HARRINGTON DISCOVERY INSTITUTE AT UNIVERSITY HOSPITALS

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Dr Anderson’s MAPK8Ip3 Research Work: The MAPK8IP3 Axonopathy Therapeutics (MAT) project is a research and development initiative led by Matthew P. Anderson, M.D., Ph.D., at Harrington Discovery Institute at University Hospitals. The project’s primary objective is to develop a viable therapy for individuals suffering from MAPK8IP3 gene mutations, specifically the R578C mutation. This mutation results in significant motor and cognitive deficits associated with the atrophy of peripheral and central white matter axons.
The development strategy utilizes a dual-track approach: Gene Therapy (including AAV-mediated replacement, allele-selective knock-down via ASO or shRNA, and CRISPR editing) and Small Molecule Therapeutics. Key to this program is the establishment of “genetic reversibility”—proving that removing the mutant protein can restore axonal structure and function. The program is structured around rigorous decision gates, aiming for an Investigational New Drug (IND) submission for a lead candidate
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