... · 0 citations