Therapy for mitochondrial fatty acid beta-oxidation and transport disorders

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United States of America Patent

PATENT NO 11813258
APP PUB NO 20210308128A1
SERIAL NO

17344084

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Abstract

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Methods of treating mitochondrial fatty acid β-oxidation and/or transport disorders arising from mutant proteins in the mitochondrial fatty acid β-oxidation and transport metabolic pathways in patients are provided. The methods modulate the mitochondrial fatty acid β-oxidation pathway at the last step so that the product of the mutant protein accumulates and stabilizes the mutant protein and/or the substrate(s)/product(s) of the down stream reactions accumulate and possibly bind to allosteric sites on the mutant protein to stabilize it. Trimetazidine pharmacodynamics function as such in the β-oxidation pathway. Further, a synergistic effect is observed where trimetazidine and PPARδ agonist combination enhanced enzyme activity and presence significantly more than either alone.

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Patent Owner(s)

Patent OwnerAddress
UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION130 THACKERAY AVENUE 1ST FLOOR GARDNER STEEL CONFERENCE CENTER PITTSBURGH PA 15260

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Mohsen, Al-Walid A Gibsonia, US 11 1
Vockley, Gerard Pittsburgh, US 11 6

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