The NextGen Precision Health Discovery Series provides learning opportunities for èappSystem faculty and staff across disciplines, the statewide community and our other partners to learn about the scope of precision health research and identify potential collaborative opportunities. The series consists of monthly lectures geared toward a broad multidisciplinary audience so all can participate and appreciate the spectrum of precision health efforts.
Information about this upcoming talk, including continuing education, is available below.
“The Holy Grail: Therapy and Mechanism of Spinal Muscular Atrophy”
Speaker: Arthur Burghes, Ph.D., Affiliated Professor, Molecular & Cellular Biochemistry; Professor, Biological Chemistry and Pharmacology, College of Medicine and College of Arts & Sciences, The Ohio State University
Date: March 12, 2024, noon-1 p.m.
Location: Roy Blunt NextGen Precision Health Building, Atkins Family Seminar Room
Description
Spinal Muscular Atrophy (SMA) research has generated three therapies for SMA that clearly work, and this talk presents how this was done. With the development of therapy, we need to know how these can be improved and how reduced levels of SMN give rise to this disease. In this regard, suppressor screens can be used to obtain SMN independent therapies and understand the critical function of SMN in SMA.
èapp the Speaker
Professor Arthur Burghes received his Ph.D. in Biochemistry from the University of London. He performed his graduate work at the Hammersmith Hospital, which lies adjacent to Wormwood Scrubs — Her Majesty’s prison service, which he did not attend. He then moved to the Hospital for Sick Children in Toronto under the direction of Dr. Worton and was involved in cloning the dystrophin gene. He then moved to take up an assistant professor position in the Department of Biological Chemistry and Pharmacology where he started his work on Spinal Muscular Atrophy. At The Ohio State University, he has risen through the ranks to professor and undertaken research in SMA that has led to the current era of treatments. In particular, he was involved in early mapping studies to identify the location of the gene, characterization of the role of SMN2 in modifying the disease phenotypes an the development of animal model of SMA both mice and pigs. The mice and pig models were further used by researchers at nationwide Children’s Hospital and the Burghes laboratory to develop gene therapy for SMA. Currently the laboratory is undertaking fundamental studies to determine which functions of SMN are critical to the development of SMA using both suppressor and mutant SMNs.
Reviewed 2024-09-30