Precision nanomedicine in neurodegenerative diseases

Abstract

The treatment of neurodegenerative diseases remains a tremendous challenge due to the limited access of molecules across the blood-brain barrier, especially large molecules such as peptides and proteins. As a result, at most, a small percentage of a drug that is administered systemically will reach the central nervous system in its active form. Currently, research in the field focuses on developing safer and more effective approaches to deliver peptides and proteins into the central nervous system. Multiple strategies have been developed for this purpose. However, noninvasive approaches, such as nanostructured protein delivery carriers and intranasal administration, seem to be the most promising strategies for the treatment of chronic diseases, which require long-term interventions. These approaches are both target-specific and able to rapidly bypass the blood-brain barrier. In this Perspective, we detail some of these strategies and discuss some of the potential pitfalls and opportunities in this field. The next generation strategies will most likely be more cell-type-specific. Devising these strategies to target the brain may ultimately become a novel therapeutic modality to treat neurodegenerative diseases.