Our research focuses on identifying how cell biological processes are disrupted in Parkinson's disease and aging, with the overarching goal of our work to identify novel biomarkers and neuroprotective strategies for Parkinson's disease, as well as other neurological disorders. In particular, we examine how lysosomal and glycosphingolipid pathways are altered, as well as mitochondrial function and calcium homeostasis. We use molecular, neurochemical, biochemical and behavioral techniques to understand early neuronal dysfunction in transgenic, toxin and viral overexpression-based animal models of Parkinson's disease. We utilize patient-derived human cells, including induced pluripotent stem cells (iPSCs) made from patients with different genetic forms of Parkinson's disease, for in vitro modeling of disrupted cell organelle function in order to define shared cellular processes and mechanisms underlying Parkinson's disease pathology. Gene delivery and pharmacological tools are used to promote cell biological function, in order to slow or halt the degeneration of neurons in Parkinson's disease.
Our research interests also extend to cell therapy, whereby iPSC- derived midbrain dopamine neurons are used to replace the dopamine neurons that have degenerated in Parkinson's disease. This technique holds great promise for providing long-term synaptic and functional repair. Our studies develop improved methods to generate iPSC-dopamine neuron preparations, which are tested for functional efficacy following transplantation into preclinical Parkinson's disease in vivo models.
Neuroregeneration Research Institute
115 Mill Street
Belmont, MA 02478