Arthur Burghes

Professor

Ohio State University College of Medicine

Position in Extended Committee - Preclinical Expert

Arthur completed his BS in Cellular & Microbial Biology in 1978 at the University of Calgary and his PhD in Biochemistry at University of London.

Arthur has served on numerous Ph.D. and candidacy exam committees for many students. He has also served on many University and College of Medicine committees. He is on the Ohio State University and Nationwide Children’s Hospital Muscle executive committee and chair of the Muscle Group fellowship committee.

Arthur’s work mainly focuses on the molecular understanding of genetic neuromuscular disorders, in particular, on unraveling the molecular biology of Spinal Muscular Atrophy (SMA). His lab has developed an animal model of SMA in the mouse and shown that high copy number of the SMN2 gene can rescue the SMA mouse. They are using this animal model of SMA to understand why motor neurons are affected and to develop treatments for SMA. A high throughput screen to identify compounds that can activate SMN has been started. It is hoped that these compounds can act as therapeutic reagents for SMA.

Arthur’s lab is also developing gene therapy methods for treatment of Duchenne Muscular Dystrophy (DMD) and is particularly interested in developing strategies that allow Adeno Associated Virus to be used for gene therapy of DMD.