LAUSR

Abstract Accumulation of α-synuclein (SNCA) in the midbrain is considered as a major pathological characteristic of Parkinson's disease (PD). Inhibiting SNCA expression is an efficient approach in PD treatment. Here, we developed a novel brain-targeted ZnO quantum dots (QDs) nanoplatform delivering gene for interfering SNCA expression into the brain for PD treatment. The glutathione (GSH)-modified ZnO QDs composites loading gene, and simultaneously grafting nerve growth factor (NGF) were prepared and used to treat PD models. The fluorescent tracking results indicated that ZnO QDs composites could deliver gene into the brain across blood–brain barrier (BBB) and subsequently release gene through lysosomal escape. Furthermore, in vitro and in vivo results demonstrated that these ZnO QDs composites loading interfering gene and NGF could efficiently provide neuroprotection and reverse neurodegenerative process with low toxicity in PD model treatment. Taken together, this multifunctional ZnO QDs nanoplatform provides a potential theranostic strategy for PD treatment.