Developing Nanotherapies for Neurodegenerative Diseases: ORMOSIL and Its Potential in Axonal Transport

Abstract

In neurons, essential components packaged into vesicles are transported down microtubules to the ends of axons (synapses) where they are utilized. Components are also transported from the synapse to the cell body. This transport pathway is crucial for normal development, cell survival and plasticity. Recent work has established that defects in transport can contribute to the initiation of neurodegenerative disease, culminating in cell death and degeneration. Thus, delivering therapeutic treatments to an early defect is critical since many current strategies target pathology that occurs at later stages in the disease. Current treatments also affect the entire organism, causing side-effects that are often more deleterious than the disease. This article discusses how engineered synthetic structures can be used to directly target axonal transport – a pathway that is affected during the early stages of disease. Studies in this area will require the exchange of fundamental knowledge between biologists, chemists and engineers to effectively manufacture novel biomaterials for medical use.

Acknowledgements

The author regrets that some work could not be cited due to space limitations. The author thanks the members of the Gunawardena laboratory for helpful discussions, K Kowalski for editorial assistance and P Karunaratne for constant support.

Financial & competing interests disclosure

The development of ideas presented here was supported in part by the JR Oishei Foundation. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.