McLean Hospital, Center for Neuroregeneration Research
The overall goal of the project in the Neuroregeneration lab is to assess, using preclinical in vivo and in vitro analyses, iPSC-derived neurons (midbrain dopaminergic) as a cell restoration therapy for Parkinson's disease (PD). As many as one million Americans live with PD and approximately 60,000 people are diagnosed with PD each year in the US (2015 Parkinson's Disease Foundation, Inc.). This neurodegenerative disorder caused by the progressive loss of dopaminergic neurons in the substantia nigra pars compacta is characterized by severe motor dysfunction including gait impairment, bradykinesia, resting tremor (Shulman et al., 2011) and no neuroprotective treatments are currently available.
Specifically, this master thesis aims to determine the safety and efficacy of IPSC-derived dopaminergic neurons (human and primate) following xenotransplantation into a rodent model of PD, using a short-term bioassay, and a long-term functional assay.