boulisNicholas Boulis, M.D.

Adjunct Associate Professor of Neurology at U-M

Associate Professor of Neurosurgery at Emory University

Developing Cellular and Molecular Therapies for the Nervous System

Nicholas Boulis' laboratory seeks to develop cellular and molecular therapies for the nervous system. He has worked principally to use stem cells and gene therapy either to keep neurons healthy in the face of neurodegenerative diseases, or adjust their activity to treat diseases that stem from neural overstimulation. His laboratory has focused on spinal motor neurons as both a model and target for curing disease. While keeping motor neurons alive can be directly applied to motor neuron disease (Amyotrophic lateral sclerosis, or ALS, and Spinal Muscular Atrophy,or SMA), these approaches can also be applied to other neurodegenerative diseases like Alzheimer's disease, Parkinson's disease and neuropathy.

In order to preserve the survival of motor neurons, his laboratory has pioneered innovative approaches to the delivery of therapeutic genes to motor neurons, including retrograde transport from nerves and muscle, direct spinal cord injection, and intrathecal administration. This work has culminated in the first human spinal cord stem cell transplantation trial, conducted in ALS patients in collaboration with the University of Michigan. His laboratory is supporting the development of stem cell transplantation trials from multiple teams around the United States and abroad.

His work on controlling neural activity with gene delivery has been funded by two consecutive National Institutes of Health grants. He has explored the delivery of genes to alter neurotransmitter production, presynaptic vesicle docking, and ion channels. He is actively supporting the development of a clinical trial for epilepsy gene therapy.

In addition to basic and translational research, he leads and participates in a variety of groundbreaking clinical trials. In addition to stem cell transplantation, he is conducting human trials in both Alzheimer's disease and Parkinson's disease gene therapy.

 


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