Award
National Institute of Neurological Disorders and Stroke 5R01NS134932-03
Harnessing mitochondria transfer pathways to ameliorate Leigh Syndrome-like disease
Recipient
Washington University, Saint Louis, MO
Award Amount
$584,115.00
Ceiling
$584,115.00
Awarded
June 30, 2026
Identifier
5R01NS134932-03
Research grant to develop therapeutic strategies involving mitochondrial transfer and bone marrow transplantation to treat Leigh Syndrome, an inherited mitochondrial disease.
Description
This project aims to develop two therapeutic strategies: (1) postnatal administration of exogenous mitochondria, and (2) bone marrow transplantation to provide a systemic source of healthy mitochondria. The study will investigate how these approaches improve morbidity and mortality in Leigh Syndrome, an inherited mitochondrial disease with prominent neurologic symptoms and childhood mortality, with no effective therapies currently available.