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Last Updated: April 1, 2026

Claims for Patent: 10,123,985


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Summary for Patent: 10,123,985
Title:Therapeutic strategies for treating mitochondrial disorders
Abstract:Provided herein are methods, compositions, and systems for treating mitochondrial disorders (e.g., MERRF, MELAS, Kearns-Sayre syndrome, chronic progressive external ophthalmoplegia, diabetes mellitus and deafness, lactic acidosis, Leber's hereditary optic neuropathy, Wolff-Parkinson-White syndrome, Leigh syndrome, NARP, myoneurogenic gastrointestinal encephalopathy, mitochondrial DNA depletion syndrome) or neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease) by administering aspartate, or an analog or prodrug thereof, or an agent that increases intracellular levels of aspartate. Pharmaceutical compositions and kits for use in treating mitochondrial disorders and neurodegenerative diseases are also described herein. Also provided are methods for treating disease by modulating the redox state of a cell, and methods of treating a proliferative disease by administering a cytosolic aspartate aminotransferase (GOT1) inhibitor.
Inventor(s):David M. Sabatini, Kivanc Birsoy, Matthew George Vander Heiden, Lucas Bryan Sullivan, Dan Yi Gui
Assignee: Massachusetts Institute of Technology , Whitehead Institute for Biomedical Research
Application Number:US15/177,243

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