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Acetyl-4′-phosphopantetheine is stable in serum and prevents phenotypes induced by pantothenate kinase deficiency

Article scientifique 2017 Anglais

Résumé

Coenzyme A is an essential metabolite known for its central role in over one hundred cellular metabolic reactions. In cells, Coenzyme A is synthesized de novo in five enzymatic steps with vitamin B5 as the starting metabolite, phosphorylated by pantothenate kinase. Mutations in the pantothenate kinase 2 gene cause a severe form of neurodegeneration for which no treatment is available. One therapeutic strategy is to generate Coenzyme A precursors downstream of the defective step in the pathway. Here we describe the synthesis, characteristics and in vivo rescue potential of the acetyl-Coenzyme A precursor S-acetyl-4'-phosphopantetheine as a possible treatment for neurodegeneration associated with pantothenate kinase deficiency.

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Meo, I., Colombelli, C., Srinivasan, B., Villiers, M., Hamada, J., Jeong, S., Fox, R., Woltjer, R., Tepper, P., Lahaye, L., Rizzetto, E., Harrs, C., Boer, T., Zwaag, M., Jenko, B., Čusak, A., Pahor, J., Kosec, G., Grzeschik, N., Hayflick, S., Tiranti, V., Sibon, O. (2017). Acetyl-4′-phosphopantetheine is stable in serum and prevents phenotypes induced by pantothenate kinase deficiency. https://doi.org/10.1038/s41598-017-11564-8

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Auteur(s)

Ivano Di Meo Cristina Colombelli Balaji Srinivasan Marianne de Villiers Jeffrey Hamada Suh Young Jeong Rachel Fox Randall L. Woltjer Pieter G. Tepper Liza L. Lahaye Emanuela Rizzetto Clara Harrs Theo de Boer Marianne van der Zwaag Branko Jenko Alen Čusak Jerca Pahor Gregor Kosec Nicola A. Grzeschik Susan J. Hayflick Valeria Tiranti Ody C.M. Sibon

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