Personal profile
Biography
Dr. Barrington G. Burnett is a Professor and Vice Chair for Research in the Department of Anatomy, Physiology and Genetics at the Uniformed Services University of the Health Sciences (USU). He earned his Ph.D. in Pharmacology from the University of Pennsylvania and completed a postdoctoral fellowship in Neurogenetics at NINDS/NIH before joining the USU faculty in 2013.
Dr. Burnett's research focuses on the molecular mechanisms of neuromuscular disease, with a primary emphasis on spinal muscular atrophy (SMA). His group was the first to characterize SMN protein turnover, identify the E3 ubiquitin ligase Mindbomb-1 as a critical regulator of SMN degradation, and develop a first-in-class small molecule inhibitor that rescues the SMA phenotype in preclinical models. Beyond this core therapeutic work, his group has contributed to understanding the multi-system nature of SMA, including demonstrating that SMN deficiency impairs myoblast fusion, disrupts intracellular calcium signaling in cardiomyocytes, and drives neuroinflammatory changes in glial cells.
His broader research portfolio addresses the role of the ubiquitin-proteasome system in the central nervous system more widely, including characterizing how proteasome remodeling following traumatic brain injury shapes the neuroinflammatory response, and identifying novel genetic variants associated with hereditary neurological conditions including Charcot-Marie-Tooth disease, hereditary spastic paraplegia, and a recently described neurodevelopmental syndrome.
Dr. Burnett teaches in the medical student curriculum, directs the graduate course on the Molecular Basis of Neuroscience, and leads a session on motor neuron disease for Neurology residents.
Education/Academic qualification
Pharmacology, PhD, Perelman School of Medicine at the University of Pennsylvania
2000 → 2005
Award Date: 16 May 2005
Keywords
- RM Therapeutics. Pharmacology
- Spinal Muscular Atrophy
- Ubiquitin-Proteasome System
- Neuromuscular Disease
- Motor Neuron Disease
- Neuroinflammation
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Collaborations and top research areas from the last five years
Projects
- 4 Finished
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Neuroinflammation and motor neuron loss in SMA
Ebert, A. D. (PI) & Burnett, B. G. (CoPI)
National Institute of Neurological Disorders and Stroke
1/09/23 → 31/08/24
Project: Research
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Calcium Dysregulation and Cell Function in Spinal Muscular Atrophy
Flagg, T. (PI) & Burnett, B. G. (CoPI)
National Institute of Neurological Disorders and Stroke
20/01/21 → 31/12/25
Project: Research
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TARGETING THE UBIQUITIN PROTEASOME SYSTEM TO DEVELOP TREATMENT FOR TRAUMATIC BRAIN INJURIES
Burnett, B. G. (PI)
Center for Neuroscience and Regenerative Medicine
1/08/17 → 30/08/19
Project: Research
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Targeting the Ubiquitin Proteasome System to Treat Spinal Muscular Atrophy
Burnett, B. G. (PI)
National Institute of Neurological Disorders and Stroke
1/04/16 → 31/12/20
Project: Research
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Advancing neurogenetics in Africa: past achievements, current developments and shaping the future
Landouré, G., Yalcouyé, A., Diarra, S., Maiga, A. D. B., Dembélé, M. E., Cissé, C. A. K., Bocoum, A., Cissé, L., Bamba, S., Samassékou, O., Fischbeck, K. H. & Burnett, B. G., Jul 2025, In: Nature Reviews Neurology. 21, 7, p. 383-393 11 p.Research output: Contribution to journal › Review article › peer-review
1 Scopus citations -
Heterozygous UBR5 variants result in a neurodevelopmental syndrome with developmental delay, autism, and intellectual disability
Sabeh, P., Dumas, S. A., Maios, C., Daghar, H., Korzeniowski, M., Rousseau, J., Lines, M., Guerin, A., Millichap, J. J., Landsverk, M., Grebe, T., Lindstrom, K., Strober, J., Ait Mouhoub, T., Zweier, C., Steinraths, M., Hebebrand, M., Callewaert, B., Abou Jamra, R. & Kautza-Lucht, M. & 49 others, , 2 Jan 2025, In: American Journal of Human Genetics. 112, 1, p. 75-86 12 p.Research output: Contribution to journal › Article › peer-review
Open Access4 Scopus citations -
IL-1ra and CCL5, but not IL-10, are promising targets for treating SMA astrocyte-driven pathology
Allison, R. L., Mangione, C. C., Suneja, M., Gawrys, J., Melvin, B. M., Belous, N., LaCroix, M., Harmelink, M., Burnett, B. G. & Ebert, A. D., 5 Feb 2025, In: Molecular Therapy. 33, 2, p. 734-751 18 p.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
Key features of the innate immune response is mediated by the immunoproteasome in microglia
Izadjoo, S., Moritz, K. E., Khayrullina, G., Bergman, E. M., Melvin, B. M., Stinson, M. W., Paulson, S. G., McCormack, N. M., Anderson, K. N., Lewis, L. A., Rotty, J. D. & Burnett, B. G., Dec 2025, In: Scientific Reports. 15, 1, 41349.Research output: Contribution to journal › Article › peer-review
Open Access -
The Ubiquitin E3 Ligase UBE3A Regulates GRIPAP1 and PACSIN1 Proteins Linked to the Endocytic Recycling of AMPA Receptors
Drebushenko, L., Belous, N., Lischka, F. W., Zhou, Q., Malci, A., Sidorov, M. S., Burnett, B. & Doughty, M. L., 2025, In: Molecular and Cellular Biology. 45, 9, p. 353-368 16 p.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations
Press/Media
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Study Results from Uniformed Services University of the Health Sciences Update Understanding of Glutamate Receptors (The Ubiquitin E3 Ligase Ube3a Regulates Gripap1 and Pacsin1 Proteins Linked To the Endocytic Recycling of Ampa Receptors)
Barrington Burnett & Martin Doughty
13/08/25
1 item of Media coverage
Press/Media
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Researchers at Uniformed Services University of the Health Sciences Release New Data on Spinal Muscular Atrophy (Transcriptional Reprogramming In Sma Mouse Hearts Reveals Signatures of Early Heart Failure and Dysregulated Calcium Signaling)
Clifton Dalgard, Barrington Burnett & Thomas Flagg
22/05/25
1 item of Media coverage
Press/Media
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Scripps Research Institute: 'Scavenger' Cells In The Nervous System Linked To Rare Genetic Disease
Dalgard, C., Rotty, J. & Burnett, B.
1/10/22
1 item of Media coverage
Press/Media
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