Table 2. Selected Examples of Nanoparticle-Based RNA Formulations for in Vivo Brain Delivery via Systemic Administrationa.
| formulation | nucleic acid | animal model | target | ref |
|---|---|---|---|---|
| Lipid-Based NPs | ||||
| Angiopep-2- targeted liposomes | GOLPH3siRNA | U87-GFP-Luciferase-bearing BALB/c mouse | LRP-1 | (72) |
| RVG-9r-targeted liposomes | Ataxin3 siRNAs | C57 BL/6 ataxin-3 [Q69]-transgenic | nACh | (73) |
| Calcium phosphate lipid NPs | SOD1 | Transgenic zebrafish | (74) | |
| ASO | ||||
| Polymeric NPs | ||||
| GLUT-1 targeted polymeric NPs | MALAT1-ASO | BALB/c mice | Glucose transporter | (75) |
| PBAE NPs | siRNA | Orthotopic GBM1A mouse model | (76) | |
| Hybrid NPs | ||||
| Angiopep-2 lipid-PLGA NPs | GOLPH3 | Nude U87 xenograft mice | LRP-1 | (77) |
| siRNA | ||||
| Cyclodextrin | ||||
| Transferrin targeted CD | RRM2 | Monkeys | Transferrin receptor | (78) |
| siRNA | ||||
| Exosomes | ||||
| RVG-targeted exosomes | BACE1 | C57BL/6 | nACh | (79) |
| siRNA | ||||
| T7-peptide targeted exosomes | microRNA-21 ASO | Glioblastoma rat model | Transferrin receptor | (80) |
a
Abbreviations: CD, cyclodextrin; GOLPH3, Golgi phosphoprotein 3; LRP-1, low density lipoprotein receptor-related protein 1; nACh, nicotinic acetylcholine receptor; NPs, nanoparticles; PBAE, poly(β-aminoester); RRM2, ribonucleotide reductase subunit M2; RVG, rabies virus glycoprotein; SOD, superoxide dismutase I.