IGERT Trainee Brendan Harmon Receives ASPET Graduate Student Travel Award

Date: 02/02/2011

Brendan Harmon, a doctoral candidate in the Department of Pharmaceutical Sciences, working in Professor Barbara Waszczak’s laboratory had received the American Society for Pharmacology and Experimental Therapeutics (ASPET) Graduate Student Travel Award. Brendan’s doctoral thesis research focuses on “Intranasal Delivery of pGDNF-Expressing Nanoparticles for Parkinson’s Disease”

Delivery of genes encoding therapeutic proteins has potential for treatment of neurodegenerative disorders of the brain. Most studies have used intracerebral injections of viral vectors to achieve CNS transfection. However, safety concerns including invasive surgical methods to introduce the gene to target brain areas decrease clinical applicability of this approach. We have shown successful delivery of neurotrophic proteins to the brain via the intranasal route of administration, which bypasses the blood-brain barrier and avoids systemic absorption. The focus of this work is to determine whether this route can also be utilized for non-viral CNS gene therapy. Copernicus Therapeutics has developed a non-viral DNA nanoparticle delivery technology in which single molecules of DNA are compacted with polycations, such as poly(ethylene glycol) substituted lysine 30-mers (PEG-CK30), formulating nanorods of 8-11 nm. These nanoparticles are non-immunogenic, non-inflammatory and can deliver genes to post-mitotic cells in the brain. In our studies, rats were intranasally administered reporter protein-expressing pDNA compacted with PEG-CK30, sacrificed, and their brains analyzed for protein expression using ELISA and immunohistochemistry. Achieving successful transfection in rat brain provides proof of principle that intranasal delivery can be used for neurotrophic protein expression aimed at treatment of CNS disorders.

Brendan is a Nanomedicine Fellow, supported by Northeastern University’s Interdisciplinary Graduate Education and Research Training (IGERT) program in Nanomedicine Science and Technology. This work is also supported by the Michael J. Fox Foundation for Parkinson’s Research and Copernicus Therapeutics (Cleveland, OH).