{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T14:31:45Z","timestamp":1791210705233,"version":"4.1.0"},"reference-count":149,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2015,2,1]],"date-time":"2015-02-01T00:00:00Z","timestamp":1422748800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2014,10,2]],"date-time":"2014-10-02T00:00:00Z","timestamp":1412208000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-sa\/3.0\/"}],"funder":[{"DOI":"10.13039\/100000009","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["1R01DK08251"],"award-info":[{"award-number":["1R01DK08251"]}],"id":[{"id":"10.13039\/100000009","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000009","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["P01-HD32652"],"award-info":[{"award-number":["P01-HD32652"]}],"id":[{"id":"10.13039\/100000009","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000009","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["2R44HL072539-04"],"award-info":[{"award-number":["2R44HL072539-04"]}],"id":[{"id":"10.13039\/100000009","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["mgmjournal.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2015,2]]},"DOI":"10.1016\/j.ymgme.2014.09.011","type":"journal-article","created":{"date-parts":[[2014,10,9]],"date-time":"2014-10-09T05:57:18Z","timestamp":1412834238000},"page":"83-93","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":42,"title":["Lysosomal storage disease: Gene therapy on both sides of the blood\u2013brain barrier"],"prefix":"10.1016","volume":"114","author":[{"given":"Elena L.","family":"Aronovich","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Perry B.","family":"Hackett","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymgme.2014.09.011_bb0005","first-page":"3427","article-title":"The mucopolysaccharidoses","volume":"vol. III","author":"Neufeld","year":"2001"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0010","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1007\/s10545-008-1040-5","article-title":"Pathogenic cascades in lysosomal disease\u2014why so complex?","volume":"32","author":"Walkley","year":"2009","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0015","doi-asserted-by":"crossref","first-page":"1902","DOI":"10.1073\/pnas.252784899","article-title":"Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB","volume":"100","author":"Ohmi","year":"2003","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0020","doi-asserted-by":"crossref","first-page":"974","DOI":"10.1093\/brain\/awg089","article-title":"Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis","volume":"126","author":"Jeyakumar","year":"2003","journal-title":"Brain"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0025","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1007\/s10545-012-9508-8","article-title":"Mucopolysaccharide diseases: a complex interplay between neuroinflammation, microglial activation and adaptive immunity","volume":"36","author":"Archer","year":"2013","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0030","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1038\/nrm3565","article-title":"Signals from the lysosome: a control centre for cellular clearance and energy metabolism","volume":"14","author":"Settembre","year":"2013","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0035","doi-asserted-by":"crossref","first-page":"35765","DOI":"10.1074\/jbc.M705124200","article-title":"Lysosomal dysfunction results in altered energy balance","volume":"282","author":"Woloszynek","year":"2007","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0040","doi-asserted-by":"crossref","first-page":"1724","DOI":"10.1093\/brain\/aws095","article-title":"Contribution of brain inflammation to neuronal cell death in neuronopathic forms of Gaucher's disease","volume":"135","author":"Vitner","year":"2012","journal-title":"Brain"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0045","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.2014.09.011_bb0050","doi-asserted-by":"crossref","first-page":"1","DOI":"10.3310\/hta16390","article-title":"The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders","volume":"16","author":"Wyatt","year":"2012","journal-title":"Health Technol. Assess."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0055","doi-asserted-by":"crossref","first-page":"1333","DOI":"10.1517\/14712598.7.9.1333","article-title":"Gene therapy for mucopolysaccharidosis","volume":"7","author":"Ponder","year":"2007","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0060","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/s00439-006-0280-4","article-title":"New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy","volume":"121","author":"Beck","year":"2007","journal-title":"Hum. Genet."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0065","doi-asserted-by":"crossref","first-page":"227","DOI":"10.2174\/156652306776359522","article-title":"Cell and gene-based therapies for the lysosomal storage diseases","volume":"6","author":"Hodges","year":"2006","journal-title":"Curr. Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0070","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1093\/ilar.50.2.112","article-title":"Gene therapy for lysosomal storage diseases (LSDs) in large animal models","volume":"50","author":"Haskins","year":"2009","journal-title":"ILAR J."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0075","doi-asserted-by":"crossref","first-page":"363","DOI":"10.1007\/s10545-010-9078-6","article-title":"Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders","volume":"33","author":"Hemsley","year":"2010","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0080","doi-asserted-by":"crossref","first-page":"570","DOI":"10.1126\/science.162.3853.570","article-title":"Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts","volume":"162","author":"Fratantoni","year":"1968","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0085","doi-asserted-by":"crossref","first-page":"462","DOI":"10.1096\/fasebj.1.6.3315809","article-title":"Trafficking of lysosomal enzymes","volume":"1","author":"Kornfeld","year":"1987","journal-title":"FASEB J."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0090","doi-asserted-by":"crossref","first-page":"451","DOI":"10.1007\/s10545-012-9515-9","article-title":"An innovative approach to the treatment of Gaucher disease and possibly other metabolic disorders of the brain","volume":"36","author":"Brady","year":"2013","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0095","doi-asserted-by":"crossref","first-page":"563","DOI":"10.1016\/j.tibs.2006.08.004","article-title":"The surprising complexity of signal sequences","volume":"31","author":"Hegde","year":"2006","journal-title":"Trends Biochem. Sci."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0100","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1016\/j.ymgme.2013.11.015","article-title":"Early initiation of enzyme replacement therapy for the mucopolysaccharidoses","volume":"111","author":"Muenzer","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0105","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1016\/S1474-4422(07)70326-5","article-title":"Strategies to advance translational research into brain barriers","volume":"7","author":"Neuwelt","year":"2008","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0110","doi-asserted-by":"crossref","first-page":"209","DOI":"10.1002\/ajmg.1320460222","article-title":"Long-term outcome of Hurler syndrome following bone marrow transplantation","volume":"46","author":"Whitley","year":"1993","journal-title":"Am. J. Med. Genet."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0115","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1097\/00019052-199904000-00007","volume":"12","author":"Krivit","year":"1999","journal-title":"Curr. Opin. Neurol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0120","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1038\/sj.bmt.1703839","article-title":"Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines","volume":"31","author":"Peters","year":"2003","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0125","doi-asserted-by":"crossref","first-page":"2737","DOI":"10.1182\/blood.V83.9.2737.2737","article-title":"Cells expressing human glucocerebrosidase from a retroviral vector repopulate macrophages and central nervous system microglia after murine bone marrow transplantation","volume":"83","author":"Krall","year":"1994","journal-title":"Blood"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0130","doi-asserted-by":"crossref","first-page":"385","DOI":"10.1016\/0963-6897(95)00021-O","article-title":"Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases","volume":"4","author":"Krivit","year":"1995","journal-title":"Cell Transplant."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0135","doi-asserted-by":"crossref","first-page":"e58544","DOI":"10.1371\/journal.pone.0058544","article-title":"Bone marrow cell recruitment to the brain in the absence of irradiation or parabiosis bias","volume":"8","author":"Kierdorf","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0140","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/S1096-7192(03)00116-1","article-title":"Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow","volume":"79","author":"Zheng","year":"2003","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0145","doi-asserted-by":"crossref","first-page":"3070","DOI":"10.1172\/JCI28873","article-title":"Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice","volume":"116","author":"Biffi","year":"2006","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0150","doi-asserted-by":"crossref","first-page":"381","DOI":"10.2174\/156652312802762572","article-title":"Genetically-modified hematopoietic stem cells and their progeny for widespread and efficient protein delivery to diseased sites: the case of lysosomal storage disorders","volume":"12","author":"Biffi","year":"2012","journal-title":"Curr. Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0155","doi-asserted-by":"crossref","first-page":"1610","DOI":"10.1038\/mt.2012.82","article-title":"Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice","volume":"20","author":"Langford-Smith","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0160","doi-asserted-by":"crossref","first-page":"1233158","DOI":"10.1126\/science.1233158","article-title":"Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy","volume":"341","author":"Biffi","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0165","doi-asserted-by":"crossref","first-page":"3981","DOI":"10.1182\/blood-2012-09-455238","article-title":"Eurocord; Inborn Errors Working Party of European Blood and Marrow Transplant group; Duke University Blood and Marrow Transplantation Program; Centre for International Blood and Marrow Research","volume":"121","author":"Boelens","year":"2013","journal-title":"Blood"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0170","doi-asserted-by":"crossref","DOI":"10.1111\/bjh.13059","article-title":"Transplantation in inborn errors of metabolism: current considerations and future perspectives","author":"Boelens","year":"2014","journal-title":"Br. J. Haematol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0175","doi-asserted-by":"crossref","first-page":"1299","DOI":"10.1038\/mt.2014.68","article-title":"Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10","volume":"22","author":"Yang","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0180","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1016\/j.trsl.2012.12.005","article-title":"Evaluating risks of insertional mutagenesis by DNA transposons in gene therapy","volume":"161","author":"Hackett","year":"2013","journal-title":"Transl. Res."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0185","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1111\/j.1471-4159.2009.06544.x","article-title":"Macromolecular drug transport into the brain using targeted therapy","volume":"113","author":"Lichota","year":"2010","journal-title":"J. Neurochem."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0190","doi-asserted-by":"crossref","first-page":"648","DOI":"10.1002\/ana.23648","article-title":"The blood\u2013brain barrier in health and disease","volume":"72","author":"Daneman","year":"2012","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0195","doi-asserted-by":"crossref","first-page":"1584","DOI":"10.1038\/nm.3407","article-title":"Development, maintenance and disruption of the blood\u2013brain barrier","volume":"19","author":"Obermeier","year":"2013","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0200","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1007\/s10545-013-9608-0","article-title":"Blood\u2013brain barrier structure and function and the challenges for CNS drug delivery","volume":"36","author":"Abbott","year":"2013","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0205","doi-asserted-by":"crossref","first-page":"1566","DOI":"10.2174\/138161208784705504","article-title":"Lysosomal storage diseases and the blood\u2013brain barrier","volume":"14","author":"Begley","year":"2008","journal-title":"Curr. Pharm. Des."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0210","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1038\/jcbfm.2012.153","article-title":"Immunologic privilege in the central nervous system and the blood\u2013brain barrier","volume":"33","author":"Muldoon","year":"2013","journal-title":"J. Cereb. Blood Flow Metab."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0215","doi-asserted-by":"crossref","first-page":"703545","DOI":"10.1155\/2013\/703545","article-title":"Receptor-mediated endocytosis and brain delivery of therapeutic biologics","volume":"2013","author":"Xiao","year":"2013","journal-title":"Int. J. Cell Biol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0220","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."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0225","doi-asserted-by":"crossref","first-page":"1261","DOI":"10.1038\/mt.2008.84","article-title":"Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood\u2013brain barrier in the newborn mouse","volume":"16","author":"Urayama","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0230","doi-asserted-by":"crossref","first-page":"1959","DOI":"10.1038\/jcbfm.2012.126","article-title":"Drug transport across the blood\u2013brain barrier","volume":"32","author":"Pardridge","year":"2012","journal-title":"J. Cereb. Blood Flow Metab."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0235","doi-asserted-by":"crossref","first-page":"655","DOI":"10.1002\/emmm.201302668","article-title":"The final frontier \u2014 crossing the blood\u2013brain barrier","volume":"5","author":"Sly","year":"2013","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0240","doi-asserted-by":"crossref","first-page":"7594","DOI":"10.1073\/pnas.0702170104","article-title":"Targeted delivery of proteins across the blood\u2013brain barrier","volume":"104","author":"Spencer","year":"2007","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0245","doi-asserted-by":"crossref","first-page":"740","DOI":"10.1111\/j.1601-183X.2008.00413.x","article-title":"Effect of high-dose, repeated intra-cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice","volume":"7","author":"Hemsley","year":"2008","journal-title":"Genes Brain Behav."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0250","doi-asserted-by":"crossref","first-page":"264","DOI":"10.1016\/j.jconrel.2011.08.017","article-title":"Transport of drugs across the blood\u2013brain barrier by nanoparticles","volume":"161","author":"Wohlfart","year":"2012","journal-title":"J. Control Release"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0255","doi-asserted-by":"crossref","first-page":"393","DOI":"10.1016\/j.cbpa.2013.03.023","article-title":"Bispecific antibodies for delivery into the brain","volume":"17","author":"Watts","year":"2013","journal-title":"Curr. Opin. Chem. Biol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0260","doi-asserted-by":"crossref","first-page":"14777","DOI":"10.1073\/pnas.0506892102","article-title":"Overcoming the blood\u2013brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII","volume":"102","author":"Vogler","year":"2005","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0265","doi-asserted-by":"crossref","first-page":"889","DOI":"10.1038\/sj.mt.6300112","article-title":"Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation","volume":"15","author":"Ma","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0270","doi-asserted-by":"crossref","first-page":"1136","DOI":"10.1038\/mt.2009.87","article-title":"Systemic correction of storage disease in MPS I NOD\/SCID mice using the Sleeping Beauty transposon system","volume":"17","author":"Aronovich","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0275","doi-asserted-by":"crossref","first-page":"1549","DOI":"10.1096\/fj.05-3826fje","article-title":"Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy","volume":"19","author":"Lee","year":"2005","journal-title":"FASEB J."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0280","doi-asserted-by":"crossref","first-page":"3437","DOI":"10.1093\/hmg\/ddn237","article-title":"Reversal of peripheral and central neural storage and ataxia after recombinant enzyme replacement therapy in alpha-mannosidosis mice","volume":"17","author":"Blanz","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0285","doi-asserted-by":"crossref","first-page":"1139","DOI":"10.1093\/hmg\/ddi126","article-title":"Enzyme replacement improves nervous system pathology and function in a mouse model for metachromatic leukodystrophy","volume":"14","author":"Matzner","year":"2005","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0290","doi-asserted-by":"crossref","first-page":"600","DOI":"10.1038\/mt.2008.305","article-title":"Enzyme replacement improves ataxic gait and central nervous system histopathology in a mouse model of metachromatic leukodystrophy","volume":"17","author":"Matzner","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0295","doi-asserted-by":"crossref","first-page":"4871","DOI":"10.1093\/hmg\/ddq420","article-title":"Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy","volume":"19","author":"Polito","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0300","doi-asserted-by":"crossref","first-page":"116","DOI":"10.1016\/j.ymgme.2013.09.008","article-title":"High-dose enzyme replacement therapy in murine Hurler syndrome","volume":"111","author":"Ou","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0305","doi-asserted-by":"crossref","first-page":"501","DOI":"10.1016\/S0092-8674(00)80436-5","article-title":"Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells","volume":"91","author":"Ivics","year":"1997","journal-title":"Cell"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0310","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1038\/75568","article-title":"Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system","volume":"25","author":"Yant","year":"2000","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0315","doi-asserted-by":"crossref","first-page":"756","DOI":"10.1002\/bies.201000027","article-title":"Translating Sleeping Beauty transposition to molecular therapy: victories and challenges","volume":"32","author":"Izsvak","year":"2010","journal-title":"BioEssays"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0320","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1002\/jgm.1028","article-title":"Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses","volume":"9","author":"Aronovich","year":"2007","journal-title":"J. Gene Med."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0325","doi-asserted-by":"crossref","first-page":"495","DOI":"10.1016\/S1525-0016(03)00168-0","article-title":"Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo","volume":"8","author":"Chen","year":"2003","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0330","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1038\/mt.2012.244","article-title":"Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level","volume":"21","author":"Gracey-Maniar","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0335","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1038\/mt.2010.249","article-title":"Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of \u03b1-l-iduronidase in mice with mucopolysaccharidosis type I","volume":"19","author":"Osborn","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0340","doi-asserted-by":"crossref","first-page":"1258","DOI":"10.1038\/sj.gt.3300947","article-title":"Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA","volume":"6","author":"Liu","year":"1999","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0345","doi-asserted-by":"crossref","first-page":"1735","DOI":"10.1089\/10430349950017734","article-title":"High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA","volume":"10","author":"Zhang","year":"1999","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0350","doi-asserted-by":"crossref","first-page":"3153","DOI":"10.1038\/nprot.2007.471","article-title":"Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection","volume":"2","author":"Bell","year":"2007","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0355","doi-asserted-by":"crossref","first-page":"210","DOI":"10.1089\/hum.2009.128","article-title":"Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons","volume":"21","author":"Podetz-Pedersen","year":"2010","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0360","doi-asserted-by":"crossref","first-page":"749","DOI":"10.1038\/360749a0","article-title":"Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer","volume":"360","author":"Wolfe","year":"1992","journal-title":"Nature"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0365","doi-asserted-by":"crossref","first-page":"1423","DOI":"10.1038\/sj.mt.6300201","article-title":"Correction of clinical manifestations of canine mucopolysacchariodsis I with neonatal retroviral vector gene therapy","volume":"33","author":"Traas","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0370","doi-asserted-by":"crossref","first-page":"972","DOI":"10.1002\/jgm.1229","article-title":"Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice","volume":"10","author":"Herati","year":"2008","journal-title":"J. Gene Med."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0375","doi-asserted-by":"crossref","first-page":"334","DOI":"10.1038\/mt.2009.236","article-title":"A self-inactivating gamma-retroviral vector reduces manifestations of mucopolysaccharidosis I in mice","volume":"18","author":"Metcalf","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0380","doi-asserted-by":"crossref","first-page":"1025","DOI":"10.1038\/mt.2011.34","article-title":"Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 transblood\u2013brain barrier gene delivery","volume":"19","author":"Fu","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0385","doi-asserted-by":"crossref","first-page":"e27209","DOI":"10.4161\/rdis.27209","article-title":"Toward a gene therapy for neurological and somatic MPSIIIA","volume":"1","author":"Haurigot","year":"2013","journal-title":"Rare Dis."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0390","doi-asserted-by":"crossref","DOI":"10.1038\/mt.2014.135","article-title":"Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I","volume":"22","author":"Hinderer","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0395","doi-asserted-by":"crossref","first-page":"3254","DOI":"10.1172\/JCI66778","article-title":"Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy","volume":"123","author":"Haurigot","year":"2013","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0400","doi-asserted-by":"crossref","first-page":"6381","DOI":"10.1128\/JVI.78.12.6381-6388.2004","article-title":"Clades of adeno-associated viruses are widely disseminated in human tissues","volume":"78","author":"Gao","year":"2004","journal-title":"J. Virol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0405","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1038\/nbt.1515","article-title":"Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes","volume":"27","author":"Foust","year":"2009","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0410","doi-asserted-by":"crossref","first-page":"1187","DOI":"10.1038\/mt.2009.71","article-title":"Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons","volume":"17","author":"Duque","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0415","doi-asserted-by":"crossref","first-page":"1871","DOI":"10.1038\/mt.2011.157","article-title":"Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders","volume":"19","author":"Bevan","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0420","doi-asserted-by":"crossref","first-page":"1237","DOI":"10.1089\/hum.2012.029","article-title":"Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer","volume":"23","author":"Ruzo","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0425","doi-asserted-by":"crossref","first-page":"1440","DOI":"10.1038\/mt.2011.98","article-title":"Several rAAV vectors efficiently cross the blood\u2013brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system","volume":"19","author":"Zhang","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0430","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1038\/gt.2014.16","article-title":"Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats","volume":"21","author":"Bucher","year":"2014","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0435","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1038\/mt.2010.240","article-title":"Efficacy and safety of long-term prophylaxis in severe hemophilia a dogs following liver gene therapy using AAV vectors","volume":"19","author":"Sabatino","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0440","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1089\/hum.2011.106","article-title":"Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia","volume":"23","author":"Demaster","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0445","doi-asserted-by":"crossref","first-page":"28","DOI":"10.1038\/gt.2013.54","article-title":"Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain","volume":"21","author":"Swain","year":"2014","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0450","doi-asserted-by":"crossref","first-page":"852","DOI":"10.1038\/gt.2011.130","article-title":"Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs","volume":"19","author":"Federici","year":"2012","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0455","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1038\/gt.2011.216","article-title":"Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems","volume":"20","author":"Mattar","year":"2013","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0460","doi-asserted-by":"crossref","first-page":"72","DOI":"10.1089\/humc.2013.208","article-title":"Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates","volume":"25","author":"Murrey","year":"2014","journal-title":"Hum. Gene Ther. Clin. Dev."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0465","doi-asserted-by":"crossref","first-page":"480","DOI":"10.1182\/blood-2002-09-2889","article-title":"Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway","volume":"102","author":"Davidoff","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0470","doi-asserted-by":"crossref","first-page":"3079","DOI":"10.1182\/blood-2004-10-3867","article-title":"Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2\/8-mediated, liver-directed gene therapy","volume":"105","author":"Wang","year":"2005","journal-title":"Blood"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0475","doi-asserted-by":"crossref","first-page":"60","DOI":"10.1038\/gt.2008.137","article-title":"Enhancing transduction of the liver by adeno-associated viral vectors","volume":"16","author":"Nathwani","year":"2009","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0480","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1038\/mt.2011.220","article-title":"Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice","volume":"20","author":"Ruzo","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0485","doi-asserted-by":"crossref","first-page":"e78161","DOI":"10.1371\/journal.pone.0078161","article-title":"Quantitative analysis of \u03b1-l-iduronidase expression in immunocompetent mice treated with the Sleeping Beauty transposon system","volume":"8","author":"Aronovich","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0490","doi-asserted-by":"crossref","first-page":"1470","DOI":"10.1038\/mt.2013.95","article-title":"Mouse gender influences brain transduction by intravascularly administered AAV9","volume":"21","author":"Maguire","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0495","doi-asserted-by":"crossref","first-page":"2357","DOI":"10.1056\/NEJMoa1108046","article-title":"Adenovirus-associated virus vector-mediated gene transfer in hemophilia B","volume":"365","author":"Nathwani","year":"2011","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0500","doi-asserted-by":"crossref","first-page":"342","DOI":"10.1038\/nm1358","article-title":"Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response","volume":"12","author":"Manno","year":"2006","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0505","doi-asserted-by":"crossref","first-page":"321","DOI":"10.2174\/156652311796150354","article-title":"Immune responses to AAV in clinical trials","volume":"11","author":"Mingozzi","year":"2011","journal-title":"Curr. Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0510","doi-asserted-by":"crossref","first-page":"419","DOI":"10.1038\/nm1549","article-title":"CD8(+) T-cell responses to adeno-associated virus capsid in humans","volume":"13","author":"Mingozzi","year":"2007","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0515","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1038\/mt.2011.177","article-title":"Neutralizing antibodies against AAV serotypes 1, 2, 6, and 9 in sera of commonly used animal models","volume":"20","author":"Rapti","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0520","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1038\/gt.2012.101","article-title":"Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates","volume":"20","author":"Gray","year":"2013","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0525","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1038\/mt.2013.266","article-title":"AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinflammation driven by antigen-presenting cell transduction","volume":"22","author":"Samaranch","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0530","doi-asserted-by":"crossref","first-page":"15018","DOI":"10.1073\/pnas.1205858109","article-title":"Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation","volume":"109","author":"Capotondo","year":"2012","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0535","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1093\/glycob\/11.1.99","article-title":"A novel missense mutation in lysosomal sulfamidase is the basis of MPS III A in a spontaneous mouse mutant","volume":"11","author":"Bhattacharyya","year":"2001","journal-title":"Glycobiology"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0540","doi-asserted-by":"crossref","first-page":"1389","DOI":"10.1093\/glycob\/9.12.1389","article-title":"A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome)","volume":"9","author":"Bhaumik","year":"1999","journal-title":"Glycobiology"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0545","doi-asserted-by":"crossref","first-page":"2693","DOI":"10.1093\/hmg\/ddm223","article-title":"Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes","volume":"16","author":"Fraldi","year":"2007","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0550","doi-asserted-by":"crossref","first-page":"e35787","DOI":"10.1371\/journal.pone.0035787","article-title":"Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB","volume":"7","author":"Wilkinson","year":"2012","journal-title":"PLoS One"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0555","doi-asserted-by":"crossref","first-page":"191","DOI":"10.1016\/j.bbr.2004.08.019","article-title":"Development of motor deficits in a murine model of mucopolysaccharidosis type IIIA (MPS-IIIA)","volume":"158","author":"Hemsley","year":"2005","journal-title":"Behav. Brain Res."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0560","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1038\/mt.2010.265","article-title":"Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes","volume":"19","author":"Ellinwood","year":"2011","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0565","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1007\/s10545-012-9530-x","article-title":"Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits","volume":"36","author":"Baldo","year":"2013","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0570","doi-asserted-by":"crossref","first-page":"4708","DOI":"10.1073\/pnas.062047599","article-title":"Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of l-dopa using rAAV-mediated gene transfer","volume":"99","author":"Kirik","year":"2002","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0575","doi-asserted-by":"crossref","first-page":"5130","DOI":"10.1182\/blood-2010-04-278234","article-title":"Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model","volume":"116","author":"Visigalli","year":"2010","journal-title":"Blood"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0580","doi-asserted-by":"crossref","first-page":"197","DOI":"10.1016\/j.ijporl.2006.09.016","article-title":"Age-related functional and histopathological changes of the ear in the MPS I mouse","volume":"71","author":"Schachern","year":"2007","journal-title":"Int. J. Pediatr. Otorhinolaryngol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0585","doi-asserted-by":"crossref","first-page":"1323","DOI":"10.1097\/MAO.0b013e3182659cc3","article-title":"Inner ear changes in mucopolysaccharidosis type I\/Hurler syndrome","volume":"33","author":"Kariya","year":"2012","journal-title":"Otol. Neurotol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0590","doi-asserted-by":"crossref","first-page":"1605","DOI":"10.1038\/mt.2014.114","article-title":"Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates","volume":"22","author":"Chuah","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0595","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1002\/emmm.201202083","article-title":"A highly secreted sulphamidase engineered to cross the blood\u2013brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA","volume":"5","author":"Sorrentino","year":"2013","journal-title":"EMBO J. Mol. Med."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0600","doi-asserted-by":"crossref","first-page":"1938","DOI":"10.1038\/mt.2013.141","article-title":"Myeloid\/microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease","volume":"21","author":"Sergijenko","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0605","doi-asserted-by":"crossref","first-page":"2999","DOI":"10.1073\/pnas.1222742110","article-title":"Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood\u2013brain barrier","volume":"110","author":"Wang","year":"2013","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0610","doi-asserted-by":"crossref","first-page":"e28881","DOI":"10.1371\/journal.pone.0028881","article-title":"A carrier for non-covalent delivery of functional beta-galactosidase and antibodies against amyloid plaques and IgM to the brain","volume":"6","author":"Sarkar","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0615","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1038\/mt.2013.267","article-title":"Effective intravenous therapy for neurodegenerative disease with a therapeutic enzyme and a peptide that mediates delivery to the brain","volume":"22","author":"Meng","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0620","doi-asserted-by":"crossref","first-page":"2","DOI":"10.1016\/j.addr.2013.08.008","article-title":"Drug delivery to the central nervous system by polymeric nanoparticles: what do we know?","volume":"71","author":"Kreuter","year":"2014","journal-title":"Adv. Drug Deliv. Rev."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0625","doi-asserted-by":"crossref","first-page":"183ra57","DOI":"10.1126\/scitranslmed.3005338","article-title":"Addressing safety liabilities of TfR bispecific antibodies that cross the blood\u2013brain barrier","volume":"5","author":"Couch","year":"2013","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0630","doi-asserted-by":"crossref","first-page":"2669","DOI":"10.1073\/pnas.0437941100","article-title":"Transgene produces massive overexpression of human beta-glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors","volume":"100","author":"Vogler","year":"2003","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0635","doi-asserted-by":"crossref","first-page":"821","DOI":"10.1089\/hum.2007.048","article-title":"Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy","volume":"18","author":"Capotondo","year":"2007","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0640","doi-asserted-by":"crossref","first-page":"58ra84","DOI":"10.1126\/scitranslmed.3001522","article-title":"Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy","volume":"2","author":"Gentner","year":"2010","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0645","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1038\/nature13420","article-title":"Targeted genome editing in human repopulating haematopoietic stem cells","volume":"510","author":"Genovese","year":"2014","journal-title":"Nature"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0650","doi-asserted-by":"crossref","first-page":"94","DOI":"10.1093\/ilar.47.2.94","article-title":"Mouse phenome research: implications of genetic background","volume":"47","author":"Yoshiki","year":"2006","journal-title":"ILAR J."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0655","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1186\/1745-6150-9-18","article-title":"Parallel universes of Black Six biology","volume":"9","author":"Kraev","year":"2014","journal-title":"Biol. Direct"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0660","doi-asserted-by":"crossref","first-page":"1001","DOI":"10.1126\/science.291.5506.1001","article-title":"Principles for the buffering of genetic variation","volume":"291","author":"Hartman","year":"2001","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0665","doi-asserted-by":"crossref","first-page":"993","DOI":"10.1002\/bies.950181209","article-title":"Mouse models of human genetic disease: which mouse is more like man?","volume":"18","author":"Erickson","year":"1996","journal-title":"BioEssays"},{"issue":"R1","key":"10.1016\/j.ymgme.2014.09.011_bb0670","doi-asserted-by":"crossref","first-page":"14","DOI":"10.1093\/hmg\/ddr140","article-title":"The Sleeping Beauty transposon system: a non-viral vector for gene therapy","volume":"20","author":"Aronovich","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0675","doi-asserted-by":"crossref","first-page":"341","DOI":"10.2174\/156652311797415827","article-title":"Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies","volume":"11","author":"Hackett","year":"2011","journal-title":"Curr. Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0680","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1016\/B978-0-12-404742-6.00002-8","article-title":"Precision editing of large animal genomes","volume":"80","author":"Tan","year":"2012","journal-title":"Adv. Genet."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0685","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1016\/j.brainres.2006.01.008","article-title":"Long-term memory for aversive training is impaired in Idua(\u2212\/\u2212) mice, a genetic model of mucopolysaccharidosis type I","volume":"1076","author":"Reolon","year":"2006","journal-title":"Brain Res."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0690","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1016\/j.brainresbull.2007.07.018","article-title":"Characterization of an immunodeficient mouse model of mucopolysaccharidosis type 1 suitable for preclinical testing of human stem cell and gene therapy","volume":"74","author":"Garcia-Rivera","year":"2007","journal-title":"Brain Res. Bull."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0695","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1016\/j.brainres.2007.10.036","article-title":"Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome","volume":"1188","author":"Pan","year":"2008","journal-title":"Brain Res."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0700","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1016\/j.nbd.2011.02.015","article-title":"Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I","volume":"43","author":"Wolf","year":"2011","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0705","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.brainres.2006.05.079","article-title":"Characterization of a C57BL\/6 congenic mouse strain of mucopolysaccharidosis type IIIA","volume":"1104","author":"Crawley","year":"2006","journal-title":"Brain Res."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0710","doi-asserted-by":"crossref","first-page":"130","DOI":"10.1016\/j.bbr.2008.03.024","article-title":"Open field locomotor activity and anxiety-related behaviors in mucopolysaccharidosistype IIIA mice","volume":"191","author":"Lau","year":"2008","journal-title":"Behav. Brain Res."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0715","doi-asserted-by":"crossref","first-page":"e80142","DOI":"10.1371\/journal.pone.0080142","article-title":"Amyloidosis, synucleinopathy, and prion encephalopathy in a neuropathic lysosomal storage disease: the CNS-biomarker potential of peripheral blood","volume":"8","author":"Naughton","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.ymgme.2014.09.011_bb0720","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1002\/ana.24053","article-title":"Metallothioneins as dynamic markers for brain disease in lysosomal disorders","volume":"75","author":"Cesani","year":"2014","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0725","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1016\/j.bbr.2012.08.020","article-title":"High-throughput screening of stem cell therapy for globoid cell leukodystrophy using automated neurophenotyping of twitcher mice","volume":"236","author":"Scruggs","year":"2013","journal-title":"Behav. Brain Res."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0730","doi-asserted-by":"crossref","first-page":"506","DOI":"10.1089\/hum.2013.238","article-title":"Intracerebral administration of AAV rh.10 carrying human SGSH and SUMF1 cDNAs in children with MPSIIIA disease: results of a phase I\/II trial","volume":"25","author":"Tardieu","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0735","doi-asserted-by":"crossref","first-page":"263","DOI":"10.31887\/DCNS.2013.15.3\/goz","article-title":"Animal models and high field imaging and spectroscopy","volume":"15","author":"Oz","year":"2013","journal-title":"Dialogues Clin. Neurosci."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0740","doi-asserted-by":"crossref","first-page":"1299","DOI":"10.1172\/JCI67677","article-title":"Brain-wide pathway for waste clearance captured by contrast-enhanced MRI","volume":"123","author":"Iliff","year":"2013","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.ymgme.2014.09.011_bb0745","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1016\/j.neuroimage.2013.05.041","article-title":"The WU-Minn Human Connectome Project: an overview","volume":"80","author":"Van Essen","year":"2013","journal-title":"Neuroimage"}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719214003060?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719214003060?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2022,4,19]],"date-time":"2022-04-19T13:34:13Z","timestamp":1650375253000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719214003060"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015,2]]},"references-count":149,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2015,2]]}},"alternative-id":["S1096719214003060"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2014.09.011","relation":{},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2015,2]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Lysosomal storage disease: Gene therapy on both sides of the blood\u2013brain barrier","name":"articletitle","label":"Article Title"},{"value":"Molecular Genetics and Metabolism","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymgme.2014.09.011","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2014 The Authors. Published by Elsevier Inc.","name":"copyright","label":"Copyright"}]}}