Methods for the treatment of mitochondrial diseases associated with a mutation in SURF 1 or POLG gene resulting in a disruption of mitochondrial oxidative phosphorylation
AssigneeStealth BioTherapeutics Inc.
InventorD. Travis Wilson
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Abstract
The disclosure provides methods of preventing, ameliorating or treating disruption of mitochondrial function and symptoms thereof. The methods provide administering aromatic-cationic peptides in effective amounts to prevent, treat or ameliorate the disruption of mitochondrial oxidative phosphorylation in a cell such as that found in a subject suffering from, or predisposed to a mitochondrial disease or disorder. In some embodiments, the methods comprise administering to a subject suffering from, or at risk for a mitochondrial disease or disorder, an effective amount of an aromatic-cationic peptide to subjects in need thereof.
Claims6
A method for ameliorating a disease selected from the group consisting of Leigh syndrome, Alpers' disease, ataxia-neuropathy disorders, and progressive external ophthalmoplegia in a subject in need thereof, the method comprising:
- administering to the subject a therapeutically effective amount of the peptide D-Arg-2′,6′-Dmt-Lys-Phe-NH2 or a pharmaceutically acceptable salt thereof, wherein the Leigh syndrome is associated with a loss-of-function mutation in the SURF 1 gene resulting in a disruption of mitochondrial oxidative phosphorylation due to impairment of the complete assembly of at least one mitochondrial complex selected from the group consisting of Complex I;
- Complex II;
- Complex III;
- Complex IV, and the Alpers' disease, ataxia-neuropathy disorders, and progressive external ophthalmoplegia are associated with a loss-of-function mutation in the POLG gene resulting in a disruption of mitochondrial oxidative phosphorylation.
Dependencies are inferred from the claim text.