Cockayne Syndrome

MSI PI Christina Pacak (assistant professor, Neurology) has received a new $1.8M grant to develop an efficient manufacturing process of a gene therapy for Cockayne Syndrome, a rare accelerated aging disorder that currently has no treatment. Professor Pacak is the PI on this project along with Dr. George Aslanidi of the Hormel Institute - University of Minnesota, Medical Research Center. The grant was awarded by the Center for Biologics Evaluation and Research at the U.S. Food and Drug Administration. A story about this grant and Professor Pacak can be found on the Medical School’s news website: U of M Faculty-Led Team Awarded $1.8M to Develop Gene Therapy for Cockayne Syndrome.

Professor Pacak uses MSI for various projects that investigate the underlying mechanisms in rare disorders that impact mitochondrial function.