{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,7]],"date-time":"2026-02-07T04:06:17Z","timestamp":1770437177614,"version":"3.49.0"},"reference-count":78,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2012,6,1]],"date-time":"2012-06-01T00:00:00Z","timestamp":1338508800000},"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":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2012,6]]},"DOI":"10.1016\/j.ymgme.2012.03.022","type":"journal-article","created":{"date-parts":[[2012,4,5]],"date-time":"2012-04-05T22:25:19Z","timestamp":1333664719000},"page":"202-213","source":"Crossref","is-referenced-by-count":30,"title":["Skeletal response to lentiviral mediated gene therapy in a mouse model of MPS VII"],"prefix":"10.1016","volume":"106","author":[{"given":"Carmen E.","family":"Macsai","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ainslie L.K.","family":"Derrick-Roberts","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xiaodan","family":"Ding","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Krystyna H.","family":"Zarrinkalam","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chantelle","family":"McIntyre","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paul H.","family":"Anderson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Don S.","family":"Anson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sharon","family":"Byers","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymgme.2012.03.022_bb0005","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1001\/jama.281.3.249","article-title":"Prevalence of lysosomal storage disorders","volume":"281","author":"Meikle","year":"1999","journal-title":"JAMA"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0010","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1002\/humu.20828","article-title":"Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII","volume":"30","author":"Tomatsu","year":"2009","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0015","series-title":"The Metabolic and Molecular Basis of Inherited Disease","first-page":"3421","article-title":"The mucopolysaccharidoses","author":"Neufeld","year":"2001"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0020","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1006\/geno.1999.5825","article-title":"Molecular basis of feline beta-glucuronidase deficiency: an animal model of mucopolysaccharidosis VII","volume":"58","author":"Fyfe","year":"1999","journal-title":"Genomics"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0025","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1177\/030098589403100405","article-title":"Feline mucopolysaccharidosis VII due to beta-glucuronidase deficiency","volume":"31","author":"Gitzelmann","year":"1994","journal-title":"Vet. Pathol."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0030","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1016\/j.ymgme.2008.07.003","article-title":"Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy","volume":"95","author":"Herati","year":"2008","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0035","doi-asserted-by":"crossref","first-page":"112","DOI":"10.2353\/ajpath.2008.070564","article-title":"Mechanism of glycosaminoglycan-mediated bone and joint disease: implications for the mucopolysaccharidoses and other connective tissue diseases","volume":"172","author":"Simonaro","year":"2008","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0040","doi-asserted-by":"crossref","first-page":"616","DOI":"10.1002\/jor.21030","article-title":"Altered lumbar spine structure, biochemistry, and biomechanical properties in a canine model of mucopolysaccharidosis type VII","volume":"28","author":"Smith","year":"2009","journal-title":"J. Orthop. Res."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0045","doi-asserted-by":"crossref","first-page":"1258","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":"10.1016\/j.ymgme.2012.03.022_bb0050","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1007\/s10545-007-0483-4","article-title":"Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII","volume":"30","author":"Donsante","year":"2007","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0055","doi-asserted-by":"crossref","first-page":"202","DOI":"10.1016\/j.ymgme.2009.03.005","article-title":"Mechanism of shortened bones in mucopolysaccharidosis VII","volume":"97","author":"Metcalf","year":"2009","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0060","doi-asserted-by":"crossref","first-page":"453","DOI":"10.1007\/s10024-005-0025-8","article-title":"Early onset of lysosomal storage disease in a murine model of mucopolysaccharidosis type VII: undegraded substrate accumulates in many tissues in the fetus and very young MPS VII mouse","volume":"8","author":"Vogler","year":"2005","journal-title":"Pediatr. Dev. Pathol."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0065","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. U. S. A."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0070","doi-asserted-by":"crossref","first-page":"2324","DOI":"10.1172\/JCI117237","article-title":"Enzyme replacement therapy for murine mucopolysaccharidosis type VII","volume":"93","author":"Sands","year":"1994","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0075","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":"10.1016\/j.ymgme.2012.03.022_bb0080","doi-asserted-by":"crossref","first-page":"1050","DOI":"10.1203\/00006450-199606000-00019","article-title":"Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival","volume":"39","author":"Vogler","year":"1996","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0085","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1006\/mthe.2002.0594","article-title":"Enhanced secretion and uptake of beta-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice","volume":"5","author":"Elliger","year":"2002","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0090","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":"10.1016\/j.ymgme.2012.03.022_bb0095","doi-asserted-by":"crossref","first-page":"406","DOI":"10.1038\/sj.gt.3301869","article-title":"Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII","volume":"10","author":"Kamata","year":"2003","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0100","doi-asserted-by":"crossref","first-page":"718","DOI":"10.1016\/j.ymthe.2003.07.004","article-title":"Improvement of skeletal lesions in mice with mucopolysaccharidosis type VII by neonatal adenoviral gene transfer","volume":"8","author":"Kanaji","year":"2003","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0105","doi-asserted-by":"crossref","first-page":"687","DOI":"10.1177\/096368970000900515","article-title":"Phenotype correction in murine mucopolysaccharidosis type VII by transplantation of human amniotic epithelial cells after adenovirus-mediated gene transfer","volume":"9","author":"Kosuga","year":"2000","journal-title":"Cell Transplant."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0110","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. U. S. A."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0115","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1006\/mthe.2000.0121","article-title":"Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice","volume":"2","author":"Gao","year":"2000","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0120","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1016\/j.ymgme.2004.01.015","article-title":"Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs","volume":"82","author":"Mango","year":"2004","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0125","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":"10.1016\/j.ymgme.2012.03.022_bb0130","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":"10.1016\/j.ymgme.2012.03.022_bb0135","doi-asserted-by":"crossref","first-page":"1291","DOI":"10.1038\/sj.gt.3301420","article-title":"Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer","volume":"8","author":"Daly","year":"2001","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0140","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":"10.1016\/j.ymgme.2012.03.022_bb0145","doi-asserted-by":"crossref","first-page":"242","DOI":"10.1038\/sj.mt.6300016","article-title":"Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS","volume":"15","author":"Liu","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0150","doi-asserted-by":"crossref","first-page":"507","DOI":"10.1089\/10430340050015707","article-title":"Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse","volume":"11","author":"Sferra","year":"2000","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0155","doi-asserted-by":"crossref","first-page":"370","DOI":"10.1016\/j.ymgme.2010.08.013","article-title":"Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII","volume":"101","author":"Bielicki","year":"2010","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0160","doi-asserted-by":"crossref","first-page":"1139","DOI":"10.1089\/10430340050015194","article-title":"Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer","volume":"11","author":"Bosch","year":"2000","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0165","doi-asserted-by":"crossref","first-page":"776","DOI":"10.1016\/j.ymthe.2004.10.006","article-title":"Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector","volume":"11","author":"Kobayashi","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0170","doi-asserted-by":"crossref","first-page":"850","DOI":"10.1006\/mthe.2001.0325","article-title":"In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors","volume":"3","author":"Stein","year":"2001","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0175","doi-asserted-by":"crossref","first-page":"10319","DOI":"10.1073\/pnas.94.19.10319","article-title":"Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector","volume":"94","author":"Miyoshi","year":"1997","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0180","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1126\/science.272.5259.263","article-title":"In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector","volume":"272","author":"Naldini","year":"1996","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0185","first-page":"383","article-title":"Lentiviral vectors for gene transfer to the central nervous system. Applications in lysosomal storage disease animal models","volume":"76","author":"Watson","year":"2003","journal-title":"Methods Mol. Med."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0190","doi-asserted-by":"crossref","first-page":"14982","DOI":"10.1073\/pnas.232570999","article-title":"Missense models [Gustm(E536A) Sly, Gustm (E536Q) Sly, and Gustm(L175F) Sly] of murine mucopolysaccharidosis type VII produced by targeted mutagenesis","volume":"99","author":"Tomatsu","year":"2002","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0195","series-title":"Protocols for Gene Transfer in Neuroscience","first-page":"263","article-title":"Towards gene therapy of neurologic disorders","author":"Wolfe","year":"1996"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0200","doi-asserted-by":"crossref","first-page":"538","DOI":"10.1203\/01.pdr.0000203090.41012.a6","article-title":"Intra-articular enzyme administration for joint disease in feline mucopolysaccharidosis VI: enzyme dose and interval","volume":"59","author":"Auclair","year":"2006","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0205","doi-asserted-by":"crossref","first-page":"1390","DOI":"10.1002\/jgm.803","article-title":"Optimisation of a multipartite human immunodeficiency virus based vector system; control of virus infectivity and large-scale production","volume":"7","author":"Koldej","year":"2005","journal-title":"J. Gene Med."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0210","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/1479-0556-5-1","article-title":"Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA","volume":"5","author":"Anson","year":"2007","journal-title":"Genet. Vaccines Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0215","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1042\/bj0780151","article-title":"Studies on glucosaminidase. 4. The fluorimetric assay of N-acetyl-beta-glucosaminidase","volume":"78","author":"Leaback","year":"1961","journal-title":"Biochem. J."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0220","series-title":"Protocols for Gene Transfer in Neuroscience: Towards Gene Therapy of Neurological Disorders","author":"Lowenstein","year":"1996"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0225","doi-asserted-by":"crossref","first-page":"309","DOI":"10.1203\/01.pdr.0000233037.00707.da","article-title":"Inhibition of glycosaminoglycan synthesis using rhodamine B in a mouse model of mucopolysaccharidosis type IIIA","volume":"60","author":"Roberts","year":"2006","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0230","series-title":"Mouse Behavioural Testing: How to Use Mice in Behavioural Neuroscience","author":"Wahlsten","year":"2011"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0235","doi-asserted-by":"crossref","first-page":"e772","DOI":"10.1371\/journal.pone.0000772","article-title":"Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo syndrome type B","volume":"2","author":"Heldermon","year":"2007","journal-title":"PLoS One"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0240","doi-asserted-by":"crossref","first-page":"302","DOI":"10.1111\/j.2042-7158.1968.tb09743.x","article-title":"Quantitative measurement of motor incoordination in na\u00efve mice using an accelerated rotarod","volume":"20","author":"Jones BJ","year":"1968","journal-title":"J. Pharm. Pharmacol."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0245","doi-asserted-by":"crossref","first-page":"115","DOI":"10.1016\/j.ymgme.2007.06.016","article-title":"Improvement in behaviour after substrate deprivation therapy with rhodamine B in a mouse model of MPS IIIA","volume":"92","author":"Roberts","year":"2007","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0250","doi-asserted-by":"crossref","first-page":"491","DOI":"10.1038\/sj.bmt.1703395","article-title":"Outcome of second hematopoietic cell transplantation in Hurler syndrome","volume":"29","author":"Grewal","year":"2002","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0255","doi-asserted-by":"crossref","first-page":"533","DOI":"10.1016\/j.jpeds.2005.12.014","volume":"148","author":"Harmatz","year":"2006","journal-title":"J. Pediatr."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0260","first-page":"359","article-title":"Stem cell bone marrow transplantation in patients with metabolic storage diseases","volume":"49","author":"Krivit","year":"2002","journal-title":"Adv. Pediatr."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0265","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1016\/j.ymgme.2006.09.001","article-title":"A phase I\/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II","volume":"90","author":"Muenzer","year":"2007","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0270","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1097\/01.gim.0000232477.37660.fb","article-title":"A phase II\/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II","volume":"8","author":"Muenzer","year":"2006","journal-title":"Genet Med."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0275","doi-asserted-by":"crossref","first-page":"1005","DOI":"10.1038\/bmt.2008.20","article-title":"Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation","volume":"41","author":"Polgreen","year":"2008","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0280","doi-asserted-by":"crossref","first-page":"1960","DOI":"10.1056\/NEJMoa032613","article-title":"Cord-blood transplants from unrelated donors in patients with Hurler's syndrome","volume":"350","author":"Staba","year":"2004","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0285","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1136\/adc.76.2.92","article-title":"Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres","volume":"76","author":"Vellodi","year":"1997","journal-title":"Arch. Dis. Child."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0290","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1016\/S1096-7192(03)00007-6","article-title":"Replacement therapy in Mucopolysaccharidosis type VI: advantages of early onset of therapy","volume":"78","author":"Auclair","year":"2003","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0295","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1016\/S8756-3282(97)00175-0","article-title":"Effect of enzyme replacement therapy on bone formation in a feline model of mucopolysaccharidosis type VI","volume":"21","author":"Byers","year":"1997","journal-title":"Bone"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0300","doi-asserted-by":"crossref","first-page":"651","DOI":"10.1172\/JCI119208","article-title":"Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI","volume":"99","author":"Crawley","year":"1997","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0305","doi-asserted-by":"crossref","first-page":"821","DOI":"10.1007\/s10545-007-0551-9","article-title":"Enzyme replacement therapy in 12 patients with MPS I-H\/S with homozygous p. Leu490Pro mutation","volume":"30","author":"Arora","year":"2007","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0310","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1016\/j.ymgme.2006.08.007","article-title":"A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years","volume":"90","author":"Sifuentes","year":"2007","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0315","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1007\/s10545-010-9059-9","article-title":"Efficacy of recombinant human alpha-l-iduronidase (laronidase) on restricted range of motion of upper extremities in mucopolysaccharidosis type I patients","volume":"33","author":"Tylki-Szymanska","year":"2010","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0320","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1016\/j.ymgme.2009.08.008","article-title":"Anthropometric data of 14 patients with mucopolysaccharidosis I: retrospective analysis and efficacy of recombinant human alpha-l-iduronidase (laronidase)","volume":"99","author":"Tylki-Szymanska","year":"2010","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0325","doi-asserted-by":"crossref","first-page":"208","DOI":"10.1016\/j.ymgme.2006.01.010","article-title":"Lysosomal storage diseases: natural history and ethical and economic aspects","volume":"88","author":"Beutler","year":"2006","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0330","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1089\/hum.2005.16.81","article-title":"Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector","volume":"16","author":"Di Domenico","year":"2005","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0335","doi-asserted-by":"crossref","first-page":"717","DOI":"10.1002\/jgm.1489","article-title":"Correction of mucopolysaccharidosis type IIIA somatic and central nervous system pathology by lentiviral-mediated gene transfer","volume":"12","author":"McIntyre","year":"2010","journal-title":"J. Gene Med."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0340","doi-asserted-by":"crossref","first-page":"411","DOI":"10.1016\/j.ymgme.2007.11.008","article-title":"Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA","volume":"93","author":"McIntyre","year":"2008","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0345","doi-asserted-by":"crossref","first-page":"908","DOI":"10.1007\/BF01800472","article-title":"Low beta-glucuronidase activity in a healthy member of a family with mucopolysaccharidosis VII","volume":"14","author":"Chabas","year":"1991","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0350","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1007\/s004390050656","article-title":"Low beta-glucuronidase enzyme activity and mutations in the human beta-glucuronidase gene in mild mucopolysaccharidosis type VII, pseudodeficiency and a heterozygote","volume":"102","author":"Vervoort","year":"1998","journal-title":"Hum. Genet."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0355","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1016\/S8756-3282(01)00679-2","article-title":"Abnormal osteoclast morphology and bone remodeling in a murine model of a lysosomal storage disease","volume":"30","author":"Monroy","year":"2002","journal-title":"Bone"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0360","doi-asserted-by":"crossref","first-page":"495","DOI":"10.1016\/S8756-3282(00)00357-4","article-title":"Quantitative histomorphometric analysis of the human growth plate from birth to adolescence","volume":"27","author":"Byers","year":"2000","journal-title":"Bone"},{"key":"10.1016\/j.ymgme.2012.03.022_bb0365","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":"10.1016\/j.ymgme.2012.03.022_bb0370","first-page":"681","article-title":"The blood supply of the growth cartilage in young rats","volume":"98","author":"Irving","year":"1964","journal-title":"J. Anat. Lond."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0375","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1302\/0301-620X.42B1.97","article-title":"The vascular contribution to osteogenesis. I. Studies by the injection method","volume":"42-B","author":"Trueta","year":"1960","journal-title":"J. Bone Joint Surg. Br."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0380","doi-asserted-by":"crossref","first-page":"312","DOI":"10.1007\/s00223-001-2006-x","article-title":"The blood supply of the growth plate and the epiphysis: a comparative scanning electron microscopy and histological experimental study in growing sheep","volume":"70","author":"Wirth","year":"2002","journal-title":"Calcif. Tissue Int."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0385","doi-asserted-by":"crossref","first-page":"555","DOI":"10.1089\/hum.2009.189","article-title":"Different serum enzyme levels are required to rescue the various systemic features of the mucopolysaccharidoses","volume":"21","author":"Cotugno","year":"2010","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2012.03.022_bb0390","doi-asserted-by":"crossref","first-page":"12658","DOI":"10.1073\/pnas.0405042101","article-title":"Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood\u2013brain barrier","volume":"101","author":"Urayama","year":"2004","journal-title":"Proc. Natl. Acad. Sci. U. S. A."}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719212001230?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719212001230?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2020,7,5]],"date-time":"2020-07-05T14:13:21Z","timestamp":1593958401000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719212001230"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2012,6]]},"references-count":78,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2012,6]]}},"alternative-id":["S1096719212001230"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2012.03.022","relation":{},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2012,6]]}}}