Bongarzone Lab, Department of Anatomy & Cell Biology, College of Medicine
Phone: (312) 355-3952
I have an interest in understanding the mechanisms of neurodegenerative diseases. Currently I am focused on the infantile disease Globoid Cell Leukodystrophy (or Krabbe disease). My work is aimed at both understanding new mechanisms that contribute to the pathophysiology of this disease, along with developing new therapeutic strategies to treat Krabbe patients. We are using a mouse model of Krabbe to optimize a gene therapy regimen that utilizes the adeno-associated virus (AAV) vector to deliver healthy copies of the underlying mutated gene.
Smith, B. R.*, Santos, M. B.*, Marshall, M. S.*, Cantuti-Castelvetri, L., Lopez-Rosas, A., Li, G., Bongarzone, E. R. (2014). Neuronal inclusions of alpha-synuclein contribute to the pathogenesis of Krabbe disease. J Pathol. doi: 10.1002/path.4328. *These authors contributed equally to this work.
Cantuti-Castelvetri L, Maravilla E, Marshall M, Tamayo T, D’auria L, Monge J, Jeffries J, Sural-Fehr T, Lopez-Rosas A, Li G, Garcia K, van Breemen R, Vite C, Garcia J, Bongarzone ER. (2015). Mechanisms of neuromuscular dysfunction in Krabbe disease. J Neurosci. 2015 Jan 28;35(4):1606-16. doi: 10.1523/JNEUROSCI.2431-12.2015
Marshall, Michael and Bongarzone, Ernesto. (2016) Beyond Krabbe’s Disease: The Potential Contribution of Galactosylceramidase Deficiency to Neuronal Vulnerability in Late-Onset Synucleinopathies. J. Neuroscience Research. AccepteI am interested in understanding, on the synaptic, cellular and systems levels, the integrated homeostatic mechanisms that facilitate memory, cognition and complex behavior, as well as the dyshomeostatic events that ultimately lead to neurodegeneration, with a particular focus on Alzheimer’s disease.d 2016 Mar 30 doi: 10.1002/jnr.23751