Federal Project Grant Award Summary

Washington University in St. Louis received a $427,625 Project Grant from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853), with a project period from June 1, 2026, through May 31, 2028. This award funds the development of induced pluripotent stem cell (iPSC)-derived disease models to study and evaluate potential treatments for Mitchell Syndrome, a rare autosomal dominant neurodegenerative disorder caused by a gain-of-function variant in the ACOX1 gene. The research will leverage the institution's unique position as a primary treatment and research center for Mitchell Syndrome patients, including access to extensive biofluid biobanks and post-mortem tissues.

The grant supports two primary research deliverables: (1) development of iPSC-derived oligodendrocyte models to evaluate ACOX1 gain-of-function in these cells, and (2) creation of iPSC-derived sensory neuron models to investigate ACOX1 dysfunction in neuronal tissue. Upon completion, the project will deliver two scalable, human-based disease models that recapitulate key pathological features of Mitchell Syndrome for use in mechanistic studies and therapeutic screening, addressing the disease's complex interplay between lipid metabolism, oxidative stress, and neuronal/glial degeneration.