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Funded Studies

Jeffrey Millbrandt, MD, PhD

Chairman and Professor, Department of Genetics at Washington University School of Medicine

Location: St. Louis, MO United States

My major interest is in elucidating molecular mechanisms of neurological diseases. Our overall goal is to identify pathways that can be targeted to develop new treatments for these diseases. The focus of the proposed research is to investigate the axonal protection mediated by Nmnat within the context of trophic factor deprivation induced axonal degeneration, which is thought to underlie a number of neurological disorders. We have made major discoveries including the identification of immediate-early genes (Egr and Nur families), the identification and characterization of the GDNF related neurotrophic factors Neurturin, Persephin and Artemin and many of their receptors, and most recently, the role of the NAD biosynthetic enzyme Nmnat1 in mediating axonal protection against a broad range of neuronal insults. I have been involved in the clinical application of these discoveries, and through our licensee, Ceregene, a second Phase II trial to treat Parkinson’s disease with AAV2-Neurturin is underway.

Associated Grants

  • Protecting innervation: Nmnat1 gene therapy for Parkinson's disease


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