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NextGen Discovery Series – March 12, 2024

The NextGen Precision Health Discovery Series provides learning opportunities for UM System 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.

For questions about this event or any others in the Discovery Series, please reach out to Veronica Lemme at

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


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.

About 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 Arthur Burghesservice, 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 and 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.


Continuing Professional Education Credit

Successful Completion of this activity, which includes participating in the educational offering, participating in the evaluation process and completing the verification of attendance, enables the learner to satisfy the requirements for continuing education.

Nursing Contact Hours

University of Missouri Sinclair School of Nursing is approved as a provider of nursing continuing professional development by the Midwest Multistate Division, an accredited approver by the American Nurses Credentialing Center’s Commission on Accreditation (ANCC). 

1.0 contact hour may be awarded.


The University of Missouri-Columbia School of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians. 

The University of Missouri-Columbia School of Medicine designates this live educational activity for a maximum of 1 AMA PRA Category 1 Credit(s)™. Physicians should only claim the credit commensurate with the extent of their participation in the activity.

Missouri Board of Pharmacy

The Continuing Education Committee of the Missouri Board of Pharmacy has reviewed and approved an application for continuing education for the NextGen Precision Health Discovery-The Holy Grail: Therapy and Mechanism of Spinal Muscular Atrophy session.

Name of Program 

NextGen Precision Health Discovery-The Holy Grail: Therapy and Mechanism of Spinal Muscular Atrophy

Program Number 


Approved Dates: 

March 12, 2024

Approved Contact Hours: One(s) (1) CE(s) per session

Financial Relationships

Current ACCME and Midwest Multistate Division, ANCC rules state that participants in continuing education activities should be made aware of any relevant affiliation or financial interest in the previous 24 months that may affect the planning of an educational activity or a speaker’s presentation(s). Each planning committee member and speaker has been requested to complete a financial relationship reporting form for the NextGen Precision Health Discovery Series. No planning committee member or speaker has a relevant financial interest.