@article{eaefb25b59864e339958bc092dc7b55f,
title = "Acetyl-4′-phosphopantetheine is stable in serum and prevents phenotypes induced by pantothenate kinase deficiency",
abstract = "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.",
author = "{Di Meo}, Ivano and Cristina Colombelli and Balaji Srinivasan and {De Villiers}, Marianne and Jeffrey Hamada and Jeong, {Suh Y.} and Rachel Fox and Woltjer, {Randall L.} and Tepper, {Pieter G.} and Lahaye, {Liza L.} and Emanuela Rizzetto and Harrs, {Clara H.} and {De Boer}, Theo and {Van Der Zwaag}, Marianne and Branko Jenko and Alen {\v C}usak and Jerca Pahor and Gregor Kosec and Grzeschik, {Nicola A.} and Hayflick, {Susan J.} and Valeria Tiranti and Sibon, {Ody C.M.}",
note = "Funding Information: This work was supported by the European Commission{\textquoteright}s Seventh Framework Programme [grant number FP7/2007–2013, HEALTH-F2–2011, grant agreement number 277984, TIRCON] to V.T., S.H., G.K. and O.C.M.S., by a GUIDE research school grant to B.S., a VICI grant to O.S (NWO-grant 865.10.012), a Telethon-Italia grant (Grants no. GGP16234) to V.T., the Mariani Foundation of Milan and a grant from AISNAF Foundation Italy to I.D.M and a “KROP” grant to Acies Bio (Contract No. C2130-14-090124), awarded by the European Social Fund and the Ministry of Economic Development and Technology of Slovenia. Part of the work has been performed at the UMCG Microscopy and Imaging Center (UMIC), which is sponsored by NWO-grant 175–010–2009–023. Publisher Copyright: {\textcopyright} 2017 The Author(s).",
year = "2017",
month = dec,
day = "1",
doi = "10.1038/s41598-017-11564-8",
language = "English (US)",
volume = "7",
journal = "Scientific Reports",
issn = "2045-2322",
publisher = "Nature Publishing Group",
number = "1",
}