Abstract
Studies aimed at supporting different treatment approaches for pantothenate kinase-associated neurodegeneration (PKAN) have revealed the complexity of coenzyme A (CoA) metabolism and the limits of our current knowledge about disease pathogenesis. Here we offer a foundation for critically evaluating the myriad approaches, argue for the importance of unbiased disease models, and highlight some of the outstanding questions that are central to our understanding and treating PKAN.
Original language | English (US) |
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Pages (from-to) | 283-291 |
Number of pages | 9 |
Journal | Molecular Genetics and Metabolism |
Volume | 137 |
Issue number | 3 |
DOIs | |
State | Published - Nov 2022 |
Keywords
- Brain iron
- Coenzyme A
- Mitochondrial acyl carrier protein
- PKAN
- Pantothenate kinase
- Phosphopantetheinylation
ASJC Scopus subject areas
- Endocrinology, Diabetes and Metabolism
- Biochemistry
- Molecular Biology
- Genetics
- Endocrinology