Intranasal gene therapy for the treatment of neurological disorders

Abstract Complex nature of central nervous system (CNS) results in difficulty to pass the drug/gene through BBB in the treatment of neurological disorders. Significant advances in targeted gene delivery have opened up ways of treating several neurological disorders across a variety of diverse modalities ranging from exogenous expression to endogenous suppression of genes. Recent nanotechnology-based advances for improved delivery of gene sequences have centered on extremely specific polymer-based gene delivery, viral vector designs, and plasmid transfection nanoparticle-based therapies. This chapter inculcates various neurological disorders and current approaches for their treatment. It also includes different challenges for intranasal gene delivery to CNS and gene delivery modalities. The authors also concentrated on several ideal properties of nanomaterials for intranasal gene delivery and various strategies involved for efficient nose-to-brain gene delivery. The chapter also put forth various factors affecting gene transportation using various nanomaterials. Nanoparticle-based approaches for intranasal gene delivery with translational medicines in the treatment of neurological disorders were also included.