M. Winter

Immune profiling and treatment cessation provide mechanistic insights into disease pathogenesis and considerations for translating pre-clinical strategies in Leigh syndrome

Genetic mitochondrial diseases (GMDs) are major challenges to human health accounting for a significant fraction of heritable neurologic diseases, myopathies, and inborn errors of metabolism. Leigh syndrome (LS) is the most common clinical presentation of GMD in pediatric patients. LS is a severe and complex disease fo...