{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,12]],"date-time":"2026-09-12T04:42:45Z","timestamp":1789188165118,"version":"build-2803163510"},"reference-count":85,"publisher":"Oxford University Press (OUP)","issue":"7","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2015,4,1]]},"DOI":"10.1093\/hmg\/ddu727","type":"journal-article","created":{"date-parts":[[2014,12,19]],"date-time":"2014-12-19T11:15:43Z","timestamp":1418987743000},"page":"2078-2095","source":"Crossref","is-referenced-by-count":51,"title":["Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy"],"prefix":"10.1093","volume":"24","author":[{"given":"Albert","family":"Ribera","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Virginia","family":"Haurigot","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miguel","family":"Garcia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara","family":"Marc\u00f3","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra","family":"Motas","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pilar","family":"Villacampa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luca","family":"Maggioni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xavier","family":"Le\u00f3n","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria","family":"Molas","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"V\u00edctor","family":"S\u00e1nchez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sergio","family":"Mu\u00f1oz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christian","family":"Leborgne","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xavier","family":"Moll","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mart\u00ed","family":"Pumarola","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federico","family":"Mingozzi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jes\u00fas","family":"Ruberte","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"S\u00f2nia","family":"A\u00f1or","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fatima","family":"Bosch","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2014,12,18]]},"reference":[{"key":"key\n\t\t\t\t20170503081521_DDU727C1","doi-asserted-by":"crossref","first-page":"535","DOI":"10.1111\/j.1432-1033.1977.tb11909.x","article-title":"Human alpha-n-acetylglucosaminidase. 2. Activity towards natural substrates and multiple recognition forms","volume":"80","author":"Von Figura","year":"1977","journal-title":"Eur. J. Biochem."},{"key":"key\n\t\t\t\t20170503081521_DDU727C2","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1016\/j.ymgme.2010.01.004","article-title":"Identification and characterization of a novel homozygous deletion in the alpha-N-acetylglucosaminidase gene in a patient with Sanfilippo type B syndrome (mucopolysaccharidosis IIIB)","volume":"100","author":"Champion","year":"2010","journal-title":"Mol. Genet. Metab."},{"key":"key\n\t\t\t\t20170503081521_DDU727C3","doi-asserted-by":"crossref","first-page":"264","DOI":"10.1002\/humu.1189","article-title":"Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications","volume":"18","author":"Yogalingam","year":"2001","journal-title":"Hum. Mutat."},{"key":"key\n\t\t\t\t20170503081521_DDU727C4","doi-asserted-by":"crossref","first-page":"723","DOI":"10.1083\/jcb.201208152","article-title":"The cell biology of disease: lysosomal storage disorders: the cellular impact of lysosomal dysfunction","volume":"199","author":"Platt","year":"2012","journal-title":"J. Cell Biol."},{"key":"key\n\t\t\t\t20170503081521_DDU727C5","doi-asserted-by":"crossref","first-page":"599","DOI":"10.1016\/S0387-7604(85)80008-5","article-title":"Neuropathological study and chemico-pathological correlation in sibling cases of Sanfilippo syndrome type B","volume":"7","author":"Tamagawa","year":"1985","journal-title":"Brain Dev."},{"key":"key\n\t\t\t\t20170503081521_DDU727C6","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1007\/s00401-007-0325-3","article-title":"Mechanisms of neurodegeneration in mucopolysaccharidoses II and IIIB: analysis of human brain tissue","volume":"115","author":"Hamano","year":"2008","journal-title":"Acta Neuropathol."},{"key":"key\n\t\t\t\t20170503081521_DDU727C7","doi-asserted-by":"crossref","first-page":"240","DOI":"10.1007\/s10545-008-0838-5","article-title":"Sanfilippo syndrome: a mini-review","volume":"31","author":"Valstar","year":"2008","journal-title":"J. Inherit. Metab. Dis."},{"key":"key\n\t\t\t\t20170503081521_DDU727C8","first-page":"3421","article-title":"The mucopolysaccharidoses","volume-title":"The Online Metabolic and Molecular Bases of Inherited Disease","author":"Neufeld","year":"2001"},{"key":"key\n\t\t\t\t20170503081521_DDU727C9","doi-asserted-by":"crossref","first-page":"759","DOI":"10.1007\/s10545-010-9199-y","article-title":"Mucopolysaccharidosis type IIIB may predominantly present with an attenuated clinical phenotype","volume":"33","author":"Valstar","year":"2010","journal-title":"J. Inherit. Metab. Dis."},{"key":"key\n\t\t\t\t20170503081521_DDU727C10","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","author":"Van de Kamp","year":"1981","journal-title":"Clin. Genet."},{"key":"key\n\t\t\t\t20170503081521_DDU727C11","doi-asserted-by":"crossref","first-page":"e1255","DOI":"10.1542\/peds.2007-0282","article-title":"Scoring evaluation of the natural course of mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A)","volume":"120","author":"Meyer","year":"2007","journal-title":"Pediatrics"},{"key":"key\n\t\t\t\t20170503081521_DDU727C12","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1002\/ajmg.c.30142","article-title":"Is Sanfilippo Type B in your mind when you see adults with mental retardation and behavioral problems?","volume":"145C","author":"Moog","year":"2007","journal-title":"Am J Med Genet Part C Semin Med Genet"},{"key":"key\n\t\t\t\t20170503081521_DDU727C13","doi-asserted-by":"crossref","first-page":"6101","DOI":"10.1073\/pnas.93.12.6101","article-title":"The molecular basis of Sanfilippo syndrome type B","volume":"93","author":"Zhao","year":"1996","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"key\n\t\t\t\t20170503081521_DDU727C14","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","author":"Enns","year":"2008","journal-title":"Neurosurg. Focus"},{"key":"key\n\t\t\t\t20170503081521_DDU727C15","doi-asserted-by":"crossref","first-page":"14870","DOI":"10.1073\/pnas.1416660111","article-title":"Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB","volume":"111","author":"Kan","year":"2014","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"key\n\t\t\t\t20170503081521_DDU727C16","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1042\/BJ20130845","article-title":"Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of \u03b1-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts","volume":"458","author":"Kan","year":"2014","journal-title":"Biochem. J."},{"key":"key\n\t\t\t\t20170503081521_DDU727C17","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":"key\n\t\t\t\t20170503081521_DDU727C18","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","author":"Fu","year":"2011","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170503081521_DDU727C19","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 MPS IIIB: toxicology, bio-distribution and immunological assessments in primates","volume":"25","author":"Murrey","year":"2014","journal-title":"Hum. Gene Ther. Clin. Dev"},{"key":"key\n\t\t\t\t20170503081521_DDU727C20","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","author":"Souweidane","year":"2010","journal-title":"J. Neurosurg. Pediatr."},{"key":"key\n\t\t\t\t20170503081521_DDU727C21","doi-asserted-by":"crossref","first-page":"506","DOI":"10.1089\/hum.2013.238","article-title":"Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I\/II trial","volume":"25","author":"Tardieu","year":"2014","journal-title":"Hum. Gene Ther"},{"key":"key\n\t\t\t\t20170503081521_DDU727C22","doi-asserted-by":"crossref","first-page":"10229","DOI":"10.1523\/JNEUROSCI.3558-04.2004","article-title":"Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum","volume":"24","author":"Cressant","year":"2004","journal-title":"J. Neurosci."},{"key":"key\n\t\t\t\t20170503081521_DDU727C23","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1016\/j.ymthe.2006.03.014","article-title":"Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8","volume":"14","author":"Inagaki","year":"2006","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170503081521_DDU727C24","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":"key\n\t\t\t\t20170503081521_DDU727C25","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":"key\n\t\t\t\t20170503081521_DDU727C26","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","author":"Hinderer","year":"2014","journal-title":"Mol. Ther"},{"key":"key\n\t\t\t\t20170503081521_DDU727C27","doi-asserted-by":"crossref","first-page":"14505","DOI":"10.1073\/pnas.96.25.14505","article-title":"Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase","volume":"96","author":"Li","year":"1999","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"key\n\t\t\t\t20170503081521_DDU727C28","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. USA"},{"key":"key\n\t\t\t\t20170503081521_DDU727C29","doi-asserted-by":"crossref","first-page":"212","DOI":"10.1016\/j.bbr.2010.01.045","article-title":"Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB","volume":"209","author":"Canal","year":"2010","journal-title":"Behav. Brain Res."},{"key":"key\n\t\t\t\t20170503081521_DDU727C30","doi-asserted-by":"crossref","first-page":"612","DOI":"10.1002\/jnr.21134","article-title":"Oxidative stress in brain disease from mucopolysaccharidosis IIIB","volume":"85","author":"Villani","year":"2007","journal-title":"J. Neurosci. Res."},{"key":"key\n\t\t\t\t20170503081521_DDU727C31","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":"key\n\t\t\t\t20170503081521_DDU727C32","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1007\/s11011-010-9230-x","article-title":"Differential distribution of heparan sulfate glycoforms and elevated expression of heparan sulfate biosynthetic enzyme genes in the brain of mucopolysaccharidosis IIIB mice","volume":"26","author":"McCarty","year":"2011","journal-title":"Metab. Brain Dis."},{"key":"key\n\t\t\t\t20170503081521_DDU727C33","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":"key\n\t\t\t\t20170503081521_DDU727C34","doi-asserted-by":"crossref","first-page":"e45992","DOI":"10.1371\/journal.pone.0045992","article-title":"Peripheral nervous system neuropathology and progressive sensory impairments in a mouse model of Mucopolysaccharidosis IIIB","volume":"7","author":"Fu","year":"2012","journal-title":"PLoS One"},{"key":"key\n\t\t\t\t20170503081521_DDU727C35","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":"key\n\t\t\t\t20170503081521_DDU727C36","doi-asserted-by":"crossref","first-page":"573","DOI":"10.1006\/mgme.2000.3095","article-title":"Short-term enzyme replacement in the murine model of Sanfilippo syndrome type B","volume":"71","author":"Yu","year":"2000","journal-title":"Mol. Genet. Metab."},{"key":"key\n\t\t\t\t20170503081521_DDU727C37","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1016\/j.ymgme.2004.06.004","article-title":"Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB","volume":"82","author":"Zheng","year":"2004","journal-title":"Mol. Genet. Metab."},{"key":"key\n\t\t\t\t20170503081521_DDU727C38","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1042\/BJ20041702","article-title":"Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector","volume":"388","author":"Di Natale","year":"2005","journal-title":"Biochem. J."},{"key":"key\n\t\t\t\t20170503081521_DDU727C39","doi-asserted-by":"crossref","first-page":"1340","DOI":"10.1038\/gt.2009.85","article-title":"Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice","volume":"16","author":"McCarty","year":"2009","journal-title":"Gene Ther."},{"key":"key\n\t\t\t\t20170503081521_DDU727C40","doi-asserted-by":"crossref","first-page":"873","DOI":"10.1038\/mt.2010.17","article-title":"Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB","volume":"18","author":"Heldermon","year":"2010","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170503081521_DDU727C41","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","author":"Fu","year":"2010","journal-title":"J. Gene Med."},{"key":"key\n\t\t\t\t20170503081521_DDU727C42","doi-asserted-by":"crossref","first-page":"913","DOI":"10.1038\/gt.2013.14","article-title":"Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice","volume":"20","author":"Heldermon","year":"2013","journal-title":"Gene Ther"},{"key":"key\n\t\t\t\t20170503081521_DDU727C43","article-title":"Anxiety-related behaviors in mice","volume-title":"Methods of Behavior Analysis in Neuroscience","author":"Bailey","year":"2009","edition":"2nd edn"},{"key":"key\n\t\t\t\t20170503081521_DDU727C44","doi-asserted-by":"crossref","first-page":"1197","DOI":"10.1111\/j.1460-9568.2009.06666.x","article-title":"Examination of intravenous and intra-CSF protein delivery for treatment of neurological disease","volume":"29","author":"Hemsley","year":"2009","journal-title":"Eur. J. Neurosci."},{"key":"key\n\t\t\t\t20170503081521_DDU727C45","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":"key\n\t\t\t\t20170503081521_DDU727C46","doi-asserted-by":"crossref","first-page":"473","DOI":"10.1126\/science.1174447","article-title":"A gene network regulating lysosomal biogenesis and function","volume":"325","author":"Sardiello","year":"2009","journal-title":"Science"},{"key":"key\n\t\t\t\t20170503081521_DDU727C47","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":"key\n\t\t\t\t20170503081521_DDU727C48","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1111\/j.1750-3639.1994.tb00838.x","article-title":"GFAP and astrogliosis","volume":"4","author":"Eng","year":"1994","journal-title":"Brain Pathol."},{"key":"key\n\t\t\t\t20170503081521_DDU727C49","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1007\/BF01795308","article-title":"Lectin binding by resting and reactive microglia","volume":"16","author":"Streit","year":"1987","journal-title":"J. Neurocytol."},{"key":"key\n\t\t\t\t20170503081521_DDU727C50","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."},{"issue":"Suppl. 2","key":"key\n\t\t\t\t20170503081521_DDU727C51","first-page":"S1","article-title":"Glial cell reactions in neurodegenerative diseases: pathophysiology and therapeutic interventions","volume":"12","author":"McGeer","year":"1998","journal-title":"Alzheimer Dis. Assoc. Disord.,"},{"key":"key\n\t\t\t\t20170503081521_DDU727C52","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1038\/nature10907","article-title":"Wild-type microglia arrest pathology in a mouse model of Rett syndrome","volume":"484","author":"Derecki","year":"2012","journal-title":"Nature"},{"key":"key\n\t\t\t\t20170503081521_DDU727C53","doi-asserted-by":"crossref","first-page":"978","DOI":"10.1002\/jnr.21912","article-title":"Innate and adaptive immune activation in the brain of MPS IIIB mouse model","volume":"87","author":"DiRosario","year":"2009","journal-title":"J. Neurosci. Res."},{"key":"key\n\t\t\t\t20170503081521_DDU727C54","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","author":"Archer","year":"2014","journal-title":"J. Inherit. Metab. Dis."},{"key":"key\n\t\t\t\t20170503081521_DDU727C55","first-page":"2868","article-title":"Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I","volume":"118","author":"Dickson","year":"2008","journal-title":"J. Clin. Invest."},{"key":"key\n\t\t\t\t20170503081521_DDU727C56","doi-asserted-by":"crossref","first-page":"704","DOI":"10.1089\/hum.2009.182","article-title":"Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors","volume":"21","author":"Boutin","year":"2010","journal-title":"Hum. Gene Ther."},{"key":"key\n\t\t\t\t20170503081521_DDU727C57","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":"key\n\t\t\t\t20170503081521_DDU727C58","doi-asserted-by":"crossref","first-page":"1810","DOI":"10.1182\/blood-2005-08-3229","article-title":"Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice","volume":"107","author":"Scallan","year":"2006","journal-title":"Blood"},{"key":"key\n\t\t\t\t20170503081521_DDU727C59","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","author":"Jiang","year":"2006","journal-title":"Blood"},{"key":"key\n\t\t\t\t20170503081521_DDU727C60","doi-asserted-by":"crossref","first-page":"1566","DOI":"10.2174\/138161208784705504","article-title":"Lysosomal storage diseases and the blood-brain barrier","volume":"14","author":"Begley","year":"2008","journal-title":"Curr. Pharm. Des."},{"key":"key\n\t\t\t\t20170503081521_DDU727C61","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 Biotech"},{"key":"key\n\t\t\t\t20170503081521_DDU727C62","first-page":"e27209","article-title":"Toward a gene therapy for neurological and somatic MPSIIIA","volume":"1","author":"Haurigot","year":"2013","journal-title":"Rare Dis. (Austin, Tex.)"},{"key":"key\n\t\t\t\t20170503081521_DDU727C63","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":"key\n\t\t\t\t20170503081521_DDU727C64","doi-asserted-by":"crossref","first-page":"957","DOI":"10.1182\/blood-2011-09-377630","article-title":"AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage","volume":"119","author":"Binny","year":"2012","journal-title":"Blood"},{"key":"key\n\t\t\t\t20170503081521_DDU727C65","doi-asserted-by":"crossref","first-page":"385","DOI":"10.1023\/B:BOLI.0000031101.12838.c6","article-title":"Enzyme replacement and enhancement therapies for lysosomal diseases","volume":"27","author":"Desnick","year":"2004","journal-title":"J. Inherit. Metab. Dis."},{"key":"key\n\t\t\t\t20170503081521_DDU727C66","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":"key\n\t\t\t\t20170503081521_DDU727C67","doi-asserted-by":"crossref","first-page":"183","DOI":"10.1016\/j.ymgme.2007.03.003","article-title":"Preclinical dose ranging studies for enzyme replacement therapy with idursulfase in a knock-out mouse model of MPS II","volume":"91","author":"Garcia","year":"2007","journal-title":"Mol. Genet. Metab."},{"key":"key\n\t\t\t\t20170503081521_DDU727C68","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.1007\/s10545-011-9359-8","article-title":"Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management","volume":"34","author":"Braunlin","year":"2011","journal-title":"J. Inherit. Metab. Dis."},{"key":"key\n\t\t\t\t20170503081521_DDU727C69","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1002\/cne.20355","article-title":"Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders","volume":"480","author":"McGlynn","year":"2004","journal-title":"J. Comp. Neurol."},{"key":"key\n\t\t\t\t20170503081521_DDU727C70","doi-asserted-by":"crossref","first-page":"433","DOI":"10.1016\/j.semcdb.2004.03.002","article-title":"Secondary accumulation of gangliosides in lysosomal storage disorders","volume":"15","author":"Walkley","year":"2004","journal-title":"Semin. Cell Dev. Biol."},{"key":"key\n\t\t\t\t20170503081521_DDU727C71","doi-asserted-by":"crossref","first-page":"569","DOI":"10.1354\/vp.44-5-569","article-title":"Pathology of mucopolysaccharidosis IIIA in Huntaway dogs","volume":"44","author":"Jolly","year":"2007","journal-title":"Vet. Pathol."},{"key":"key\n\t\t\t\t20170503081521_DDU727C72","doi-asserted-by":"crossref","first-page":"1949","DOI":"10.1111\/ejn.12169","article-title":"Autophagy failure in Alzheimer's disease and the role of defective lysosomal acidification","volume":"37","author":"Wolfe","year":"2013","journal-title":"Eur. J. Neurosci."},{"key":"key\n\t\t\t\t20170503081521_DDU727C73","doi-asserted-by":"crossref","first-page":"725","DOI":"10.1002\/mds.25462","article-title":"Lysosomal impairment in Parkinson's disease","volume":"28","author":"Dehay","year":"2013","journal-title":"Mov. Disord."},{"key":"key\n\t\t\t\t20170503081521_DDU727C74","doi-asserted-by":"crossref","first-page":"e2296","DOI":"10.1371\/journal.pone.0002296","article-title":"Early neurodegeneration progresses independently of microglial activation by heparan sulfate in the brain of mucopolysaccharidosis IIIB mice","volume":"3","author":"Ausseil","year":"2008","journal-title":"PLoS One"},{"key":"key\n\t\t\t\t20170503081521_DDU727C75","first-page":"411","article-title":"Toxicology testing for products intended for pediatric populations","volume-title":"Non Clinical Drug Safety Assessment: Practical Considerations for Successful Registration","author":"Barrow","year":"2007"},{"key":"key\n\t\t\t\t20170503081521_DDU727C76","doi-asserted-by":"crossref","first-page":"1597","DOI":"10.1016\/S0140-6736(09)61836-5","article-title":"Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial","volume":"374","author":"Maguire","year":"2009","journal-title":"Lancet"},{"key":"key\n\t\t\t\t20170503081521_DDU727C77","first-page":"CD009354","article-title":"Enzyme replacement therapy with laronidase (Aldurazyme) for treating mucopolysaccharidosis type I","volume":"9","author":"Jameson","year":"2013","journal-title":"Cochrane Database Syst. Rev."},{"key":"key\n\t\t\t\t20170503081521_DDU727C78","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1590\/S1415-47572014000300003","article-title":"Guidelines for diagnosis and treatment of Hunter Syndrome for clinicians in Latin America","volume":"37","author":"Giugliani","year":"2014","journal-title":"Genet. Mol. Biol."},{"key":"key\n\t\t\t\t20170503081521_DDU727C79","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","author":"Calcedo","year":"2011","journal-title":"Clin. Vaccine Immunol."},{"key":"key\n\t\t\t\t20170503081521_DDU727C80","doi-asserted-by":"crossref","first-page":"903","DOI":"10.1089\/hum.2012.015","article-title":"Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice","volume":"23","author":"Piguet","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"key\n\t\t\t\t20170503081521_DDU727C81","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":"key\n\t\t\t\t20170503081521_DDU727C82","doi-asserted-by":"crossref","first-page":"423","DOI":"10.2174\/156652310793797685","article-title":"Production, purification and characterization of adeno-associated vectors","volume":"10","author":"Ayuso","year":"2010","journal-title":"Curr. Gene Ther."},{"key":"key\n\t\t\t\t20170503081521_DDU727C83","doi-asserted-by":"crossref","first-page":"1093","DOI":"10.1023\/A:1018943613122","article-title":"Physiological parameters in laboratory animals and humans","volume":"10","author":"Davies","year":"1993","journal-title":"Pharm. Res."},{"key":"key\n\t\t\t\t20170503081521_DDU727C84","doi-asserted-by":"crossref","first-page":"1203","DOI":"10.1016\/j.neuroscience.2005.07.014","article-title":"A three-dimensional digital atlas database of the adult C57BL\/6J mouse brain by magnetic resonance microscopy","volume":"135","author":"Ma","year":"2005","journal-title":"Neuroscience"},{"key":"key\n\t\t\t\t20170503081521_DDU727C85","doi-asserted-by":"crossref","first-page":"258","DOI":"10.1111\/j.1399-0004.1985.tb00217.x","article-title":"4-Methylumbelliferyl alpha-N-acetylglucosaminidase activity for diagnosis of Sanfilippo B disease","volume":"27","author":"Marsh","year":"1985","journal-title":"Clin. Genet."}],"container-title":["Human Molecular Genetics"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/academic.oup.com\/hmg\/article-pdf\/24\/7\/2078\/14147594\/ddu727.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,8,18]],"date-time":"2019-08-18T16:12:59Z","timestamp":1566144779000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/hmg\/article-lookup\/doi\/10.1093\/hmg\/ddu727"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2014,12,18]]},"references-count":85,"journal-issue":{"issue":"7","published-online":{"date-parts":[[2014,12,18]]},"published-print":{"date-parts":[[2015,4,1]]}},"URL":"https:\/\/doi.org\/10.1093\/hmg\/ddu727","relation":{},"ISSN":["1460-2083","0964-6906"],"issn-type":[{"value":"1460-2083","type":"electronic"},{"value":"0964-6906","type":"print"}],"subject":[],"published":{"date-parts":[[2014,12,18]]}}}