Pantothenate kinase (PanK) is a metabolic enzyme that is the first and rate-controlling step in the only pathway for cellular coenzyme A (CoA) biosynthesis. A rare, life-threatening neurological disorder known as pantothenate kinase-associated neurodegeneration (PKAN), formerly known as Hallervorden-Spatz disease, arises from mutations in the human PANK2 gene. Here, we studied the neurochemical effects of a drug capable of allosterically activating PanK protein isoforms as a potential PKAN therapeutic. We applied 1HMRS to quantify changes in cerebral metabolites including Glx, GABA, lactate, and NAA in a mouse model of CoA deficiency employing the neuron-specific deletion of Pank1 and Pank2 genes.
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