{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,15]],"date-time":"2026-01-15T23:53:40Z","timestamp":1768521220950,"version":"3.49.0"},"reference-count":90,"publisher":"Oxford University Press (OUP)","issue":"11","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2010,6,1]]},"DOI":"10.1093\/hmg\/ddq099","type":"journal-article","created":{"date-parts":[[2010,3,4]],"date-time":"2010-03-04T22:42:00Z","timestamp":1267742520000},"page":"2208-2227","source":"Crossref","is-referenced-by-count":69,"title":["Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models"],"prefix":"10.1093","volume":"19","author":[{"given":"Annalisa","family":"Lattanzi","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Margherita","family":"Neri","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Claudio","family":"Maderna","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ilaria","family":"di Girolamo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sabata","family":"Martino","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Aldo","family":"Orlacchio","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mario","family":"Amendola","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Luigi","family":"Naldini","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Angela","family":"Gritti","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"286","published-online":{"date-parts":[[2010,3,4]]},"reference":[{"key":"key\n\t\t\t\t20170522131539_DDQ099C1","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1111\/j.1365-2141.2004.05293.x","article-title":"Lysosomal storage disorders","volume":"128","author":"Vellodi","year":"2005","journal-title":"Br. J. Haematol"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C2","doi-asserted-by":"crossref","first-page":"302","DOI":"10.1073\/pnas.66.2.302","article-title":"Globoid cell leucodystrophy (Krabbe's disease): deficiency of galactocerebroside beta-galactosidase","volume":"66","author":"Suzuki","year":"1970","journal-title":"Proc. Natl Acad. Sci. USA"},{"issue":"Suppl. 1","key":"key\n\t\t\t\t20170522131539_DDQ099C3","first-page":"S87","article-title":"Genetics of metachromatic leukodystrophy","volume":"1","author":"Gieselmann","year":"1994","journal-title":"Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C4","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1111\/j.1651-2227.2008.00660.x","article-title":"CNS-directed gene therapy for lysosomal storage diseases","volume":"97","author":"Sands","year":"2008","journal-title":"Acta. Paediatr. Suppl."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C5","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1038\/sj.mt.6300004","article-title":"Complete correction of enzymatic deficiency and neurochemistry in the GM1-gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery","volume":"15","author":"Broekman","year":"2007","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C6","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1038\/gt.2008.149","article-title":"Mechanisms of distribution of mouse beta-galactosidase in the adult GM1-gangliosidosis brain","volume":"16","author":"Broekman","year":"2009","journal-title":"Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C7","doi-asserted-by":"crossref","first-page":"10373","DOI":"10.1073\/pnas.0603765103","article-title":"Effective gene therapy in an authentic model of Tay-Sachs-related diseases","volume":"103","author":"Cachon-Gonzalez","year":"2006","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C8","doi-asserted-by":"crossref","first-page":"2113","DOI":"10.1093\/hmg\/ddi216","article-title":"A direct gene transfer strategy via brain internal capsule reverses the biochemical defect in Tay-Sachs disease","volume":"14","author":"Martino","year":"2005","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C9","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1002\/ana.20392","article-title":"Effective gene therapy for an inherited CNS disease in a large animal model","volume":"57","author":"Vite","year":"2005","journal-title":"Ann. Neurol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C10","doi-asserted-by":"crossref","first-page":"9928","DOI":"10.1523\/JNEUROSCI.2185-07.2007","article-title":"A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease","volume":"27","author":"Cearley","year":"2007","journal-title":"J. Neurosci."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C11","doi-asserted-by":"crossref","first-page":"242","DOI":"10.1038\/sj.mt.6300016","article-title":"Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS","volume":"15","author":"Liu","year":"2007","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C12","doi-asserted-by":"crossref","first-page":"1209","DOI":"10.1002\/ajmg.a.32861","article-title":"Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB","volume":"149A","author":"Di Domenico","year":"2009","journal-title":"Am. J. Med. Genet."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C13","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\t20170522131539_DDQ099C14","doi-asserted-by":"crossref","first-page":"1782","DOI":"10.1038\/sj.mt.6300249","article-title":"Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease","volume":"15","author":"Cabrera-Salazar","year":"2007","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C15","doi-asserted-by":"crossref","first-page":"754","DOI":"10.1016\/j.ymthe.2005.01.011","article-title":"AAV vector-mediated correction of brain pathology in a mouse model of Niemann\u2013Pick A disease","volume":"11","author":"Passini","year":"2005","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C16","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1093\/hmg\/ddi425","article-title":"Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy","volume":"15","author":"Sevin","year":"2006","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C17","doi-asserted-by":"crossref","first-page":"310","DOI":"10.1038\/85454","article-title":"In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice","volume":"7","author":"Consiglio","year":"2001","journal-title":"Nat. Med."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C18","doi-asserted-by":"crossref","first-page":"734","DOI":"10.1016\/j.ymthe.2004.12.020","article-title":"AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy","volume":"11","author":"Rafi","year":"2005","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C19","doi-asserted-by":"crossref","first-page":"422","DOI":"10.1016\/j.ymthe.2005.04.019","article-title":"AAV2\/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2","volume":"12","author":"Lin","year":"2005","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C20","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1093\/hmg\/ddp475","article-title":"Efficient intracerebral delivery of Aav5 vector encoding human Arsa in non-human primate","volume":"19","author":"Colle","year":"2009","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C21","doi-asserted-by":"crossref","first-page":"333","DOI":"10.2174\/1566523054064995","article-title":"Adeno-associated virus (AAV) vectors in the CNS","volume":"5","author":"McCown","year":"2005","journal-title":"Curr. Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C22","first-page":"225","article-title":"Gene delivery to the mouse brain with adeno-associated virus","volume":"246","author":"Passini","year":"2004","journal-title":"Methods Mol. Biol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C23","first-page":"191","article-title":"Lentiviruses as vectors for CNS diseases","volume":"261","author":"Deglon","year":"2002","journal-title":"Curr. Top. Microbiol. Immunol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C24","doi-asserted-by":"crossref","first-page":"461","DOI":"10.2174\/156652308786847996","article-title":"Applications of lentiviral vectors for biology and gene therapy of neurological disorders","volume":"8","author":"Lundberg","year":"2008","journal-title":"Curr. Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C25","doi-asserted-by":"crossref","first-page":"741","DOI":"10.1089\/hum.2005.16.741","article-title":"Stability of lentiviral vector-mediated transgene expression in the brain in the presence of systemic antivector immune responses","volume":"16","author":"Abordo-Adesida","year":"2005","journal-title":"Hum. Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C26","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1089\/hum.2008.151","article-title":"Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response","volume":"20","author":"Hadaczek","year":"2009","journal-title":"Hum. Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C27","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1089\/hum.2006.17.1","article-title":"Lentivirus-mediated gene transfer to the central nervous system: therapeutic and research applications","volume":"17","author":"Wong","year":"2006","journal-title":"Hum. Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C28","doi-asserted-by":"crossref","first-page":"474","DOI":"10.2174\/156652308786848049","article-title":"Clinical application of lentiviral vectors - concepts and practice","volume":"8","author":"Schambach","year":"2008","journal-title":"Curr. Gene Ther"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C29","doi-asserted-by":"crossref","first-page":"964","DOI":"10.1172\/JCI37630","article-title":"The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy","volume":"119","author":"Montini","year":"2009","journal-title":"J. Clin. Invest."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C30","doi-asserted-by":"crossref","first-page":"6437","DOI":"10.1523\/JNEUROSCI.22-15-06437.2002","article-title":"Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream","volume":"22","author":"Passini","year":"2002","journal-title":"J. Neurosci."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C31","doi-asserted-by":"crossref","first-page":"1118","DOI":"10.1172\/JCI200419205","article-title":"Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells","volume":"113","author":"Biffi","year":"2004","journal-title":"J. Clin. Invest."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C32","doi-asserted-by":"crossref","first-page":"669","DOI":"10.1016\/j.ymthe.2005.06.438","article-title":"Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery","volume":"12","author":"Luca","year":"2005","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C33","doi-asserted-by":"crossref","first-page":"1874","DOI":"10.1038\/sj.gt.3302087","article-title":"Adeno-associated virus vector-mediated transduction in the cat brain","volume":"10","author":"Vite","year":"2003","journal-title":"Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C34","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","author":"Ciron","year":"2009","journal-title":"Hum. Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C35","doi-asserted-by":"crossref","first-page":"463","DOI":"10.1089\/hum.2008.022","article-title":"Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA","volume":"19","author":"Worgall","year":"2008","journal-title":"Hum. Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C36","doi-asserted-by":"crossref","first-page":"108","DOI":"10.1038\/nbt1049","article-title":"Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters","volume":"23","author":"Amendola","year":"2005","journal-title":"Nat. Biotechnol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C37","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1093\/brain\/103.3.695","article-title":"Hereditary leucodystrophy in the mouse: the new mutant twitcher","volume":"103","author":"Duchen","year":"1980","journal-title":"Brain"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C38","first-page":"394","article-title":"The twitcher mouse. A model of human globoid cell leukodystrophy (Krabbe's disease)","volume":"111","author":"Suzuki","year":"1983","journal-title":"Am. J. Pathol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C39","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1002\/cne.22153","article-title":"Effects of developmental age, brain region, and time in culture on long-term proliferation and multipotency of neural stem cell populations","volume":"517","author":"Gritti","year":"2009","journal-title":"J. Comp. Neurol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C40","doi-asserted-by":"crossref","first-page":"273","DOI":"10.1016\/j.nbd.2006.03.005","article-title":"Suppression of galactosylceramidase (GALC) expression in the twitcher mouse model of globoid cell leukodystrophy (GLD) is caused by nonsense-mediated mRNA decay (NMD)","volume":"23","author":"Lee","year":"2006","journal-title":"Neurobiol. Dis."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C41","doi-asserted-by":"crossref","first-page":"2239","DOI":"10.1093\/brain\/awp174","article-title":"Delayed post-ischaemic neuroprotection following systemic neural stem cell transplantation involves multiple mechanisms","volume":"132","author":"Bacigaluppi","year":"2009","journal-title":"Brain"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C42","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":"key\n\t\t\t\t20170522131539_DDQ099C43","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1373\/clinchem.2008.115873","article-title":"Specific determination of beta-galactocerebrosidase activity via competitive inhibition of beta-galactosidase","volume":"55","author":"Martino","year":"2009","journal-title":"Clin. Chem."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C44","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1016\/S0009-8981(98)00095-3","article-title":"Expression and processing of recombinant human galactosylceramidase","volume":"276","author":"Nagano","year":"1998","journal-title":"Clin. Chim. Acta."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C45","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1042\/bj1890009","article-title":"The interrelations between high- and low-molecular weight forms of normal and mutant (Krabbe-disease) galactocerebrosidase","volume":"189","author":"Ben-Yoseph","year":"1980","journal-title":"Biochem. J."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C46","doi-asserted-by":"crossref","first-page":"14821","DOI":"10.1073\/pnas.93.25.14821","article-title":"Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy","volume":"93","author":"Hess","year":"1996","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C47","doi-asserted-by":"crossref","first-page":"1081","DOI":"10.1038\/sj.gt.3301495","article-title":"Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements","volume":"8","author":"Shen","year":"2001","journal-title":"Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C48","doi-asserted-by":"crossref","first-page":"9321","DOI":"10.1523\/JNEUROSCI.2936-05.2005","article-title":"Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors","volume":"25","author":"Liu","year":"2005","journal-title":"J. Neurosci."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C49","doi-asserted-by":"crossref","first-page":"649","DOI":"10.1038\/mt.2008.9","article-title":"Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis","volume":"16","author":"Chang","year":"2008","journal-title":"Mol. Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C50","doi-asserted-by":"crossref","first-page":"917","DOI":"10.1038\/sj.gt.3302735","article-title":"Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice","volume":"13","author":"Watson","year":"2006","journal-title":"Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C51","doi-asserted-by":"crossref","first-page":"7034","DOI":"10.1128\/JVI.77.12.7034-7040.2003","article-title":"Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-deficient mice","volume":"77","author":"Passini","year":"2003","journal-title":"J. Virol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C52","doi-asserted-by":"crossref","first-page":"411","DOI":"10.1023\/B:BOLI.0000031170.69676.68","article-title":"Treatment of lysosomal storage disorders: cell therapy and gene therapy","volume":"27","author":"Eto","year":"2004","journal-title":"J. Inherit. Metab. Dis."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C53","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1007\/BF00687958","article-title":"Histochemical similarities between human and animal globoid cells in Krabbe's disease: a lectin study","volume":"71","author":"Alroy","year":"1986","journal-title":"Acta Neuropathol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C54","doi-asserted-by":"crossref","first-page":"501","DOI":"10.1177\/34.4.3081625","article-title":"Lectin histochemistry of glycolipid storage diseases on frozen and paraffin-embedded tissue sections","volume":"34","author":"Alroy","year":"1986","journal-title":"J. Histochem. Cytochem."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C55","doi-asserted-by":"crossref","first-page":"962","DOI":"10.1002\/jgm.924","article-title":"Design and optimization of lentiviral vectors for transfer of GALC expression in twitcher brain","volume":"8","author":"Dolcetta","year":"2006","journal-title":"J. Gene Med."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C56","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1016\/j.nbd.2008.11.016","article-title":"Astrocyte-secreted GDNF and glutathione antioxidant system protect neurons against 6OHDA cytotoxicity","volume":"33","author":"Sandhu","year":"2009","journal-title":"Neurobiol. Dis."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C57","doi-asserted-by":"crossref","first-page":"e6227","DOI":"10.1371\/journal.pone.0006227","article-title":"A novel and efficient gene transfer strategy reduces glial reactivity and improves neuronal survival and axonal growth in vitro","volume":"4","author":"Desclaux","year":"2009","journal-title":"PLoS One"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C58","doi-asserted-by":"crossref","first-page":"6641","DOI":"10.1128\/JVI.71.9.6641-6649.1997","article-title":"Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector","volume":"71","author":"Blomer","year":"1997","journal-title":"J. Virol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C59","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1016\/S0304-3940(03)00833-4","article-title":"Transduction of neurons lining the cerebral external capsules in mice with feline immunodeficiency virus based vectors","volume":"351","author":"Alisky","year":"2003","journal-title":"Neurosci. Lett."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C60","doi-asserted-by":"crossref","first-page":"767","DOI":"10.1126\/science.290.5492.767","article-title":"Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease","volume":"290","author":"Kordower","year":"2000","journal-title":"Science"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C61","doi-asserted-by":"crossref","first-page":"6216","DOI":"10.1073\/pnas.082011999","article-title":"Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors","volume":"99","author":"Brooks","year":"2002","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C62","doi-asserted-by":"crossref","first-page":"216","DOI":"10.1159\/000091919","article-title":"Cerebellar neurons and glial cells are transducible by lentiviral vectors without decrease of cerebellar functions","volume":"28","author":"Croci","year":"2006","journal-title":"Dev. Neurosci."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C63","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1002\/jnr.20626","article-title":"Lentiviral transduction of murine oligodendrocytes in vivo","volume":"82","author":"McIver","year":"2005","journal-title":"J. Neurosci. Res."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C64","doi-asserted-by":"crossref","first-page":"14835","DOI":"10.1073\/pnas.0404180101","article-title":"Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors","volume":"101","author":"Consiglio","year":"2004","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C65","doi-asserted-by":"crossref","first-page":"1139","DOI":"10.1089\/10430340050015194","article-title":"Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer","volume":"11","author":"Bosch","year":"2000","journal-title":"Hum. Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C66","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1097\/00062752-199911000-00004","article-title":"Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome","volume":"6","author":"Krivit","year":"1999","journal-title":"Curr. Opin. Hematol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C67","doi-asserted-by":"crossref","first-page":"1017","DOI":"10.3174\/ajnr.A1476","article-title":"Diffusion tensor imaging detects abnormalities in the corticospinal tracts of neonates with infantile Krabbe disease","volume":"30","author":"Escolar","year":"2009","journal-title":"AJNR Am. J. Neuroradiol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C68","doi-asserted-by":"crossref","first-page":"1636","DOI":"10.1210\/endo.131.4.1396308","article-title":"Expression of insulin-like growth factor-II (IGF-II) and IGF-II\/mannose-6-phosphate receptor in the rat hippocampus: an in situ hybridization and immunocytochemical study","volume":"131","author":"Couce","year":"1992","journal-title":"Endocrinology"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C69","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1002\/cne.10578","article-title":"Insulin-like growth factor-II\/mannose-6-phosphate receptor: widespread distribution in neurons of the central nervous system including those expressing cholinergic phenotype","volume":"458","author":"Hawkes","year":"2003","journal-title":"J. Comp. Neurol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C70","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1016\/j.brainresrev.2003.11.002","article-title":"The insulin-like growth factor-II\/mannose-6-phosphate receptor: structure, distribution and function in the central nervous system","volume":"44","author":"Hawkes","year":"2004","journal-title":"Brain Res. Brain Res. Rev."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C71","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1146\/annurev.bi.60.070191.001353","article-title":"Lysosomal storage diseases","volume":"60","author":"Neufeld","year":"1991","journal-title":"Annu. Rev. Biochem."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C72","doi-asserted-by":"crossref","first-page":"2520","DOI":"10.1096\/fj.06-6169com","article-title":"Single-dose intracerebroventricular administration of galactocerebrosidase improves survival in a mouse model of globoid cell leukodystrophy","volume":"21","author":"Lee","year":"2007","journal-title":"FASEB J."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C73","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1016\/0005-2760(93)90175-9","article-title":"Galactocerebrosidase from human urine: purification and partial characterization","volume":"1170","author":"Chen","year":"1993","journal-title":"Biochim. Biophys. Acta."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C74","doi-asserted-by":"crossref","first-page":"305","DOI":"10.1042\/BJ20061783","article-title":"SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies","volume":"403","author":"Fraldi","year":"2007","journal-title":"Biochem. J."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C75","doi-asserted-by":"crossref","first-page":"713","DOI":"10.1038\/nrn1725","article-title":"Storage solutions: treating lysosomal disorders of the brain","volume":"6","author":"Jeyakumar","year":"2005","journal-title":"Nat. Rev. Neurosci."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C76","doi-asserted-by":"crossref","first-page":"2362","DOI":"10.1002\/stem.163","article-title":"Neural stem cell transplantation benefits a monogenic neurometabolic disorder during the symptomatic phase of disease","volume":"27","author":"Jeyakumar","year":"2009","journal-title":"Stem Cells"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C77","doi-asserted-by":"crossref","first-page":"577","DOI":"10.1002\/jgm.885","article-title":"Immune responses to AAV in a phase I study for Canavan disease","volume":"8","author":"McPhee","year":"2006","journal-title":"J. Gene Med."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C78","doi-asserted-by":"crossref","first-page":"347","DOI":"10.2174\/156652307782151498","article-title":"Immune responses to adenovirus and adeno-associated vectors used for gene therapy of brain diseases: the role of immunological synapses in understanding the cell biology of neuroimmune interactions","volume":"7","author":"Lowenstein","year":"2007","journal-title":"Curr. Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C79","doi-asserted-by":"crossref","first-page":"526","DOI":"10.1038\/sj.ejhg.5200348","article-title":"Neuroanatomy of the fragile X knockout mouse brain studied using in vivo high resolution magnetic resonance imaging","volume":"7","author":"Kooy","year":"1999","journal-title":"Eur. J. Hum. Genet."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C80","doi-asserted-by":"crossref","first-page":"715","DOI":"10.1016\/j.nurt.2007.07.010","article-title":"Immunology of neurological gene therapy: how T cells modulate viral vector-mediated therapeutic transgene expression through immunological synapses","volume":"4","author":"Lowenstein","year":"2007","journal-title":"Neurotherapeutics"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C81","doi-asserted-by":"crossref","first-page":"2069","DOI":"10.1056\/NEJMoa042604","article-title":"Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease","volume":"352","author":"Escolar","year":"2005","journal-title":"N. Engl. J. Med."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C82","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1089\/hum.2006.17.291","article-title":"Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain","volume":"17","author":"Hadaczek","year":"2006","journal-title":"Hum. Gene Ther."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C83","doi-asserted-by":"crossref","first-page":"2407","DOI":"10.1073\/pnas.0810682106","article-title":"Efficient gene therapy-based method for the delivery of therapeutics to primate cortex","volume":"106","author":"Kells","year":"2009","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C84","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1016\/j.jviromet.2004.08.017","article-title":"Optimized large-scale production of high titer lentivirus vector pseudotypes","volume":"122","author":"Sena-Esteves","year":"2004","journal-title":"J. Virol. Methods"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C85","doi-asserted-by":"crossref","first-page":"308","DOI":"10.1196\/annals.1345.007","article-title":"A phase I\/II clinical trial of beta-globin gene therapy for beta-thalassemia","volume":"1054","author":"Bank","year":"2005","journal-title":"Ann. N. Y. Acad. Sci."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C86","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1126\/science.1171242","article-title":"Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy","volume":"326","author":"Cartier","year":"2009","journal-title":"Science"},{"key":"key\n\t\t\t\t20170522131539_DDQ099C87","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1111\/j.1750-3639.1995.tb00601.x","article-title":"The twitcher mouse: a model for Krabbe disease and for experimental therapies","volume":"5","author":"Suzuki","year":"1995","journal-title":"Brain Pathol."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C88","doi-asserted-by":"crossref","first-page":"1118","DOI":"10.1046\/j.1471-4159.1996.66031118.x","article-title":"Molecular cloning and expression of cDNA for murine galactocerebrosidase and mutation analysis of the twitcher mouse, a model of Krabbe's disease","volume":"66","author":"Sakai","year":"1996","journal-title":"J. Neurochem."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C89","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1111\/j.1471-4159.2009.05919.x","article-title":"Neural precursor cell cultures from GM2-gangliosidosis animal models recapitulate the biochemical and molecular hallmarks of the brain pathology","volume":"109","author":"Martino","year":"2009","journal-title":"J. Neurochem."},{"key":"key\n\t\t\t\t20170522131539_DDQ099C90","doi-asserted-by":"crossref","first-page":"243","DOI":"10.1016\/j.jbiotec.2005.01.018","article-title":"Expression and purification of a human, soluble arylsulfatase A for metachromatic leukodystrophy enzyme replacement therapy","volume":"117","author":"Martino","year":"2005","journal-title":"J. Biotechnol."}],"container-title":["Human Molecular Genetics"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/academic.oup.com\/hmg\/article-pdf\/19\/11\/2208\/13905586\/ddq099.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,5,28]],"date-time":"2020-05-28T18:46:31Z","timestamp":1590691591000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/hmg\/article-lookup\/doi\/10.1093\/hmg\/ddq099"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2010,3,4]]},"references-count":90,"journal-issue":{"issue":"11","published-online":{"date-parts":[[2010,3,4]]},"published-print":{"date-parts":[[2010,6,1]]}},"URL":"https:\/\/doi.org\/10.1093\/hmg\/ddq099","relation":{},"ISSN":["1460-2083","0964-6906"],"issn-type":[{"value":"1460-2083","type":"electronic"},{"value":"0964-6906","type":"print"}],"subject":[],"published":{"date-parts":[[2010,3,4]]}}}