{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,24]],"date-time":"2026-03-24T22:35:22Z","timestamp":1774391722850,"version":"3.50.1"},"reference-count":48,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2007,8,1]],"date-time":"2007-08-01T00:00:00Z","timestamp":1185926400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Biochemical and Biophysical Research Communications"],"published-print":{"date-parts":[[2007,8]]},"DOI":"10.1016\/j.bbrc.2007.06.048","type":"journal-article","created":{"date-parts":[[2007,6,19]],"date-time":"2007-06-19T11:09:18Z","timestamp":1182251358000},"page":"375-380","source":"Crossref","is-referenced-by-count":12,"title":["4-Phenylbutyrate rescues trafficking incompetent mutant \u03b1-galactosidase A without restoring its functionality"],"prefix":"10.1016","volume":"360","author":[{"given":"Gary Hin-Fai","family":"Yam","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J\u00fcrgen","family":"Roth","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christian","family":"Zuber","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.bbrc.2007.06.048_bib1","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1016\/j.semcdb.2003.12.010","article-title":"Roles of molecular chaperones in protein misfolding diseases","volume":"15","author":"Barral","year":"2004","journal-title":"Seminars in Cell and Developmental Biology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib2","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1093\/jb\/mvi068","article-title":"Roles of molecular chaperones in endoplasmic reticulum (ER) quality control and ER-associated degradation (ERAD)","volume":"137","author":"Nishikawa","year":"2005","journal-title":"Journal of Biochemical (Tokyo)"},{"key":"10.1016\/j.bbrc.2007.06.048_bib3","doi-asserted-by":"crossref","first-page":"E207","DOI":"10.1038\/35041139","article-title":"Conformation disease","volume":"2","author":"Kopito","year":"2000","journal-title":"Nature Cell Biology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib4","doi-asserted-by":"crossref","first-page":"781","DOI":"10.1034\/j.1600-0854.2002.31103.x","article-title":"Traffic jams II: an update of diseases of intracellular transport","volume":"3","author":"Aridor","year":"2002","journal-title":"Traffic"},{"key":"10.1016\/j.bbrc.2007.06.048_bib5","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1021\/cr000423j","article-title":"Protein N-glycosylation along the secretory pathway: relationship to organelle topography and function, protein quality control, and cell interactions","volume":"102","author":"Roth","year":"2002","journal-title":"Chemical Reviews"},{"key":"10.1016\/j.bbrc.2007.06.048_bib6","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1038\/nrm1052","article-title":"Quality control in the endoplasmic reticulum","volume":"4","author":"Ellgaard","year":"2003","journal-title":"Nature Reviews Molecular Cell Biology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib7","doi-asserted-by":"crossref","first-page":"766","DOI":"10.1038\/ncb0805-766","article-title":"ERAD: the long road to destruction","volume":"7","author":"Meusser","year":"2005","journal-title":"Nature Cell Biology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib8","doi-asserted-by":"crossref","first-page":"701","DOI":"10.1146\/annurev.physiol.67.032003.154107","article-title":"Assembly of functional CFTR chloride channels","volume":"67","author":"Riordan","year":"2005","journal-title":"Annual Review of Physiology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib9","doi-asserted-by":"crossref","first-page":"633","DOI":"10.1172\/JCI118459","article-title":"Congenital sucrase\u2013isomaltase deficiency\u2014identification of a glutamine to proline substitution that leads to a transport block of sucrase-isomaltase in a pre-Golgi compartment","volume":"97","author":"Ouwendijk","year":"1996","journal-title":"Journal of Clinical Investigation"},{"key":"10.1016\/j.bbrc.2007.06.048_bib10","doi-asserted-by":"crossref","first-page":"2267","DOI":"10.1097\/01.ASN.0000027355.41663.14","article-title":"Cell-biologic and functional analyses of five new Aquaporin-2 missense mutations that cause recessive nephrogenic diabetes insipidus","volume":"13","author":"Marr","year":"2002","journal-title":"Journal of the American Society of Nephrology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib11","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1093\/embo-reports\/kvd052","article-title":"Aggresomes and Russell bodies. Symptoms of cellular indigestion?","volume":"1","author":"Kopito","year":"2000","journal-title":"EMBO Reports"},{"key":"10.1016\/j.bbrc.2007.06.048_bib12","doi-asserted-by":"crossref","first-page":"1552","DOI":"10.1126\/science.292.5521.1552","article-title":"Impairment of the ubiquitin-proteasome system by protein aggregation","volume":"292","author":"Bence","year":"2001","journal-title":"Science"},{"key":"10.1016\/j.bbrc.2007.06.048_bib13","doi-asserted-by":"crossref","first-page":"10887","DOI":"10.1073\/pnas.0409283102","article-title":"Formation of morphologically similar globular aggregates from diverse aggregation-prone proteins in mammalian cells","volume":"102","author":"Mukai","year":"2005","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"key":"10.1016\/j.bbrc.2007.06.048_bib14","doi-asserted-by":"crossref","first-page":"1585","DOI":"10.1172\/JCI0216782","article-title":"alpha(1)-Antitrypsin polymerization and the serpinopathies: pathobiology and prospects for therapy","volume":"110","author":"Lomas","year":"2002","journal-title":"Journal of Clinical Investigation"},{"key":"10.1016\/j.bbrc.2007.06.048_bib15","first-page":"173","article-title":"Endocrinopathies in the family of endoplasmic reticulum (ER) storage diseases: disorders of protein trafficking and the role of ER molecular chaperones","volume":"19","author":"Kim","year":"1998","journal-title":"Endocrine Reviews"},{"key":"10.1016\/j.bbrc.2007.06.048_bib16","doi-asserted-by":"crossref","first-page":"100","DOI":"10.2353\/ajpath.2007.060806","article-title":"Aggregated myocilin induces Russell bodies and causes apoptosis: implications for the pathogenesis of myocilin-caused primary open-angle glaucoma","volume":"170","author":"Yam","year":"2007","journal-title":"The American Journal of Pathology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib17","doi-asserted-by":"crossref","first-page":"739","DOI":"10.1146\/annurev.biochem.73.011303.074134","article-title":"The mammalian unfolded protein response","volume":"74","author":"Schroder","year":"2005","journal-title":"Annual Review of Biochemistry"},{"key":"10.1016\/j.bbrc.2007.06.048_bib18","doi-asserted-by":"crossref","first-page":"487","DOI":"10.1146\/annurev.cellbio.21.122303.120200","article-title":"Intracellular signaling by the unfolded protein response","volume":"22","author":"Bernales","year":"2006","journal-title":"Annual Review of Cell and Developmental Biology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib19","doi-asserted-by":"crossref","first-page":"11709","DOI":"10.1074\/jbc.M111958200","article-title":"A principal role for the proteasome in endoplasmic reticulum-associated degradation of misfolded intracellular cystic fibrosis transmembrane conductance regulator","volume":"277","author":"Gelman","year":"2002","journal-title":"Journal of Biological Chemistry"},{"key":"10.1016\/j.bbrc.2007.06.048_bib20","doi-asserted-by":"crossref","first-page":"905","DOI":"10.1038\/nature02265","article-title":"Therapeutic approaches to protein-misfolding diseases","volume":"426","author":"Cohen","year":"2003","journal-title":"Nature"},{"key":"10.1016\/j.bbrc.2007.06.048_bib21","doi-asserted-by":"crossref","first-page":"815","DOI":"10.1111\/j.1600-0854.2004.00231.x","article-title":"A cure for traffic jams: small molecule chaperones in the endoplasmic reticulum","volume":"5","author":"Romisch","year":"2004","journal-title":"Traffic"},{"key":"10.1016\/j.bbrc.2007.06.048_bib22","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1038\/4801","article-title":"Accelerated transport and maturation of lysosomal alpha-galactosidase A in Fabry lymphoblasts by an enzyme inhibitor","volume":"5","author":"Fan","year":"1999","journal-title":"Nature Medicine"},{"key":"10.1016\/j.bbrc.2007.06.048_bib23","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1096\/fj.04-2375com","article-title":"A synthetic chaperone corrects the trafficking defect and disease phenotype in a protein misfolding disorder","volume":"19","author":"Yam","year":"2005","journal-title":"FASEB Journal"},{"key":"10.1016\/j.bbrc.2007.06.048_bib24","doi-asserted-by":"crossref","first-page":"1628","DOI":"10.1093\/emboj\/21.7.1628","article-title":"Ligands act as pharmacological chaperones and increase the efficiency of delta opioid receptor maturation","volume":"21","author":"Petaja-Repo","year":"2002","journal-title":"EMBO Journal"},{"key":"10.1016\/j.bbrc.2007.06.048_bib25","doi-asserted-by":"crossref","first-page":"485","DOI":"10.1038\/nm0502-485","article-title":"Calcium-pump inhibitors induce functional surface expression of Delta F508-CFTR protein in cystic fibrosis epithelial cells","volume":"8","author":"Egan","year":"2002","journal-title":"Nature Medicine"},{"key":"10.1016\/j.bbrc.2007.06.048_bib26","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1379\/1466-1268(1996)001<0109:IOMACC>2.3.CO;2","article-title":"Influence of molecular and chemical chaperones on protein folding","volume":"1","author":"Welch","year":"1996","journal-title":"Cell Stress Chaperones"},{"key":"10.1016\/j.bbrc.2007.06.048_bib27","doi-asserted-by":"crossref","first-page":"2257","DOI":"10.1172\/JCI2303","article-title":"Defective aquaporin-2 trafficking in nephrogenic diabetes insipidus and correction by chemical chaperones","volume":"101","author":"Tamarappoo","year":"1998","journal-title":"Journal of Clinical Investigation"},{"key":"10.1016\/j.bbrc.2007.06.048_bib28","doi-asserted-by":"crossref","first-page":"2564","DOI":"10.1172\/JCI24898","article-title":"Small-molecule correctors of defective DeltaF508-CFTR cellular processing identified by high-throughput screening","volume":"115","author":"Pedemonte","year":"2005","journal-title":"Journal of Clinical Investigation"},{"key":"10.1016\/j.bbrc.2007.06.048_bib29","doi-asserted-by":"crossref","first-page":"2457","DOI":"10.1172\/JCI119788","article-title":"In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR","volume":"100","author":"Rubenstein","year":"1997","journal-title":"Journal of Clinical Investigation"},{"key":"10.1016\/j.bbrc.2007.06.048_bib30","doi-asserted-by":"crossref","first-page":"1796","DOI":"10.1073\/pnas.97.4.1796","article-title":"Chemical chaperones mediate increased secretion of mutant alpha 1-antitrypsin (alpha 1-AT) Z: a potential pharmacological strategy for prevention of liver injury and emphysema in alpha 1-AT deficiency","volume":"97","author":"Burrows","year":"2000","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"key":"10.1016\/j.bbrc.2007.06.048_bib31","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1165\/rcmb.2002-0086OC","article-title":"Modulation of deltaF508 cystic fibrosis transmembrane regulator trafficking and function with 4-phenylbutyrate and flavonoids","volume":"31","author":"Lim","year":"2004","journal-title":"American Journal of Respiratory Cell and Molecular Biology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib32","doi-asserted-by":"crossref","first-page":"1259","DOI":"10.1111\/j.1471-4159.2006.03782.x","article-title":"Suppressive effects of 4-phenylbutyrate on the aggregation of Pael receptors and endoplasmic reticulum stress","volume":"97","author":"Kubota","year":"2006","journal-title":"Journal of Neurochemistry"},{"key":"10.1016\/j.bbrc.2007.06.048_bib33","doi-asserted-by":"crossref","first-page":"1683","DOI":"10.1167\/iovs.06-0943","article-title":"Sodium 4-phenylbutyrate acts as a chemical chaperone on misfolded myocilin to rescue cells from endoplasmic reticulum stress and apoptosis","volume":"48","author":"Yam","year":"2007","journal-title":"Investigative Ophthalmology and Visual Science"},{"key":"10.1016\/j.bbrc.2007.06.048_bib34","doi-asserted-by":"crossref","first-page":"1881","DOI":"10.1111\/j.1742-4658.2007.05735.x","article-title":"4-Phenylbutyrate restores the functionality of a misfolded mutant low-density lipoprotein receptor","volume":"274","author":"Tveten","year":"2007","journal-title":"FEBS Journal"},{"key":"10.1016\/j.bbrc.2007.06.048_bib35","series-title":"The Metabolic and Molecular Bases of Inherited Disease","first-page":"3733","article-title":"\u03b1-Galactosidase A deficiency: Fabry disease","author":"Desnick","year":"2001"},{"key":"10.1016\/j.bbrc.2007.06.048_bib36","doi-asserted-by":"crossref","first-page":"C1076","DOI":"10.1152\/ajpcell.00426.2005","article-title":"Pharmacological chaperone corrects lysosomal storage in Fabry disease caused by trafficking-incompetent variants","volume":"290","author":"Yam","year":"2006","journal-title":"American Journal of Physiology and Cell Physiology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib37","doi-asserted-by":"crossref","first-page":"15428","DOI":"10.1073\/pnas.192582899","article-title":"Chemical chaperones increase the cellular activity of N370S beta-glucosidase: a therapeutic strategy for Gaucher disease","volume":"99","author":"Sawkar","year":"2002","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"key":"10.1016\/j.bbrc.2007.06.048_bib38","doi-asserted-by":"crossref","first-page":"15912","DOI":"10.1073\/pnas.2536657100","article-title":"Chemical chaperone therapy for brain pathology in GM1-gangliosidosis","volume":"100","author":"Matsuda","year":"2003","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"key":"10.1016\/j.bbrc.2007.06.048_bib39","doi-asserted-by":"crossref","first-page":"1585","DOI":"10.1006\/bbrc.1993.2659","article-title":"Characterization of a mutant alpha-galactosidase gene product for the late-onset cardiac form of Fabry disease","volume":"197","author":"Ishii","year":"1993","journal-title":"Biochemical and Biophysical Research Communications"},{"key":"10.1016\/j.bbrc.2007.06.048_bib40","doi-asserted-by":"crossref","first-page":"591","DOI":"10.1023\/A:1006915926732","article-title":"Alpha-galactosidase transgenic mouse: heterogeneous gene expression and posttranslational glycosylation in tissues","volume":"15","author":"Ishii","year":"1998","journal-title":"Glycoconjugate Journal"},{"key":"10.1016\/j.bbrc.2007.06.048_bib41","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1016\/S0065-3101(24)00072-0","article-title":"Urea cycle disorders: diagnosis, pathophysiology, and therapy","volume":"43","author":"Brusilow","year":"1996","journal-title":"Advances in Pediatrics"},{"key":"10.1016\/j.bbrc.2007.06.048_bib42","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1152\/physiolgenomics.00160.2003","article-title":"Gene expression profile analysis of 4-phenylbutyrate treatment of IB3-1 bronchial epithelial cell line demonstrates a major influence on heat-shock proteins","volume":"16","author":"Wright","year":"2004","journal-title":"Physiological Genomics"},{"key":"10.1016\/j.bbrc.2007.06.048_bib43","doi-asserted-by":"crossref","first-page":"12300","DOI":"10.1073\/pnas.0404764101","article-title":"Chemical chaperones protect from effects of apoptosis-inducing mutation in carbonic anhydrase IV identified in retinitis pigmentosa 17","volume":"101","author":"Bonapace","year":"2004","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"key":"10.1016\/j.bbrc.2007.06.048_bib44","doi-asserted-by":"crossref","first-page":"1731","DOI":"10.1097\/01.ASN.0000129826.28932.FD","article-title":"Defective trafficking of nephrin missense mutants rescued by a chemical chaperone","volume":"15","author":"Liu","year":"2004","journal-title":"Journal of the American Society of Nephrology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib45","doi-asserted-by":"crossref","first-page":"1137","DOI":"10.1126\/science.1128294","article-title":"Chemical chaperones reduce ER stress and restore glucose homeostasis in a mouse model of type 2 diabetes","volume":"313","author":"\u00d6zcan","year":"2006","journal-title":"Science"},{"key":"10.1016\/j.bbrc.2007.06.048_bib46","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1007\/3-540-29717-0_16","article-title":"Chemical chaperones: mechanisms of action and potential use","author":"Papp","year":"2006","journal-title":"Handbook of Experimental Pharmacology"},{"key":"10.1016\/j.bbrc.2007.06.048_bib47","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1146\/annurev.biochem.69.1.69","article-title":"Protein glucosylation and its role in protein folding","volume":"69","author":"Parodi","year":"2000","journal-title":"Annual Review of Biochemistry"},{"key":"10.1016\/j.bbrc.2007.06.048_bib48","doi-asserted-by":"crossref","first-page":"228","DOI":"10.1006\/abbi.2000.1743","article-title":"Role of Ser-65 in the activity of alpha-galactosidase A: characterization of a point mutation (S65T) detected in a patient with Fabry disease","volume":"377","author":"Ishii","year":"2000","journal-title":"Archives of Biochemistry and Biophysics"}],"container-title":["Biochemical and Biophysical Research Communications"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0006291X07012892?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0006291X07012892?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,2,15]],"date-time":"2024-02-15T04:05:30Z","timestamp":1707969930000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0006291X07012892"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2007,8]]},"references-count":48,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2007,8]]}},"alternative-id":["S0006291X07012892"],"URL":"https:\/\/doi.org\/10.1016\/j.bbrc.2007.06.048","relation":{},"ISSN":["0006-291X"],"issn-type":[{"value":"0006-291X","type":"print"}],"subject":[],"published":{"date-parts":[[2007,8]]}}}