Team:NYC B1O/Implementation
Proposed Implementation:
Treatment for neurodegenerative disease is still in its infancy and has yet to show truly effective results. We envision our project treating any of the 10 million patients suffering from Parkinson’s disease at any stage in the real world. However, our protein constructs would need to be delivered into neurons. We have considered two ways of doing this: using nanoparticles to surround either the mRNA form or plasmid form of our protein-expressing constructs, or injecting a viral vector for the expression our protein constructs into the brain. Nanoparticles are more convenient to deliver our solution than through a viral vector as it does not require invasive surgery to open up the brain for an injection to be made. However, nanoparticles currently do not have data demonstrating their safety when targeting the central nervous system, and they themselves may be toxic to the brain due to their formulation. Nanoparticles also allow us to inject the mRNA coding for our solution and is safer than a viral vector form in this aspect as our solution will not be integrated into neuronal genomes and possibly cause activation of proto-oncogenes or insertional mutagenesis. Viral vectors are not without benefits, though. The use of viral vectors are more established in the scientific community and there are several clinical trials undergoing using viral vectors to deliver transgenes. The results of these trials can be used to infer the safety of using viral vectors. Our project can serve as a proof of concept for other researchers to follow. Our method of directly targeting protein inclusions may be applied to other neurodegenerative diseases that involve protein aggregation. Our project can be extended to proteinopathies in general, especially diseases that don’t show genetic relationships.