Compounds for treating mitochondrial dna disorders
AssigneesUCL Business PLC, United Kingdom Research and Innovation
InventorsAntonella Spinazzola, Ian Holt, Boris Pantic, Daniel Ives
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AssigneesUCL Business PLC, United Kingdom Research and Innovation
InventorsAntonella Spinazzola, Ian Holt, Boris Pantic, Daniel Ives
Estimating the term…
There is described a compound for use in the treatment of a mitochondrial DNA disorder, wherein the compound is a glycolysis inhibitor, an inhibitor of glutamine consumption or is L-asparaginase or pegaspargase. The glycolysis inhibitor may be a glucose analogue such as 2-deoxy-D-glucose (2DG) or 5-thioglucose (5TG). Also described is a method of treating a mitochondrial DNA disorder comprising administering a therapeutically effective amount of the compound above to a patient suffering from a mitochondrial DNA disorder. In addition, there is described a method of reducing the mtDNA heteroplasmy in the cells of a patient comprising administering a therapeutically effective amount of the compound above to the patient.
A method of treating a mitochondrial DNA disorder, comprising administering a therapeutically effective amount of a compounds to a patient suffering from a mitochondrial DNA disorder, wherein the compound is a glycolysis inhibitor, an inhibitor of glutamine consumption, L-asparaginase, or pegaspargase, thereby treating the mitochondrial DNA disorder in the patient.
22-27. (canceled)
The method according to claim 27, wherein the compound is administered orally to the patient in the form of a hard or soft capsule, pill, cachet, lozenge, tablet, liquid, powder or granule.
(canceled)
A method of reducing the mtDNA heteroplasmy in cells of a patient, comprising administering a therapeutically effective amount of a compound to a patient in need thereof, wherein the compound is a glycolysis inhibitor, an inhibitor of glutamine consumption, L-asparaginase, or pegaspargase, thereby reducing mtDNA heteroplasmy in the patient.
33-34. (canceled)
A method of reducing the mtDNA heteroplasmy in a cell comprising administering an effective amount of a compound to the cell, wherein the compound is glycolysis inhibitor, an inhibitor of glutamine consumption, L-asparaginase, or pegaspargase, thereby reducing mtDNA heteroplasmy in cell.
Dependencies are inferred from the claim text.