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Emory
I have conducted basic, translational, and clinical neuroscience in six laboratories over a 16-year period prior to launching an independent laboratory in 2001. My research evolved from an interest in neuroplasticity applied to emotion, learning, and memory into a focus on repair of the nervous system for traumatic and degenerative disease. My laboratory focuses on the development of gene and cellular therapies for neurodegenerative and functional diseases of the nervous system. In parallel, the Boulis laboratory focuses on the development of tissue-specific targeting strategies. These approaches are designed to deliver molecular therapeutics to an anatomically defined site of interest. Much of this effort has concentrated on motor neuron-specific gene delivery. In 2004, I began to concentrate on the development of surgical techniques for brain and spinal cord therapeutics delivery. In the context of this work, I developed techniques and devices allowing for accurate human spinal cord transplantation and have provided support for a variety of teams interested in spinal cord transplantation in the United States and Europe for ALS, Spinal Cord Injury, and Transverse Myelitis. My laboratory published the first reports of CSF mediated gene delivery to the CNS using particular serotypes of Adeno-associated Virus. These observations laid the foundation for numerous clinical trials in gene replacement and in particular for motor neuron gene delivery in Spinal Muscular Atrophy and Familial ALS. Current efforts in the laboratory have expanded to include the application of lentiviral CNS gene delivery to glioma modeling in rodents and pigs.
Disclosure(s): BrainTrust Bio: Ownership Interest (Ongoing); BrainXell: Consultant (Ongoing); Kriya Therapeutics: Consultant (Ongoing); Novonordisk: Consultant (Ongoing); Trames: Consultant (Ongoing); UCB: Consultant (Ongoing); Uniqure: Consultant (Ongoing)
Are We Making Progress? Stem Cells in Motor Neuron Disorders
Friday, February 21, 2025
4:27 PM – 4:35 PM EST