{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,9]],"date-time":"2026-05-09T02:03:57Z","timestamp":1778292237855,"version":"3.51.4"},"reference-count":72,"publisher":"Oxford University Press (OUP)","issue":"8","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2017,4,15]]},"DOI":"10.1093\/hmg\/ddx058","type":"journal-article","created":{"date-parts":[[2017,2,16]],"date-time":"2017-02-16T20:17:01Z","timestamp":1487276221000},"page":"1535-1551","source":"Crossref","is-referenced-by-count":52,"title":["Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID"],"prefix":"10.1093","volume":"26","author":[{"given":"Carles","family":"Roca","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sandra","family":"Motas","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sara","family":"Marc\u00f3","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Albert","family":"Ribera","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"V\u00edctor","family":"S\u00e1nchez","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xavier","family":"S\u00e1nchez","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joan","family":"Bertolin","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xavier","family":"Le\u00f3n","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer","family":"P\u00e9rez","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Miguel","family":"Garcia","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pilar","family":"Villacampa","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jes\u00fas","family":"Ruberte","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anna","family":"Pujol","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Virginia","family":"Haurigot","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fatima","family":"Bosch","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"286","published-online":{"date-parts":[[2017,2,17]]},"reference":[{"key":"key\n\t\t\t\t20170417091747_ddx058-B1","doi-asserted-by":"crossref","first-page":"240","DOI":"10.1007\/s10545-008-0838-5","article-title":"Sanfilippo syndrome: a mini-review","volume":"31","year":"2008","journal-title":"J. Inherit. Metab. Dis"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B2","first-page":"269","article-title":"Sanfilippo syndrome: causes, consequences, and treatments","volume":"8","year":"2015","journal-title":"Appl. Clin. Genet"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B3","first-page":"1348","article-title":"Mucopolysaccharidosis type IIID: 12 new patients and 15 novel mutations","volume":"31","year":"2010","journal-title":"Hum. Mutat"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B4","doi-asserted-by":"crossref","first-page":"192","DOI":"10.1136\/jmg.40.3.192","article-title":"Sanfilippo syndrome type D: identification of the first mutation in the N-acetylglucosamine-6-sulphatase gene","volume":"40","year":"2003","journal-title":"J. Med. Genet"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B5","doi-asserted-by":"crossref","first-page":"1629","DOI":"10.1001\/archneur.64.11.1629","article-title":"Sanfilippo syndrome type D: natural history and identification of 3 novel mutations in the GNS Gene","volume":"64","year":"2007","journal-title":"Arch. Neurol"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B6","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/S0888-7543(02)00014-9","article-title":"Genomic basis of mucopolysaccharidosis type IIID (MIM 252940) revealed by sequencing of GNS encoding N-acetylglucosamine-6-sulfatase","volume":"81","year":"2003","journal-title":"Genomics"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B7","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1016\/j.ymgme.2006.07.014","article-title":"Identification and characterisation of an 8.7\u2009kb deletion and a novel nonsense mutation in two Italian families with Sanfilippo syndrome type D (mucopolysaccharidosis IIID)","volume":"90","year":"2007","journal-title":"Mol. Genet. Metab"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B8","first-page":"133","article-title":"A novel loss-of-function mutation in the GNS gene causes Sanfilippo syndrome type D","volume":"20","year":"2009","journal-title":"Genet. Couns"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B9","doi-asserted-by":"crossref","first-page":"152","DOI":"10.1111\/j.1399-0004.1981.tb01821.x","article-title":"Genetic heterogeneity and clinical variability in the Sanfilippo syndrome (types A, B, and C)","volume":"20","year":"1981","journal-title":"Clin. Genet"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B10","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1136\/jmg.28.6.402","article-title":"Sanfilippo syndrome type D in two adolescent sisters","volume":"28","year":"1991","journal-title":"J. Med. Genet"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B11","doi-asserted-by":"crossref","first-page":"168","DOI":"10.1007\/BF00441147","article-title":"Sanfilippo type D disease: clinical findings in two patients with a new variant of mucopolysaccharidosis III","volume":"138","year":"1982","journal-title":"Eur. J. Pediatr"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B12","doi-asserted-by":"crossref","first-page":"130","DOI":"10.1007\/BF00441662","article-title":"Clinical heterogeneity in Sanfilippo disease (mucopolysaccharidosis III) type D: presentation of two new cases","volume":"140","year":"1983","journal-title":"Eur. J. Pediatr"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B13","doi-asserted-by":"crossref","first-page":"E12.","DOI":"10.3171\/FOC\/2008\/24\/3-4\/E11","article-title":"Central nervous system therapy for lysosomal storage disorders","volume":"24","year":"2008","journal-title":"Neurosurg. Focus"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B14","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","year":"2011","journal-title":"Mol. Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B15","doi-asserted-by":"crossref","first-page":"350","DOI":"10.1089\/hum.2008.155","article-title":"Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution","volume":"20","year":"2009","journal-title":"Hum. Gene Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B16","doi-asserted-by":"crossref","first-page":"115","DOI":"10.3171\/2010.4.PEDS09507","article-title":"Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations","volume":"6","year":"2010","journal-title":"J. Neurosurg. Pediatr"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B17","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","year":"2009","journal-title":"Nat. Biotechnol"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B18","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","year":"2009","journal-title":"Mol. Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B19","doi-asserted-by":"crossref","first-page":"1073","DOI":"10.1038\/mt.2008.76","article-title":"Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection","volume":"16","year":"2008","journal-title":"Mol. Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B20","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","year":"2012","journal-title":"Hum. Gene Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B21","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 trans-blood-brain barrier gene delivery","volume":"19","year":"2011","journal-title":"Mol. Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B22","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","year":"2013","journal-title":"J. Clin. Invest"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B23","doi-asserted-by":"crossref","first-page":"2078","DOI":"10.1093\/hmg\/ddu727","article-title":"Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy","volume":"24","year":"2015","journal-title":"Hum. Mol. Genet"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B24","doi-asserted-by":"crossref","first-page":"1298","DOI":"10.1038\/mt.2015.99","article-title":"Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates","volume":"23","year":"2015","journal-title":"Mol. Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B25","first-page":"e86696.","article-title":"CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)","volume":"1","year":"2016","journal-title":"JCI Insight"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B26","doi-asserted-by":"crossref","first-page":"906","DOI":"10.1089\/hum.2016.101","article-title":"Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice","volume":"27","year":"2016","journal-title":"Hum. Gene Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B27","doi-asserted-by":"crossref","first-page":"206","DOI":"10.1038\/mt.2015.189","article-title":"Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII","volume":"24","year":"2016","journal-title":"Mol. Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B28","doi-asserted-by":"crossref","first-page":"1158","DOI":"10.1097\/00005072-199710000-00010","article-title":"Human mucopolysaccharidosis IIID: clinical, biochemical, morphological and immunohistochemical characteristics","volume":"56","year":"1997","journal-title":"J. Neuropathol. Exp. Neurol"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B29","doi-asserted-by":"crossref","first-page":"999","DOI":"10.1242\/dmm.025171","article-title":"Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC","volume":"9","year":"2016","journal-title":"Dis. Model. Mech"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B30","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","year":"2003","journal-title":"Proc. Natl. Acad. Sci. U.S.A"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B31","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","year":"2012","journal-title":"PloS One"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B32","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/s10545-013-9613-3","article-title":"Mucopolysaccharide diseases: a complex interplay between neuroinflammation, microglial activation and adaptive immunity","volume":"37","year":"2014","journal-title":"J. Inherit. Metab. Dis"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B33","first-page":"417.","article-title":"Neuroinflammatory paradigms in lysosomal storage diseases","volume":"9","year":"2015","journal-title":"Front. Neurosci"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B34","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1111\/j.1750-3639.1994.tb00838.x","article-title":"GFAP and astrogliosis","volume":"4","year":"1994","journal-title":"Brain. Pathol"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B35","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1007\/BF01795308","article-title":"Lectin binding by resting and reactive microglia","volume":"16","year":"1987","journal-title":"J. Neurocytol"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B36","doi-asserted-by":"crossref","first-page":"624","DOI":"10.1002\/jgm.1480","article-title":"Restoration of central nervous system alpha-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery","volume":"12","year":"2010","journal-title":"J. Gene Med"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B37","doi-asserted-by":"crossref","first-page":"15055.","DOI":"10.1038\/mtm.2015.55","article-title":"AAV9-mediated central nervous system-targeted gene delivery via cisterna magna route in mice","volume":"3","year":"2016","journal-title":"Mol. Ther. Methods. Clin. Dev"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B38","doi-asserted-by":"crossref","first-page":"473","DOI":"10.1126\/science.1174447","article-title":"A gene network regulating lysosomal biogenesis and function","volume":"325","year":"2009","journal-title":"Science"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B39","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1038\/mt.2014.210","article-title":"Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates","volume":"23","year":"2015","journal-title":"Mol. Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B40","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","year":"2013","journal-title":"Gene Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B41","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1089\/hum.2011.200","article-title":"Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates","volume":"23","year":"2012","journal-title":"Hum. Gene Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B42","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","year":"2012","journal-title":"Mol. Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B43","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1007\/s10545-012-9522-x","article-title":"Assessment of bone dysplasia by micro-CT and glycosaminoglycan levels in mouse models for mucopolysaccharidosis type I, IIIA, IVA, and VII","volume":"36","year":"2013","journal-title":"J. Inherit. Metab. Dis"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B44","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1007\/s10545-005-5673-3","article-title":"Keratan sulphate levels in mucopolysaccharidoses and mucolipidoses","volume":"28","year":"2005","journal-title":"J. Inherit. Metab. Dis"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B45","doi-asserted-by":"crossref","first-page":"154","DOI":"10.1111\/j.1365-2567.2009.03225.x","article-title":"Inflammation in neurodegenerative diseases","volume":"129","year":"2010","journal-title":"Immunology"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B46","doi-asserted-by":"crossref","first-page":"388","DOI":"10.1016\/S1474-4422(15)70016-5","article-title":"Neuroinflammation in Alzheimer's disease","volume":"14","year":"2015","journal-title":"Lancet. Neurol"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B47","doi-asserted-by":"crossref","first-page":"612","DOI":"10.1002\/jnr.21134","article-title":"Cytokines, neurotrophins, and oxidative stress in brain disease from mucopolysaccharidosis IIIB","volume":"85","year":"2007","journal-title":"J. Neurosci. Res"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B48","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1385\/JMN:15:3:251","article-title":"Caprine mucopolysaccharidosis IIID: a preliminary trial of enzyme replacement therapy","volume":"15","year":"2000","journal-title":"J. Mol. Neurosci"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B49","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1038\/gim.2015.36","article-title":"A phase I\/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II","volume":"18","year":"2016","journal-title":"Genet. Med"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B50","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","year":"2013","journal-title":"Science"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B51","doi-asserted-by":"crossref","first-page":"476","DOI":"10.1016\/S0140-6736(16)30374-9","article-title":"Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1\/2 trial","volume":"388","year":"2016","journal-title":"Lancet"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B52","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","year":"2006","journal-title":"J. Clin. Invest"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B53","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1038\/nrg2985","article-title":"Ex vivo gene transfer and correction for cell-based therapies","volume":"12","year":"2011","journal-title":"Nat. Rev. Genet"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B54","doi-asserted-by":"crossref","first-page":"3132","DOI":"10.1172\/JCI35700","article-title":"Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1","volume":"118","year":"2008","journal-title":"J. Clin. Invest"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B55","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1126\/science.1088547","article-title":"LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1","volume":"302","year":"2003","journal-title":"Science"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B56","doi-asserted-by":"crossref","first-page":"2240","DOI":"10.1056\/NEJMoa0802315","article-title":"Safety and efficacy of gene transfer for Leber's congenital amaurosis","volume":"358","year":"2008","journal-title":"N. Engl. J. Med"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B57","doi-asserted-by":"crossref","first-page":"1994","DOI":"10.1056\/NEJMoa1407309","article-title":"Long-term safety and efficacy of factor IX gene therapy in hemophilia B","volume":"371","year":"2014","journal-title":"N. Engl. J. Med"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B58","doi-asserted-by":"crossref","first-page":"361","DOI":"10.1038\/gt.2012.43","article-title":"Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial","volume":"20","year":"2013","journal-title":"Gene Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B59","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1016\/j.coph.2015.07.006","article-title":"Adeno-associated virus serotypes for gene therapeutics","volume":"24","year":"2015","journal-title":"Curr. Opin. Pharmacol"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B60","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1517\/17425247.2015.966682","article-title":"Gene therapy for neurologic manifestations of mucopolysaccharidoses","volume":"12","year":"2015","journal-title":"Expert. Opin. Drug. Deliv"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B61","doi-asserted-by":"crossref","first-page":"3038","DOI":"10.1182\/blood-2011-09-382317","article-title":"Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer","volume":"119","year":"2012","journal-title":"Blood"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B62","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","year":"2013","journal-title":"Rare Dis"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B63","doi-asserted-by":"crossref","first-page":"2018","DOI":"10.1038\/mt.2014.135","article-title":"Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I","volume":"22","year":"2014","journal-title":"Mol. Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B64","doi-asserted-by":"crossref","first-page":"3321","DOI":"10.1182\/blood-2006-04-017913","article-title":"Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy","volume":"108","year":"2006","journal-title":"Blood"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B65","doi-asserted-by":"crossref","first-page":"1586","DOI":"10.1128\/CVI.05107-11","article-title":"Adeno-associated virus antibody profiles in newborns, children, and adolescents","volume":"18","year":"2011","journal-title":"Clin. Vaccine Immunol"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B66","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1146\/annurev.bi.60.070191.001353","article-title":"Lysosomal storage diseases","volume":"60","year":"1991","journal-title":"Annu. Rev. Biochem"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B67","volume-title":"National Neurosurgical Procedural Statistics 2006 Survey","author":"American Association of Neurological Surgeons","year":"2006"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B68","doi-asserted-by":"crossref","first-page":"1391","DOI":"10.1089\/104303402760128612","article-title":"Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain","volume":"13","year":"2002","journal-title":"Hum. Gene Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B69","first-page":"165ra163.","article-title":"Long-term follow-up after gene therapy for canavan disease","volume":"4","year":"2012","journal-title":"Sci. Transl. Med"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B70","doi-asserted-by":"crossref","first-page":"935","DOI":"10.1007\/BF00711508","article-title":"A fluorimetric enzyme assay for the diagnosis of Sanfilippo disease type D (MPS IIID)","volume":"16","year":"1993","journal-title":"J. Inherit. Metab. Dis"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B71","doi-asserted-by":"crossref","first-page":"503","DOI":"10.1038\/gt.2009.157","article-title":"High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency","volume":"17","year":"2010","journal-title":"Gene Ther"},{"key":"key\n\t\t\t\t20170417091747_ddx058-B72","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1023\/A:1006237917632","article-title":"A simple mouse model for leptomeningeal metastases and repeated intrathecal therapy","volume":"42","year":"1999","journal-title":"J. Neuroncol"}],"container-title":["Human Molecular Genetics"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/academic.oup.com\/hmg\/article-pdf\/26\/8\/1535\/13629153\/ddx058.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,6,15]],"date-time":"2025-06-15T12:35:45Z","timestamp":1749990945000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/hmg\/article-lookup\/doi\/10.1093\/hmg\/ddx058"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017,2,17]]},"references-count":72,"journal-issue":{"issue":"8","published-online":{"date-parts":[[2017,2,17]]},"published-print":{"date-parts":[[2017,4,15]]}},"URL":"https:\/\/doi.org\/10.1093\/hmg\/ddx058","relation":{},"ISSN":["0964-6906","1460-2083"],"issn-type":[{"value":"0964-6906","type":"print"},{"value":"1460-2083","type":"electronic"}],"subject":[],"published":{"date-parts":[[2017,2,17]]}}}