{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,21]],"date-time":"2026-05-21T04:02:27Z","timestamp":1779336147097,"version":"3.51.4"},"reference-count":32,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2003,1,1]],"date-time":"2003-01-01T00:00:00Z","timestamp":1041379200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2003,1,1]],"date-time":"2003-01-01T00:00:00Z","timestamp":1041379200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Gene Ther"],"published-print":{"date-parts":[[2003,1,1]]},"DOI":"10.1038\/sj.gt.3301843","type":"journal-article","created":{"date-parts":[[2003,1,13]],"date-time":"2003-01-13T15:49:00Z","timestamp":1042472940000},"page":"34-42","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":57,"title":["Viral-mediated delivery of the late-infantile neuronal ceroid lipofuscinosis gene, TPP-I to the mouse central nervous system"],"prefix":"10.1038","volume":"10","author":[{"given":"R E","family":"Haskell","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"S M","family":"Hughes","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J A","family":"Chiorini","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J M","family":"Alisky","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"B L","family":"Davidson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2003,1,13]]},"reference":[{"key":"BF3301843_CR1","first-page":"1","volume-title":"Ceroid-lipofuscinoses. Batten Disease and Allied Disorders","author":"JM Opitz","year":"1987","unstructured":"Opitz JM . Ceroid-lipofuscinoses. Batten Disease and Allied Disorders. Alan R. Liss, Inc.: New York, 1987,pp 1\u2013307."},{"key":"BF3301843_CR2","first-page":"33","volume-title":"The Neuronal Ceroid Lipofuscinoses (Batten Disease): Biomedical and Health Research","author":"HH Goebel","year":"1999","unstructured":"Goebel HH, Mole SE, Lake BD . The Neuronal Ceroid Lipofuscinoses (Batten Disease): Biomedical and Health Research. IOS Press: Amsterdam, 1999, p 33."},{"key":"BF3301843_CR3","doi-asserted-by":"publisher","first-page":"700","DOI":"10.1046\/j.1471-4159.1999.0730700.x","volume":"73","author":"I Sohar","year":"1999","unstructured":"Sohar I, Sleat DE, Jadot M, Lobel P . Biochemical characterization of a lysosomal protease deficient in classical late infantile neuronal ceroid lipofuscinosis (LINCL) and development of an enzyme-based assay for diagnosis and exclusion of LINCL in human specimens and animal models. J Neurochem 1999; 73: 700\u2013711.","journal-title":"J Neurochem"},{"key":"BF3301843_CR4","doi-asserted-by":"publisher","first-page":"561","DOI":"10.1002\/ajmg.1320420428","volume":"42","author":"DN Palmer","year":"1992","unstructured":"Palmer DN et al. Mitochondrial ATP synthase subunit c storage in the ceroid-lipofuscinoses (Batten disease). Am J Med Genet 1992; 42: 561\u2013567.","journal-title":"Am J Med Genet"},{"key":"BF3301843_CR5","doi-asserted-by":"publisher","first-page":"1802","DOI":"10.1126\/science.277.5333.1802","volume":"277","author":"DE Sleat","year":"1997","unstructured":"Sleat DE et al. Association of mutations in a lysosomal protein with classical late-infantile neuronal ceroid lipofuscinosis. Science 1997; 277: 1802\u20131805.","journal-title":"Science"},{"key":"BF3301843_CR6","doi-asserted-by":"publisher","first-page":"233","DOI":"10.1016\/S0167-4838(98)00012-0","volume":"1384","author":"D Vines","year":"1998","unstructured":"Vines D, Warburton MJ . Purification and characterisation of a tripeptidyl aminopeptidase I from rat spleen. Biochim Biophys Acta 1998; 1384: 233\u2013242.","journal-title":"Biochim Biophys Acta"},{"key":"BF3301843_CR7","doi-asserted-by":"publisher","first-page":"496","DOI":"10.1016\/S0167-4838(98)00238-6","volume":"1429","author":"ND Rawlings","year":"1999","unstructured":"Rawlings ND, Barrett AJ . Tripeptidyl-peptidase I is apparently the CLN2 protein absent in classical late-infantile neuro-nal ceroid lipofuscinosis. Biochim Biophys Acta 1999; 1429: 496\u2013500.","journal-title":"Biochim Biophys Acta"},{"key":"BF3301843_CR8","doi-asserted-by":"publisher","first-page":"131","DOI":"10.1016\/S0014-5793(98)01683-4","volume":"443","author":"DJ Vines","year":"1999","unstructured":"Vines DJ, Warburton MJ . Classical late infantile neuronal ceroid lipofuscinosis fibroblasts are deficient in lysosomal tripeptidyl peptidase I. FEBS Lett 1999; 443: 131\u2013135.","journal-title":"FEBS Lett"},{"key":"BF3301843_CR9","doi-asserted-by":"publisher","first-page":"2249","DOI":"10.1074\/jbc.M008562200","volume":"276","author":"L Lin","year":"2001","unstructured":"Lin L, Sohart I, Lackland H, Lobel P . The human CLN2 protein\/tripeptidyl-peptidase I is a serine protease that autoactivates at acIdic pH. J Biol Chem 2001; 276: 2249\u20132255.","journal-title":"J Biol Chem"},{"key":"BF3301843_CR10","doi-asserted-by":"publisher","first-page":"509","DOI":"10.1093\/oxfordjournals.jbchem.a022781","volume":"128","author":"J Ezaki","year":"2000","unstructured":"Ezaki J, Takeda-Ezaki M, Kominami E . Tripeptidyl peptidase I, the late infantile neuronal ceroid lipofuscinosis gene product, initiates the lysosomal degradation of subunit c of ATP synthase. J Biochem 2000; 128: 509\u2013516.","journal-title":"J Biochem"},{"key":"BF3301843_CR11","doi-asserted-by":"publisher","first-page":"2573","DOI":"10.1046\/j.1471-4159.1999.0722573.x","volume":"72","author":"J Ezaki","year":"1999","unstructured":"Ezaki J, Tanida I, Kanehagi N, Kominami E . A lysosomal proteinase, the late infantile neuronal ceroid lipofuscinosis gene (CLN2) product, is essential for degradation of a hydrophobic protein, the subunit c of ATP synthase. J Neurochem 1999; 72: 2573\u20132582.","journal-title":"J Neurochem"},{"key":"BF3301843_CR12","doi-asserted-by":"publisher","first-page":"49","DOI":"10.1042\/bj3570049","volume":"357","author":"L Lin","year":"2001","unstructured":"Lin L, Lobel P . Production and characterization of recombinant human CLN2 protein for enzyme-replacement therapy in late infantile neuronal ceroid lipofus. Biochem J 2001; 357: 49\u201355.","journal-title":"Biochem J"},{"key":"BF3301843_CR13","doi-asserted-by":"publisher","first-page":"640","DOI":"10.1038\/90242","volume":"19","author":"H Xia","year":"2001","unstructured":"Xia H, Mao Q, Davidson BL . The HIV Tat protein transduction domain improves the biodistribution of \u03b2-glucuronidase expressed from recombinant viral vectors. Nat Biotechnol 2001; 19: 640\u2013644.","journal-title":"Nat Biotechnol"},{"key":"BF3301843_CR14","doi-asserted-by":"publisher","first-page":"310","DOI":"10.1038\/85454","volume":"7","author":"A Consiglio","year":"2001","unstructured":"Consiglio A et al. In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice. Nat Med 2001; 7: 310\u2013316.","journal-title":"Nat Med"},{"key":"BF3301843_CR15","doi-asserted-by":"publisher","first-page":"2331","DOI":"10.1089\/hum.1998.9.16-2331","volume":"9","author":"A Ghodsi","year":"1998","unstructured":"Ghodsi A et al. Extensive \u03b2-glucuronidase activity in murine CNS after adenovirus mediated gene transfer to brain. Hum Gene Ther 1998; 9: 2331\u20132340.","journal-title":"Hum Gene Ther"},{"key":"BF3301843_CR16","doi-asserted-by":"crossref","first-page":"3424","DOI":"10.1128\/JVI.73.4.3424-3429.1999","volume":"73","author":"CS Stein","year":"1999","unstructured":"Stein CS, Ghodsi A, Derksen T, Davidson BL . Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice. J Virol 1999; 73: 3424\u20133429.","journal-title":"J Virol"},{"key":"BF3301843_CR17","doi-asserted-by":"publisher","first-page":"575","DOI":"10.1023\/A:1005423222927","volume":"21","author":"C Vogler","year":"1998","unstructured":"Vogler C et al. Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease. J Inherit Metab Dis 1998; 21: 575\u2013586.","journal-title":"J Inherit Metab Dis"},{"key":"BF3301843_CR18","doi-asserted-by":"publisher","first-page":"1139","DOI":"10.1089\/10430340050015194","volume":"11","author":"A Bosch","year":"2000","unstructured":"Bosch A et al. Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. Hum Gene Ther 2000; 11: 1139\u20131150.","journal-title":"Hum Gene Ther"},{"key":"BF3301843_CR19","doi-asserted-by":"publisher","first-page":"6216","DOI":"10.1073\/pnas.082011999","volume":"99","author":"AI Brooks","year":"2002","unstructured":"Brooks AI et al. Functional correction of established CNS deficits in an animal model of lysosomal storage disease using feline immunodeficiency virus-based vectors. Proc Natl Acad Sci USA 2002; 99: 6216\u20136221.","journal-title":"Proc Natl Acad Sci USA"},{"key":"BF3301843_CR20","doi-asserted-by":"publisher","first-page":"2669","DOI":"10.1097\/00001756-200008210-00013","volume":"11","author":"JM Alisky","year":"2000","unstructured":"Alisky JM et al. Transduction of murine cerebellar neurons with recombinant FIV and AAV5 vectors. NeuroReport 2000; 11: 2669\u20132673.","journal-title":"NeuroReport"},{"key":"BF3301843_CR21","doi-asserted-by":"publisher","first-page":"3428","DOI":"10.1073\/pnas.97.7.3428","volume":"97","author":"BL Davidson","year":"2000","unstructured":"Davidson BL et al. Recombinant AAV type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian CNS. Proc Natl Acad Sci USA 2000; 97: 3428\u20133432.","journal-title":"Proc Natl Acad Sci USA"},{"key":"BF3301843_CR22","doi-asserted-by":"publisher","first-page":"42","DOI":"10.1006\/mthe.2001.0514","volume":"5","author":"H Fu","year":"2002","unstructured":"Fu H et al. Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery. Mol Ther 2002; 5: 42\u201349.","journal-title":"Mol Ther"},{"key":"BF3301843_CR23","doi-asserted-by":"publisher","first-page":"89","DOI":"10.1023\/A:1012440428282","volume":"24","author":"JA Barranger","year":"2001","unstructured":"Barranger JA, O'Rourke E . Lessons learned from the development of enzyme therapy for Gaucher disease. J Inherit Metab Dis 2001; 24: 89\u201396.","journal-title":"J Inherit Metab Dis"},{"key":"BF3301843_CR24","doi-asserted-by":"publisher","first-page":"1","DOI":"10.5414\/CNP57001","volume":"57","author":"RJ Desnick","year":"2002","unstructured":"Desnick RJ, Banikazemi M, Wasserstein M . Enzyme replacement therapy for Fabry disease, an inherited nephropathy. Clin Nephrol 2002; 57: 1\u20138.","journal-title":"Clin Nephrol"},{"key":"BF3301843_CR25","first-page":"487","volume":"1","author":"P Leone","year":"1999","unstructured":"Leone P, Janson CG, McPhee SJ, During MJ . Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease. Curr Opin Mol Ther 1999; 1: 487\u2013492.","journal-title":"Curr Opin Mol Ther"},{"key":"BF3301843_CR26","doi-asserted-by":"publisher","first-page":"109","DOI":"10.1006\/exnr.1999.7205","volume":"160","author":"A Ghodsi","year":"1999","unstructured":"Ghodsi A et al. Systemic hyperosmolality improves \u03b2-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer. Exp Neurol 1999; 160: 109\u2013116.","journal-title":"Exp Neurol"},{"key":"BF3301843_CR27","doi-asserted-by":"publisher","first-page":"1034","DOI":"10.1038\/sj.gt.3301197","volume":"7","author":"RD Anderson","year":"2000","unstructured":"Anderson RD et al. A simple method for the rapid generation of recombinant adenovirus vectors. Gene Ther 2000; 7: 1034\u20131038.","journal-title":"Gene Ther"},{"key":"BF3301843_CR28","doi-asserted-by":"crossref","first-page":"4805","DOI":"10.1128\/jvi.70.7.4805-4810.1996","volume":"70","author":"C Chartier","year":"1996","unstructured":"Chartier C et al. Efficient generation of recombinant adenovirus vectors by homologous recombination in Escherichia coli. J Virol 1996; 70: 4805\u20134810.","journal-title":"J Virol"},{"key":"BF3301843_CR29","doi-asserted-by":"crossref","first-page":"4991","DOI":"10.1128\/JVI.73.6.4991-5000.1999","volume":"73","author":"JC Johnston","year":"1999","unstructured":"Johnston JC et al. Minimum requirements for efficient transduction of dividing and nondividing cells by feline immunodeficiency virus vectors. J Virol 1999; 73: 4991\u20135000.","journal-title":"J Virol"},{"key":"BF3301843_CR30","doi-asserted-by":"publisher","first-page":"1531","DOI":"10.1089\/hum.1995.6.12-1531","volume":"6","author":"JA Chiorini","year":"1995","unstructured":"Chiorini JA et al. High-efficiency transfer of the T cell co-stimulatory molecule B7-2 to lymphoid cells using high-titer recombinant adeno-associated virus vectors. Hum Gene Ther 1995; 6: 1531\u20131541.","journal-title":"Hum Gene Ther"},{"key":"BF3301843_CR31","unstructured":"Davidson BL, Chiorini JA . Recombinant adeno-associated virus vector types 4 and 5: preparation and application for CNS gene transfer. Viral Vectors Gene Ther 2003 (in press)."},{"key":"BF3301843_CR32","doi-asserted-by":"publisher","first-page":"354","DOI":"10.1006\/abbi.1993.1523","volume":"306","author":"AE Page","year":"1993","unstructured":"Page AE, Fuller K, Chambers TJ, Warburton MJ . Purification and characterization of a tripeptidyl peptidase I from human osteoclastomas: evidence for its role in bone resorption. Arch Biochem Biophys 1993; 306: 354\u2013359.","journal-title":"Arch Biochem Biophys"}],"container-title":["Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.nature.com\/articles\/3301843.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/3301843","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/3301843.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,3,30]],"date-time":"2022-03-30T02:05:56Z","timestamp":1648605956000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/3301843"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2003,1,1]]},"references-count":32,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2003,1,1]]}},"alternative-id":["BF3301843"],"URL":"https:\/\/doi.org\/10.1038\/sj.gt.3301843","relation":{},"ISSN":["0969-7128","1476-5462"],"issn-type":[{"value":"0969-7128","type":"print"},{"value":"1476-5462","type":"electronic"}],"subject":[],"published":{"date-parts":[[2003,1,1]]},"assertion":[{"value":"20 March 2002","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"2 June 2002","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"13 January 2003","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}}]}}