Developing Combinatorial Treatment Options for Spinal Muscular Atrophy

When:  Nov 19, 2019 from 7:30 PM to 8:30 PM (CT)

Location: Geffen Auditorium, Gonda Building, Mayo Clinic

Speaker Barrington Burnett, PhD, is an Associate Professor of Neuroscience and Cell and Molecular Biology in the Department of Anatomy, Physiology, and Genetics at Uniformed Services University in Bethesda, Maryland. His work centers on clarifying the role of protein degradation pathways in the pathogenesis of neurodegenerative diseases.

Dr. Burnett received his PhD degree in pharmacology from the University of Pennsylvania in 2005. His doctoral work provided initial evidence that ataxin 3, the polyglutamine neurodegenerative disease protein is a deubiquitinating enzyme in the ubiquitin-proteasome system that processes misfolded toxic proteins.  In his postdoctoral training at the National Institutes of Health (NIH), Dr. Burnett primarily worked to identify mechanisms that stabilize the survival motor neuron (SMN) protein, which is deficient in spinal muscular atrophy (SMA), a childhood motor neuron disease.  During his time at the NIH, Dr. Burnett developed in vitro and cell culture assays to characterize protein turnover, and he demonstrated that the ubiquitin-proteasome system is a potential therapeutic target for SMA. He was awarded an NIH Directors Innovation Award to support his work on identifying genetic modifiers of SMA.

Since joining the faculty at the Uniformed Services University in 2013, Dr. Burnett has continued to develop therapeutic targets for SMA with rodent and induced pluripotent stem cell models. He identified a novel E3 ligase inhibitor that regulates the turnover of the SMN protein. His laboratory has now identified a small molecule ligase inhibitor that slows SMN degradation and improves the disease phenotype of SMA model mice. In addition, he has expanded his studies to include effects of the proteasome on brain and spinal cord injuries.

Dr. Burnett’s presentation, “Developing Combinatorial Treatment Options for Spinal Muscular Atrophy,” will provide an overview of SMA and therapies currently under clinical consideration, with an emphasis on research targeting the protein degradation system to complement currently approved therapies.


Geffen Auditorium, Mayo Clinic
200 1st Street SW
Rochester, MN 55902


Mike Fallenstein