Cell type-targeting nanoparticles in treating central nervous system diseases: Challenges and hopes

Abstract

Abstract Effective and safe pharmacotherapies for central nervous system (CNS) disorders remain a major obstacle to human health worldwide. Nanotechnology offers promise in addressing this challenge by enabling the transport of large molecules across the blood–brain barrier (BBB) and the delivery of multiple drugs. Numerous studies have demonstrated the efficacy of nanodrugs in animal models of various CNS disorders, including Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, amyotrophic lateral sclerosis, stroke, lysosomal storage disease, and gliomas. Nanoparticles (NPs), tailor-made to enhance drug enrichment locally and promote extended drug release, can prevent neuronal death, decrease neurotoxicity, and inhibit tumor growth. In addition to drug delivery, gene therapy using nanotechnology has emerged as a potentially curative option for neurodegenerative diseases. In this review, we summarize how NPs have successfully crossed the BBB and specifically targeted different cells in various CNS disease models over the past decade. Although nanotechnology holds great promise, several drawbacks and challenges must be addressed before its effective implementation in the treatment of CNS diseases.