{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,27]],"date-time":"2026-06-27T03:17:32Z","timestamp":1782530252202,"version":"3.54.5"},"reference-count":43,"publisher":"Public Library of Science (PLoS)","issue":"6","license":[{"start":{"date-parts":[[2016,6,24]],"date-time":"2016-06-24T00:00:00Z","timestamp":1466726400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100007136","name":"Secretar\u00eda de Estado de Investigaci\u00f3n, Desarrollo e Innovaci\u00f3n","doi-asserted-by":"publisher","award":["SAF2013-47761-R"],"award-info":[{"award-number":["SAF2013-47761-R"]}],"id":[{"id":"10.13039\/501100007136","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100007136","name":"Secretar\u00eda de Estado de Investigaci\u00f3n, Desarrollo e Innovaci\u00f3n","doi-asserted-by":"publisher","award":["SAF2015-65786-R"],"award-info":[{"award-number":["SAF2015-65786-R"]}],"id":[{"id":"10.13039\/501100007136","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100007136","name":"Secretar\u00eda de Estado de Investigaci\u00f3n, Desarrollo e Innovaci\u00f3n","doi-asserted-by":"publisher","award":["RYC-2011-07643"],"award-info":[{"award-number":["RYC-2011-07643"]}],"id":[{"id":"10.13039\/501100007136","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004587","name":"Instituto de Salud Carlos III","doi-asserted-by":"publisher","award":["PI12\/01097"],"award-info":[{"award-number":["PI12\/01097"]}],"id":[{"id":"10.13039\/501100004587","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004587","name":"Instituto de Salud Carlos III","doi-asserted-by":"publisher","award":["RD12\/0019\/0006"],"award-info":[{"award-number":["RD12\/0019\/0006"]}],"id":[{"id":"10.13039\/501100004587","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Todos somos raros, todos somos u\u00ccnicos","award":["TSR-2014-1"],"award-info":[{"award-number":["TSR-2014-1"]}]},{"DOI":"10.13039\/501100006461","name":"Agencia de Innovaci\u00f3n y Desarrollo de Andaluc\u00eda","doi-asserted-by":"publisher","award":["P10-CTS-6133"],"award-info":[{"award-number":["P10-CTS-6133"]}],"id":[{"id":"10.13039\/501100006461","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100006461","name":"Agencia de Innovaci\u00f3n y Desarrollo de Andaluc\u00eda","doi-asserted-by":"publisher","award":["SAF2013-47761-R"],"award-info":[{"award-number":["SAF2013-47761-R"]}],"id":[{"id":"10.13039\/501100006461","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"DOI":"10.1371\/journal.pone.0158344","type":"journal-article","created":{"date-parts":[[2016,6,24]],"date-time":"2016-06-24T13:39:06Z","timestamp":1466775546000},"page":"e0158344","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":5,"title":["Gene Therapy Corrects Mitochondrial Dysfunction in Hematopoietic Progenitor Cells and Fibroblasts from Coq9R239X Mice"],"prefix":"10.1371","volume":"11","author":[{"given":"Eliana","family":"Barriocanal-Casado","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Cueto-Ure\u00f1a","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karim","family":"Benabdellah","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alejandra","family":"Guti\u00e9rrez-Guerrero","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mari\u00e9n","family":"Cobo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Agust\u00edn","family":"Hidalgo-Guti\u00e9rrez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juan Jos\u00e9","family":"Rodr\u00edguez-Sevilla","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francisco","family":"Mart\u00edn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3355-0298","authenticated-orcid":true,"given":"Luis C.","family":"L\u00f3pez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2016,6,24]]},"reference":[{"issue":"4","key":"ref1","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1007\/s11910-010-0112-8","article-title":"Recent advances in the genetics of mitochondrial encephalopathies","volume":"10","author":"EJ Tucker","year":"2010","journal-title":"Curr Neurol Neurosci Rep"},{"issue":"8","key":"ref2","doi-asserted-by":"crossref","first-page":"978","DOI":"10.1001\/archneurol.2012.206","article-title":"Heterogeneity of coenzyme Q10 deficiency: patient study and literature review","volume":"69","author":"V Emmanuele","year":"2012","journal-title":"Arch Neurol"},{"issue":"6","key":"ref3","doi-asserted-by":"crossref","first-page":"1233","DOI":"10.1093\/hmg\/dds530","article-title":"Dysfunctional Coq9 protein causes predominant encephalomyopathy associated with CoQ deficiency","volume":"22","author":"L Garcia-Corzo","year":"2013","journal-title":"Hum Mol Genet"},{"key":"ref4","article-title":"The clinical heterogeneity of coenzyme Q10 deficiency results from genotypic differences in the Coq9 gene","author":"M Luna-Sanchez","year":"2015","journal-title":"EMBO Mol Med"},{"issue":"3","key":"ref5","doi-asserted-by":"crossref","first-page":"484","DOI":"10.1016\/j.ymthe.2005.11.012","article-title":"Lentiviral vectors for use in the central nervous system","volume":"13","author":"J Jakobsson","year":"2006","journal-title":"Mol Ther"},{"key":"ref6","doi-asserted-by":"crossref","first-page":"32","DOI":"10.3389\/fnmol.2015.00032","article-title":"Gene therapy and peripheral nerve repair: a perspective","volume":"8","author":"SA Hoyng","year":"2015","journal-title":"Front Mol Neurosci"},{"issue":"R1","key":"ref7","doi-asserted-by":"crossref","first-page":"R28","DOI":"10.1093\/hmg\/ddr110","article-title":"Genetic therapy for the nervous system","volume":"20","author":"WJ Bowers","year":"2011","journal-title":"Hum Mol Genet"},{"issue":"9923","key":"ref8","doi-asserted-by":"crossref","first-page":"1138","DOI":"10.1016\/S0140-6736(13)61939-X","article-title":"Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1\/2 trial","volume":"383","author":"S Palfi","year":"2014","journal-title":"Lancet"},{"issue":"6148","key":"ref9","doi-asserted-by":"crossref","first-page":"1233158","DOI":"10.1126\/science.1233158","article-title":"Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy","volume":"341","author":"A Biffi","year":"2013","journal-title":"Science"},{"issue":"7573","key":"ref10","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1038\/nature15818","article-title":"Gene therapy returns to centre stage","volume":"526","author":"L Naldini","year":"2015","journal-title":"Nature"},{"issue":"11","key":"ref11","doi-asserted-by":"crossref","first-page":"3070","DOI":"10.1172\/JCI28873","article-title":"Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice","volume":"116","author":"A Biffi","year":"2006","journal-title":"J Clin Invest"},{"issue":"8","key":"ref12","doi-asserted-by":"crossref","first-page":"1118","DOI":"10.1172\/JCI200419205","article-title":"Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells","volume":"113","author":"A Biffi","year":"2004","journal-title":"J Clin Invest"},{"issue":"8","key":"ref13","doi-asserted-by":"crossref","first-page":"e23734","DOI":"10.1371\/journal.pone.0023734","article-title":"Development of an all-in-one lentiviral vector system based on the original TetR for the easy generation of Tet-ON cell lines","volume":"6","author":"K Benabdellah","year":"2011","journal-title":"PLoS One"},{"issue":"9","key":"ref14","doi-asserted-by":"crossref","first-page":"871","DOI":"10.1038\/nbt0997-871","article-title":"Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo","volume":"15","author":"R Zufferey","year":"1997","journal-title":"Nat Biotechnol"},{"issue":"12","key":"ref15","doi-asserted-by":"crossref","first-page":"956","DOI":"10.1038\/sj.gt.3302259","article-title":"Efficient lentiviral transduction of Herpesvirus saimiri immortalized T cells as a model for gene therapy in primary immunodeficiencies","volume":"11","author":"MG Toscano","year":"2004","journal-title":"Gene Ther"},{"issue":"7","key":"ref16","doi-asserted-by":"crossref","first-page":"e11897","DOI":"10.1371\/journal.pone.0011897","article-title":"Treatment of CoQ(10) deficient fibroblasts with ubiquinone, CoQ analogs, and vitamin C: time- and compound-dependent effects","volume":"5","author":"LC Lopez","year":"2010","journal-title":"PLoS One"},{"issue":"2","key":"ref17","doi-asserted-by":"crossref","first-page":"328","DOI":"10.1016\/j.mito.2012.01.001","article-title":"Microscale oxygraphy reveals OXPHOS impairment in MRC mutant cells","volume":"12","author":"F Invernizzi","year":"2012","journal-title":"Mitochondrion"},{"issue":"1","key":"ref18","doi-asserted-by":"crossref","first-page":"183","DOI":"10.1002\/stem.1821","article-title":"Mesenchymal stromal cells express GARP\/LRRC32 on their surface: effects on their biology and immunomodulatory capacity","volume":"33","author":"AB Carrillo-Galvez","year":"2015","journal-title":"Stem Cells"},{"issue":"6","key":"ref19","doi-asserted-by":"crossref","first-page":"1874","DOI":"10.1096\/fj.07-100149","article-title":"Respiratory chain dysfunction and oxidative stress correlate with severity of primary CoQ10 deficiency","volume":"22","author":"CM Quinzii","year":"2008","journal-title":"FASEB J"},{"issue":"10","key":"ref20","doi-asserted-by":"crossref","first-page":"3733","DOI":"10.1096\/fj.09-152728","article-title":"Reactive oxygen species, oxidative stress, and cell death correlate with level of CoQ10 deficiency","volume":"24","author":"CM Quinzii","year":"2010","journal-title":"FASEB J"},{"issue":"3","key":"ref21","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1172\/JCI29089","article-title":"Prenyldiphosphate synthase, subunit 1 (PDSS1) and OH-benzoate polyprenyltransferase (COQ2) mutations in ubiquinone deficiency and oxidative phosphorylation disorders","volume":"117","author":"J Mollet","year":"2007","journal-title":"J Clin Invest"},{"issue":"1","key":"ref22","doi-asserted-by":"crossref","first-page":"19","DOI":"10.4161\/auto.5.1.7174","article-title":"Coenzyme Q deficiency triggers mitochondria degradation by mitophagy","volume":"5","author":"A Rodriguez-Hernandez","year":"2009","journal-title":"Autophagy"},{"key":"ref23","article-title":"Fatal neonatal encephalopathy and lactic acidosis caused by a homozygous loss-of-function variant in COQ9","author":"K Danhauser","year":"2015","journal-title":"Eur J Hum Genet"},{"issue":"5","key":"ref24","doi-asserted-by":"crossref","first-page":"558","DOI":"10.1016\/j.ajhg.2009.03.018","article-title":"A nonsense mutation in COQ9 causes autosomal-recessive neonatal-onset primary coenzyme Q10 deficiency: a potentially treatable form of mitochondrial disease","volume":"84","author":"AJ Duncan","year":"2009","journal-title":"Am J Hum Genet"},{"issue":"3","key":"ref25","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1067\/mpd.2001.117575","article-title":"Neonatal presentation of coenzyme Q10 deficiency","volume":"139","author":"S Rahman","year":"2001","journal-title":"J Pediatr"},{"issue":"9","key":"ref26","doi-asserted-by":"crossref","first-page":"1091","DOI":"10.1093\/hmg\/ddm058","article-title":"Missense mutation of the COQ2 gene causes defects of bioenergetics and de novo pyrimidine synthesis","volume":"16","author":"JM Lopez-Martin","year":"2007","journal-title":"Hum Mol Genet"},{"issue":"1","key":"ref27","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1007\/s10545-012-9511-0","article-title":"Human neuronal coenzyme Q(10) deficiency results in global loss of mitochondrial respiratory chain activity, increased mitochondrial oxidative stress and reversal of ATP synthase activity: implications for pathogenesis and treatment","volume":"36","author":"KE Duberley","year":"2013","journal-title":"J Inherit Metab Dis"},{"issue":"1","key":"ref28","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1016\/j.abb.2007.02.016","article-title":"Saccharomyces cerevisiae Coq9 polypeptide is a subunit of the mitochondrial coenzyme Q biosynthetic complex","volume":"463","author":"EJ Hsieh","year":"2007","journal-title":"Arch Biochem Biophys"},{"issue":"36","key":"ref29","doi-asserted-by":"crossref","first-page":"31397","DOI":"10.1074\/jbc.M503277200","article-title":"COQ9, a new gene required for the biosynthesis of coenzyme Q in Saccharomyces cerevisiae","volume":"280","author":"A Johnson","year":"2005","journal-title":"J Biol Chem"},{"issue":"52","key":"ref30","doi-asserted-by":"crossref","first-page":"42627","DOI":"10.1074\/jbc.M510768200","article-title":"The Saccharomyces cerevisiae COQ10 gene encodes a START domain protein required for function of coenzyme Q in respiration","volume":"280","author":"MH Barros","year":"2005","journal-title":"J Biol Chem"},{"issue":"3","key":"ref31","doi-asserted-by":"crossref","first-page":"595","DOI":"10.1042\/BJ20121432","article-title":"nde1 deletion improves mitochondrial DNA maintenance in Saccharomyces cerevisiae coenzyme Q mutants","volume":"449","author":"F Gomes","year":"2013","journal-title":"Biochem J"},{"issue":"Suppl","key":"ref32","doi-asserted-by":"crossref","first-page":"S62","DOI":"10.1016\/j.mito.2007.03.007","article-title":"Endogenous synthesis of coenzyme Q in eukaryotes","volume":"7","author":"UC Tran","year":"2007","journal-title":"Mitochondrion"},{"issue":"2","key":"ref33","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1002\/iub.1243","article-title":"Regulation of coenzyme Q biosynthesis in yeast: a new complex in the block","volume":"66","author":"I Gonzalez-Mariscal","year":"2014","journal-title":"IUBMB Life"},{"issue":"6","key":"ref34","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1089\/hum.2013.111","article-title":"Correction of methylmalonic aciduria in vivo using a codon-optimized lentiviral vector","volume":"25","author":"ES Wong","year":"2014","journal-title":"Hum Gene Ther"},{"issue":"4","key":"ref35","doi-asserted-by":"crossref","first-page":"1026","DOI":"10.1002\/art.23283","article-title":"In vivo delivery of lentiviral vectors expressing vasoactive intestinal peptide complementary DNA as gene therapy for collagen-induced arthritis","volume":"58","author":"M Delgado","year":"2008","journal-title":"Arthritis Rheum"},{"issue":"6","key":"ref36","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1089\/hum.2006.17.635","article-title":"Lentiviral vectors mediate efficient and stable gene transfer in adult neural stem cells in vivo","volume":"17","author":"M Geraerts","year":"2006","journal-title":"Hum Gene Ther"},{"issue":"8","key":"ref37","doi-asserted-by":"crossref","first-page":"795","DOI":"10.1038\/gt.2011.24","article-title":"Hematopoietic gene therapy restores thymidine phosphorylase activity in a cell culture and a murine model of MNGIE","volume":"18","author":"J Torres-Torronteras","year":"2011","journal-title":"Gene Ther"},{"issue":"Pt 10","key":"ref38","doi-asserted-by":"crossref","first-page":"2847","DOI":"10.1093\/brain\/awv226","article-title":"Allogeneic haematopoietic stem cell transplantation for mitochondrial neurogastrointestinal encephalomyopathy","volume":"138","author":"JP Halter","year":"2015","journal-title":"Brain"},{"issue":"7263","key":"ref39","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1038\/nature08313","article-title":"Reactive oxygen species prime Drosophila haematopoietic progenitors for differentiation","volume":"461","author":"E Owusu-Ansah","year":"2009","journal-title":"Nature"},{"issue":"4","key":"ref40","doi-asserted-by":"crossref","first-page":"1472","DOI":"10.1016\/j.bbrc.2006.08.020","article-title":"Dynamic changes in mitochondrial biogenesis and antioxidant enzymes during the spontaneous differentiation of human embryonic stem cells","volume":"348","author":"YM Cho","year":"2006","journal-title":"Biochem Biophys Res Commun"},{"issue":"4","key":"ref41","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1016\/j.cmet.2011.08.007","article-title":"Mitochondrial complex III ROS regulate adipocyte differentiation","volume":"14","author":"KV Tormos","year":"2011","journal-title":"Cell Metab"},{"issue":"17","key":"ref42","doi-asserted-by":"crossref","first-page":"1984","DOI":"10.1089\/scd.2015.0076","article-title":"Differentiation of Human Neural Stem Cells into Motor Neurons Stimulates Mitochondrial Biogenesis and Decreases Glycolytic Flux","volume":"24","author":"LC O'Brien","year":"2015","journal-title":"Stem Cells Dev"},{"issue":"7","key":"ref43","doi-asserted-by":"crossref","first-page":"1022","DOI":"10.3324\/haematol.2012.079244","article-title":"Cord blood-derived CD34+ hematopoietic cells with low mitochondrial mass are enriched in hematopoietic repopulating stem cell function","volume":"98","author":"D Romero-Moya","year":"2013","journal-title":"Haematologica"}],"container-title":["PLOS ONE"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/dx.plos.org\/10.1371\/journal.pone.0158344","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2018,10,20]],"date-time":"2018-10-20T09:13:04Z","timestamp":1540026784000},"score":1,"resource":{"primary":{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0158344"}},"subtitle":[],"editor":[{"given":"Ilya","family":"Ulasov","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"editor"}]}],"short-title":[],"issued":{"date-parts":[[2016,6,24]]},"references-count":43,"journal-issue":{"issue":"6","published-online":{"date-parts":[[2016,6,24]]}},"URL":"https:\/\/doi.org\/10.1371\/journal.pone.0158344","relation":{},"ISSN":["1932-6203"],"issn-type":[{"value":"1932-6203","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,6,24]]}}}