{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,7]],"date-time":"2025-11-07T07:44:18Z","timestamp":1762501458570,"version":"build-2065373602"},"reference-count":29,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2016,9,1]],"date-time":"2016-09-01T00:00:00Z","timestamp":1472688000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,9,1]],"date-time":"2016-09-01T00:00:00Z","timestamp":1472688000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2016,4,27]],"date-time":"2016-04-27T00:00:00Z","timestamp":1461715200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001691","name":"Japan Society for the Promotion of Science","doi-asserted-by":"publisher","award":["23390140","23659527","25460389","26293120"],"award-info":[{"award-number":["23390140","23659527","25460389","26293120"]}],"id":[{"id":"10.13039\/501100001691","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001700","name":"Ministry of Education, Culture, Sports, Science and Technology","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001700","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100007077","name":"National Institute of Biomedical Innovation","doi-asserted-by":"publisher","award":["09-15"],"award-info":[{"award-number":["09-15"]}],"id":[{"id":"10.13039\/501100007077","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Biochemistry and Biophysics Reports"],"published-print":{"date-parts":[[2016,9]]},"DOI":"10.1016\/j.bbrep.2016.04.012","type":"journal-article","created":{"date-parts":[[2016,6,11]],"date-time":"2016-06-11T10:37:05Z","timestamp":1465641425000},"page":"157-163","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":2,"special_numbering":"C","title":["Combined replacement effects of human modified \u03b2-hexosaminidase B and GM2 activator protein on GM2 gangliosidoses fibroblasts"],"prefix":"10.1016","volume":"7","author":[{"given":"Keisuke","family":"Kitakaze","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chikako","family":"Tasaki","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Youichi","family":"Tajima","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Takatsugu","family":"Hirokawa","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Daisuke","family":"Tsuji","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hitoshi","family":"Sakuraba","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kohji","family":"Itoh","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.bbrep.2016.04.012_bib1","series-title":"The Metabolic and Molecular Bases of Inherited Disease","first-page":"3827","article-title":"The GM2 gangliosidoses","author":"Gravel","year":"2001"},{"key":"10.1016\/j.bbrep.2016.04.012_bib2","doi-asserted-by":"crossref","first-page":"16276","DOI":"10.1016\/S0021-9258(17)34004-8","article-title":"Expression and specificity of human GM2 activator protein","volume":"269","author":"Wu","year":"1994","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.bbrep.2016.04.012_bib3","doi-asserted-by":"crossref","first-page":"192","DOI":"10.1016\/S0167-4838(97)00002-2","article-title":"Characterization of the affinity of the GM2 activator protein for glycolipids by a fluorescence dequenching assay","volume":"1339","author":"Smiljanic-Georgijev","year":"1997","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.bbrep.2016.04.012_bib4","doi-asserted-by":"crossref","first-page":"1048","DOI":"10.1016\/j.bbrc.2004.09.159","article-title":"An \u03b1-subunit loop structure is required for GM2 activator protein binding by \u03b2-hexosaminidase A","volume":"324","author":"Zarghooni","year":"2004","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.bbrep.2016.04.012_bib5","doi-asserted-by":"crossref","first-page":"1464","DOI":"10.1056\/NEJM199105233242104","article-title":"Replacement therapy for inherited enzyme deficiency\u2013macrophage-targeted glucocerebrosidase for Gaucher's disease","volume":"324","author":"Barton","year":"1991","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.bbrep.2016.04.012_bib6","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1056\/NEJM200107053450102","article-title":"Safety and efficacy of recombinant human \u03b1-galactosidase A replacement therapy in Fabry's disease","volume":"345","author":"Eng","year":"2001","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.bbrep.2016.04.012_bib7","doi-asserted-by":"crossref","first-page":"1017","DOI":"10.1038\/mt.2011.27","article-title":"Therapeutic potential of intracerebroventricular replacement of modified human \u03b2-hexosaminidase B for GM2 gangliosidosis","volume":"19","author":"Matsuoka","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.bbrep.2016.04.012_bib8","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1016\/0304-4165(92)90168-T","article-title":"Generation of one set of monoclonal antibodies specific for a-pathway ganglio-series gangliosides","volume":"1117","author":"Kotani","year":"1992","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.bbrep.2016.04.012_bib9","doi-asserted-by":"crossref","first-page":"372","DOI":"10.1212\/WNL.52.2.372","article-title":"GM2 gangliosidosis AB variant: clinical and biochemical studies of a Japanese patient","volume":"52","author":"Sakuraba","year":"1999","journal-title":"Neurology"},{"key":"10.1016\/j.bbrep.2016.04.012_bib10","doi-asserted-by":"crossref","first-page":"193","DOI":"10.1016\/0378-1119(91)90434-D","article-title":"Efficient selection for high-expression transfectants with a novel eukaryotic vector","volume":"108","author":"Niwa","year":"1991","journal-title":"Gene"},{"key":"10.1016\/j.bbrep.2016.04.012_bib11","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1038\/nmeth.2089","article-title":"NIH Image to ImageJ: 25 years of image analysis","volume":"9","author":"Schneider","year":"2012","journal-title":"Nat. Methods"},{"key":"10.1016\/j.bbrep.2016.04.012_bib12","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1016\/j.cca.2006.10.010","article-title":"Therapeutic evaluation of GM2 gangliosidoses by ELISA using anti-GM2 ganglioside antibodies","volume":"378","author":"Tsuji","year":"2007","journal-title":"Clin. Chim. Acta"},{"key":"10.1016\/j.bbrep.2016.04.012_bib13","doi-asserted-by":"crossref","first-page":"e24657","DOI":"10.1371\/journal.pone.0024657","article-title":"Accelerating protein docking in ZDOCK using an advanced 3D convolution library","volume":"6","author":"Pierce","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.bbrep.2016.04.012_bib14","doi-asserted-by":"crossref","first-page":"913","DOI":"10.1016\/j.jmb.2006.04.004","article-title":"Crystallographic structure of human \u03b2-hexosaminidase A: interpretation of Tay-Sachs mutations and loss of GM2 ganglioside hydrolysis","volume":"359","author":"Lemieux","year":"2006","journal-title":"J. Mol. Biol."},{"key":"10.1016\/j.bbrep.2016.04.012_bib15","doi-asserted-by":"crossref","first-page":"951","DOI":"10.1016\/S0022-2836(03)00794-0","article-title":"Structural analysis of lipid complexes of GM2-activator protein","volume":"331","author":"Wright","year":"2003","journal-title":"Mol. Biol."},{"key":"10.1016\/j.bbrep.2016.04.012_bib16","doi-asserted-by":"crossref","first-page":"1093","DOI":"10.1016\/S0022-2836(03)00216-X","article-title":"Crystal structure of human \u03b2-hexosaminidase B: understanding the molecular basis of Sandhoff and Tay-Sachs disease","volume":"327","author":"Mark","year":"2003","journal-title":"J. Mol. Biol."},{"key":"10.1016\/j.bbrep.2016.04.012_bib17","doi-asserted-by":"crossref","first-page":"691","DOI":"10.1002\/ana.22262","article-title":"Highly phosphomannosylated enzyme replacement therapy for GM2 gangliosidosis","volume":"69","author":"Tsuji","year":"2011","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.bbrep.2016.04.012_bib18","doi-asserted-by":"crossref","first-page":"730","DOI":"10.1093\/hmg\/ddt459","article-title":"Reversibility of neuropathology in Tay-Sachs-related diseases","volume":"23","author":"Cach\u00f3n-Gonz\u00e1lez","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.bbrep.2016.04.012_bib19","doi-asserted-by":"crossref","first-page":"80","DOI":"10.1016\/j.ymgme.2015.05.003","article-title":"Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy","volume":"116","author":"Gray-Edwards","year":"2015","journal-title":"Mol. Genet. Metab."},{"issue":"1","key":"10.1016\/j.bbrep.2016.04.012_bib20","first-page":"81","article-title":"Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease","volume":"22","author":"McCurdy","year":"2015","journal-title":"Gene Ther."},{"key":"10.1016\/j.bbrep.2016.04.012_bib21","doi-asserted-by":"crossref","first-page":"428","DOI":"10.1126\/science.276.5311.428","article-title":"Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin","volume":"276","author":"Platt","year":"1997","journal-title":"Science"},{"key":"10.1016\/j.bbrep.2016.04.012_bib22","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1016\/j.ymgme.2009.06.005","article-title":"Substrate reduction therapy in juvenile GM2 gangliosidosis","volume":"98","author":"Maegawa","year":"2009","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.bbrep.2016.04.012_bib23","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1016\/j.ymgme.2010.09.004","article-title":"An open-label Phase I\/II clinical trial of pyrimethamine for the treatment of patients affected with chronic GM2 gangliosidosis (Tay-Sachs or Sandhoff variants)","volume":"102","author":"Clarke","year":"2011","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.bbrep.2016.04.012_bib24","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1002\/cne.10578","article-title":"Insulin-like growth factor-II\/mannose-6-phosphate receptor: widespread distribution in neurons of the central nervous system including those expressing cholinergic phenotype","volume":"458","author":"Hawkes","year":"2003","journal-title":"J. Comp. Neurol."},{"key":"10.1016\/j.bbrep.2016.04.012_bib25","doi-asserted-by":"crossref","first-page":"8325","DOI":"10.1021\/bi970571c","article-title":"Two mechanisms for the recapture of extracellular GM2 activator protein: evidence for a major secretory form of the protein","volume":"36","author":"Rigat","year":"1997","journal-title":"Biochemistry"},{"key":"10.1016\/j.bbrep.2016.04.012_bib26","doi-asserted-by":"crossref","first-page":"571","DOI":"10.1042\/bj2920571","article-title":"Over-expression of a functionally active human GM2-activator protein in Escherichia coli","volume":"292","author":"Klima","year":"1993","journal-title":"Biochem. J."},{"key":"10.1016\/j.bbrep.2016.04.012_bib27","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1016\/j.pep.2003.11.010","article-title":"Expression of the GM2-activator protein in the methylotrophic yeast Pichia pastoris, purification, isotopic labeling, and biophysical characterization","volume":"34","author":"Wendeler","year":"2004","journal-title":"Protein Expr. Purif."},{"key":"10.1016\/j.bbrep.2016.04.012_bib28","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1016\/S1046-5928(02)00599-5","article-title":"Expression of recombinant human GM2-activator protein in insect cells: purification and characterization by mass spectrometry","volume":"27","author":"Wendeler","year":"2003","journal-title":"Protein Expr. Purif."},{"key":"10.1016\/j.bbrep.2016.04.012_bib29","doi-asserted-by":"crossref","first-page":"9946","DOI":"10.1039\/C5CC02967H","article-title":"The total chemical synthesis of the monoglycosylated GM2 ganglioside activator using a novel cysteine surrogate","volume":"51","author":"Sato","year":"2015","journal-title":"Chem. Commun."}],"container-title":["Biochemistry and Biophysics Reports"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2405580816300541?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2405580816300541?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,7]],"date-time":"2025-11-07T07:39:39Z","timestamp":1762501179000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2405580816300541"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,9]]},"references-count":29,"alternative-id":["S2405580816300541"],"URL":"https:\/\/doi.org\/10.1016\/j.bbrep.2016.04.012","relation":{},"ISSN":["2405-5808"],"issn-type":[{"type":"print","value":"2405-5808"}],"subject":[],"published":{"date-parts":[[2016,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Combined replacement effects of human modified \u03b2-hexosaminidase B and GM2 activator protein on GM2 gangliosidoses fibroblasts","name":"articletitle","label":"Article Title"},{"value":"Biochemistry and Biophysics Reports","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.bbrep.2016.04.012","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2016 The Author(s). Published by Elsevier B.V.","name":"copyright","label":"Copyright"}]}}