{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,12]],"date-time":"2025-10-12T19:06:01Z","timestamp":1760295961661},"reference-count":55,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2000,12,1]],"date-time":"2000-12-01T00:00:00Z","timestamp":975628800000},"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":["Experimental Neurology"],"published-print":{"date-parts":[[2000,12]]},"DOI":"10.1006\/exnr.2000.7531","type":"journal-article","created":{"date-parts":[[2002,9,16]],"date-time":"2002-09-16T15:46:56Z","timestamp":1032191216000},"page":"276-286","source":"Crossref","is-referenced-by-count":50,"title":["Somatic Gene Therapy for a Neurodegenerative Disease Using Microencapsulated Recombinant Cells"],"prefix":"10.1006","volume":"166","author":[{"given":"C.J.D.","family":"Ross","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M.","family":"Ralph","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"P.L.","family":"Chang","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1006\/exnr.2000.7531_RF1","series-title":"The Metabolic Basis of Inherited Disease","first-page":"1565","article-title":"The metabolic basis of inherited disease","author":"Neufeld","year":"1989"},{"key":"10.1006\/exnr.2000.7531_RF2","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1177\/096368979900800413","article-title":"Evaluation of reaction of primate brain to grafted PC12 cells","volume":"8","author":"Yoshida","year":"1999","journal-title":"Cell Transplant."},{"key":"10.1006\/exnr.2000.7531_RF3","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1006\/exnr.1997.6579","article-title":"Long-term enhanced chromaffin cell survival and behavioral recovery in hemiparkinsonian rats with co-grafted polymer-encapsulated human NGF-secreting cells","volume":"147","author":"Date","year":"1997","journal-title":"Exp. Neurol."},{"key":"10.1006\/exnr.2000.7531_RF4","doi-asserted-by":"crossref","first-page":"997","DOI":"10.1089\/hum.1998.9.7-997","article-title":"Dose-dependent doxycycline-mediated adrenocorticotropic hormone secretion from encapsulated Tet-on proopiomelanocortin Neuro2A cells in the subarachnoid space","volume":"9","author":"Saitoh","year":"1998","journal-title":"Hum. Gene Ther."},{"key":"10.1006\/exnr.2000.7531_RF5","doi-asserted-by":"crossref","first-page":"395","DOI":"10.1038\/386395a0","article-title":"Protective effect of encapsulated cells producing neurotrophic factor CNTF in a monkey model of Huntington's disease","volume":"386","author":"Emerich","year":"1997","journal-title":"Nature"},{"key":"10.1006\/exnr.2000.7531_RF6","doi-asserted-by":"crossref","first-page":"696","DOI":"10.1038\/nm0696-696","article-title":"Intrathecal delivery of CNTF using encapsulated genetically modified xenogeneic cells in amyotrophic lateral sclerosis patients","volume":"2","author":"Aebischer","year":"1996","journal-title":"Nat. Med."},{"key":"10.1006\/exnr.2000.7531_RF7","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1089\/10430349950019183","article-title":"Delivery of recombinant gene products to the central nervous system with nonautologous cells in alginate microcapsules","volume":"10","author":"Ross","year":"1999","journal-title":"Hum. Gene Ther."},{"key":"10.1006\/exnr.2000.7531_RF8","series-title":"Somatic Gene Therapy","first-page":"203","article-title":"Nonautologous somatic gene therapy","author":"Chang","year":"1995"},{"key":"10.1006\/exnr.2000.7531_RF9","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1016\/0925-4439(94)00075-2","article-title":"Lysosomal storage diseases","volume":"1270","author":"Gieselmann","year":"1995","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1006\/exnr.2000.7531_RF10","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1016\/S0022-3476(73)80162-3","article-title":"Beta glucuronidase deficiency: Report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis","volume":"82","author":"Sly","year":"1973","journal-title":"J. Pediatr."},{"key":"10.1006\/exnr.2000.7531_RF11","doi-asserted-by":"crossref","first-page":"507","DOI":"10.1097\/00001756-199305000-00011","article-title":"Behavioral abnormalities in a murine model of a human lysosomal storage disease","volume":"4","author":"Chang","year":"1993","journal-title":"NeuroReport"},{"key":"10.1006\/exnr.2000.7531_RF12","doi-asserted-by":"crossref","first-page":"1180","DOI":"10.1172\/JCI117434","article-title":"Behavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII","volume":"94","author":"Bastedo","year":"1994","journal-title":"J. Clin. Invest."},{"key":"10.1006\/exnr.2000.7531_RF13","doi-asserted-by":"crossref","first-page":"2033","DOI":"10.1182\/blood.V86.5.2033.bloodjournal8652033","article-title":"Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII","volume":"86","author":"Sands","year":"1995","journal-title":"Blood"},{"key":"10.1006\/exnr.2000.7531_RF14","doi-asserted-by":"crossref","first-page":"1256","DOI":"10.1172\/JCI114010","article-title":"Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency","volume":"83","author":"Birkenmeier","year":"1989","journal-title":"J. Clin. Invest."},{"key":"10.1006\/exnr.2000.7531_RF15","first-page":"207","article-title":"A murine model of mucopolysaccharidosis VII. Gross and microscopic findings in beta-glucuronidase-deficient mice","volume":"136","author":"Vogler","year":"1990","journal-title":"Am. J. Pathol."},{"key":"10.1006\/exnr.2000.7531_RF16","doi-asserted-by":"crossref","first-page":"6567","DOI":"10.1073\/pnas.90.14.6567","article-title":"A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII","volume":"90","author":"Sands","year":"1993","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1006\/exnr.2000.7531_RF17","doi-asserted-by":"crossref","first-page":"562","DOI":"10.1007\/s004010050562","article-title":"Neuropathology of murine mucopolysaccharidosis type VII","volume":"92","author":"Levy","year":"1996","journal-title":"Acta Neuropathol. (Berlin)"},{"key":"10.1006\/exnr.2000.7531_RF18","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1097\/00007890-199308000-00034","article-title":"Continuous systemic secretion of a lysosomal enzyme by genetically modified mouse skin fibroblasts","volume":"56","author":"Moullier","year":"1993","journal-title":"Transplantation"},{"key":"10.1006\/exnr.2000.7531_RF19","doi-asserted-by":"crossref","first-page":"1596","DOI":"10.1172\/JCI119322","article-title":"Murine mucopolysaccharidosis type VII: Long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation","volume":"99","author":"Sands","year":"1997","journal-title":"J. Clin. Invest."},{"key":"10.1006\/exnr.2000.7531_RF20","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1097\/00019052-199604000-00013","article-title":"Central nervous system cell transplantation: A novel therapy for storage diseases?","volume":"9","author":"Snyder","year":"1996","journal-title":"Curr. Opin. Neurol."},{"key":"10.1006\/exnr.2000.7531_RF21","doi-asserted-by":"crossref","unstructured":"Ross, C. J. D, L. Bastedo, S. H. Meier, M. Sands, G. Hortelano, and, P. L. Chang, 2000, Somatic gene therapy of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells. Submitted for publication.","DOI":"10.1089\/104303400750001426"},{"key":"10.1006\/exnr.2000.7531_RF22","first-page":"676","article-title":"Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates","volume":"68","author":"Sands","year":"1993","journal-title":"Lab. Invest."},{"key":"10.1006\/exnr.2000.7531_RF23","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1203\/00006450-199312000-00028","article-title":"Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse","volume":"34","author":"Vogler","year":"1993","journal-title":"Pediatr. Res."},{"key":"10.1006\/exnr.2000.7531_RF24","doi-asserted-by":"crossref","first-page":"1394","DOI":"10.1172\/JCI1773","article-title":"Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function","volume":"101","author":"O'Connor","year":"1998","journal-title":"J. Clin. Invest."},{"key":"10.1006\/exnr.2000.7531_RF25","doi-asserted-by":"crossref","first-page":"838","DOI":"10.1203\/00006450-199906000-00010","article-title":"Enzyme replacement in murine mucopolysaccharidosis type VII: Neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy","volume":"45","author":"Vogler","year":"1999","journal-title":"Pediatr. Res."},{"key":"10.1006\/exnr.2000.7531_RF26","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1006\/exnr.1999.7205","article-title":"Systemic hyperosmolality improves beta-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer","volume":"160","author":"Ghodsi","year":"1999","journal-title":"Exp. Neurol."},{"key":"10.1006\/exnr.2000.7531_RF27","doi-asserted-by":"crossref","first-page":"367","DOI":"10.1038\/374367a0","article-title":"Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain","volume":"374","author":"Snyder","year":"1995","journal-title":"Nature"},{"key":"10.1006\/exnr.2000.7531_RF28","doi-asserted-by":"crossref","first-page":"771","DOI":"10.1038\/nm0797-771","article-title":"Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase","volume":"3","author":"Taylor","year":"1997","journal-title":"Nat. Med."},{"key":"10.1006\/exnr.2000.7531_RF29","doi-asserted-by":"crossref","first-page":"379","DOI":"10.1038\/ng0892-379","article-title":"Herpesvirus vector gene transfer and expression of beta-glucuronidase in the central nervous system of MPS VII mice","volume":"1","author":"Wolfe","year":"1992","journal-title":"Nat. Genet."},{"key":"10.1006\/exnr.2000.7531_RF30","doi-asserted-by":"crossref","first-page":"417","DOI":"10.3109\/13550289609146908","article-title":"Increased susceptibility to the pathogenic effects of wild-type and recombinant herpesviruses in MPS VII mice compared to normal siblings","volume":"2","author":"Wolfe","year":"1996","journal-title":"J. Neurovirol."},{"key":"10.1006\/exnr.2000.7531_RF31","doi-asserted-by":"crossref","first-page":"1287","DOI":"10.1073\/pnas.94.4.1287","article-title":"Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice","volume":"94","author":"Ohashi","year":"1997","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1006\/exnr.2000.7531_RF32","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1006\/exnr.1999.7176","article-title":"Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice","volume":"160","author":"Skorupa","year":"1999","journal-title":"Exp. Neurol."},{"key":"10.1006\/exnr.2000.7531_RF33","doi-asserted-by":"crossref","first-page":"606","DOI":"10.1006\/excr.1994.1298","article-title":"Cross-correction of beta-glucuronidase deficiency by retroviral vector-mediated gene transfer","volume":"214","author":"Taylor","year":"1994","journal-title":"Exp. Cell Res."},{"key":"10.1006\/exnr.2000.7531_RF34","first-page":"A1255","article-title":"Large scale purification of phosphorylated recombinant human beta-glucuronidase from overexpressing mouse L cells","volume":"7","author":"Grubb","year":"1993","journal-title":"FASEB J."},{"key":"10.1006\/exnr.2000.7531_RF35","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1089\/hum.1993.4.3-291","article-title":"Expression of human factor IX by microencapsulated recombinant fibroblasts","volume":"4","author":"Liu","year":"1993","journal-title":"Hum. Gene Ther."},{"key":"10.1006\/exnr.2000.7531_RF36","doi-asserted-by":"crossref","first-page":"975","DOI":"10.1126\/science.2305266","article-title":"Transplanted suprachiasmatic nucleus determines circadian period","volume":"247","author":"Ralph","year":"1990","journal-title":"Science"},{"key":"10.1006\/exnr.2000.7531_RF37","first-page":"969","article-title":"Beta-glucuronidase deficiency mucopolysaccharidosis: Methods for enzymatic diagnosis","volume":"82","author":"Glaser","year":"1973","journal-title":"J. Lab. Clin. Med."},{"key":"10.1006\/exnr.2000.7531_RF38","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1016\/S0021-9258(19)52451-6","article-title":"Protein measurement with the folin phenol reagent","volume":"293","author":"Lowry","year":"1951","journal-title":"J. Biol. Chem."},{"key":"10.1006\/exnr.2000.7531_RF39","doi-asserted-by":"crossref","first-page":"271","DOI":"10.1016\/0165-3806(87)90107-6","article-title":"Interendothelial junctional changes underlie the developmental \u2018tightening\u2019 of the blood\u2013brain barrier","volume":"429","author":"Stewart","year":"1987","journal-title":"Brain Res."},{"key":"10.1006\/exnr.2000.7531_RF40","doi-asserted-by":"crossref","first-page":"2331","DOI":"10.1089\/hum.1998.9.16-2331","article-title":"Extensive beta-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain","volume":"9","author":"Ghodsi","year":"1998","journal-title":"Hum. Gene Ther."},{"key":"10.1006\/exnr.2000.7531_RF41","doi-asserted-by":"crossref","first-page":"5437","DOI":"10.1016\/S0021-9258(17)30391-5","article-title":"Turnover of murine beta-glucuronidase. Comparison among liver, kidney, and spleen and between lysosomes and microsomes","volume":"253","author":"Smith","year":"1978","journal-title":"J. Biol. Chem."},{"key":"10.1006\/exnr.2000.7531_RF42","doi-asserted-by":"crossref","first-page":"1642","DOI":"10.1038\/sj.gt.3300775","article-title":"Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus","volume":"5","author":"Watson","year":"1998","journal-title":"Gene Ther."},{"key":"10.1006\/exnr.2000.7531_RF43","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1089\/10430349950019219","article-title":"Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice","volume":"10","author":"Daly","year":"1999","journal-title":"Hum. Gene Ther."},{"key":"10.1006\/exnr.2000.7531_RF44","doi-asserted-by":"crossref","first-page":"2296","DOI":"10.1073\/pnas.96.5.2296","article-title":"Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease","volume":"96","author":"Daly","year":"1999","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1006\/exnr.2000.7531_RF45","doi-asserted-by":"crossref","first-page":"1175","DOI":"10.1038\/sj.gt.3300931","article-title":"Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector","volume":"6","author":"Elliger","year":"1999","journal-title":"Gene Ther."},{"key":"10.1006\/exnr.2000.7531_RF46","doi-asserted-by":"crossref","first-page":"3424","DOI":"10.1128\/JVI.73.4.3424-3429.1999","article-title":"Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice","volume":"73","author":"Stein","year":"1999","journal-title":"J. Virol."},{"key":"10.1006\/exnr.2000.7531_RF47","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1038\/4698","article-title":"AAVant-garde gene therapy","volume":"5","author":"Linden","year":"1999","journal-title":"Nat. Med."},{"key":"10.1006\/exnr.2000.7531_RF48","doi-asserted-by":"crossref","first-page":"431","DOI":"10.1177\/096368979900800414","article-title":"Long-term functional assessment of encapsulated cells transfected with Tet-On system","volume":"8","author":"Hagihara","year":"1999","journal-title":"Cell Transplant."},{"key":"10.1006\/exnr.2000.7531_RF49","doi-asserted-by":"crossref","first-page":"826","DOI":"10.1126\/science.287.5454.826","article-title":"Regulation of protein secretion through controlled aggregation in the endoplasmic reticulum","volume":"287","author":"Rivera","year":"2000","journal-title":"Science"},{"key":"10.1006\/exnr.2000.7531_RF50","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1006\/exnr.1993.1111","article-title":"Transplantation of a polymer-encapsulated cell line genetically engineered to release NGF","volume":"122","author":"Hoffman","year":"1993","journal-title":"Exp. Neurol."},{"key":"10.1006\/exnr.2000.7531_RF51","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1016\/0963-6897(94)00047-N","article-title":"Polymer-encapsulated Schwannoma cells expressing human nerve growth factor promote the survival of cholinergic neurons after a fimbria\u2013fornix transection","volume":"4","author":"Schinstine","year":"1995","journal-title":"Cell Transplant."},{"key":"10.1006\/exnr.2000.7531_RF52","doi-asserted-by":"crossref","first-page":"322","DOI":"10.1016\/0014-4886(91)90022-5","article-title":"Behavioral recovery following intrastriatal implantation of microencapsulated PC12 cells","volume":"113","author":"Winn","year":"1991","journal-title":"Exp. Neurol."},{"key":"10.1006\/exnr.2000.7531_RF53","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1016\/0006-8993(91)91212-J","article-title":"Transplantation of microencapsulated bovine chromaffin cells reduces lesion-induced rotational asymmetry in rats","volume":"560","author":"Aebischer","year":"1991","journal-title":"Brain Res."},{"key":"10.1006\/exnr.2000.7531_RF54","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1016\/0014-4886(91)90093-R","article-title":"Long-term cross-species brain transplantation of a polymer-encapsulated dopamine-secreting cell line","volume":"111","author":"Aebischer","year":"1991","journal-title":"Exp. Neurol."},{"key":"10.1006\/exnr.2000.7531_RF55","doi-asserted-by":"crossref","first-page":"2135","DOI":"10.1089\/hum.1996.7.17-2135","article-title":"Central nervous system delivery of recombinant ciliary neurotrophic factor by polymer encapsulated differentiated C2C12 myoblasts","volume":"7","author":"Deglon","year":"1996","journal-title":"Hum. Gene Ther."}],"container-title":["Experimental Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014488600975319?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014488600975319?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2021,5,3]],"date-time":"2021-05-03T02:30:38Z","timestamp":1620009038000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0014488600975319"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2000,12]]},"references-count":55,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2000,12]]}},"alternative-id":["S0014488600975319"],"URL":"https:\/\/doi.org\/10.1006\/exnr.2000.7531","relation":{},"ISSN":["0014-4886"],"issn-type":[{"value":"0014-4886","type":"print"}],"subject":[],"published":{"date-parts":[[2000,12]]}}}