{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,4]],"date-time":"2026-10-04T07:27:15Z","timestamp":1791098835874,"version":"4.1.0"},"reference-count":124,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2012,1,1]],"date-time":"2012-01-01T00:00:00Z","timestamp":1325376000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2012,1]]},"DOI":"10.1016\/j.ymgme.2011.10.002","type":"journal-article","created":{"date-parts":[[2011,10,20]],"date-time":"2011-10-20T03:32:59Z","timestamp":1319081579000},"page":"56-63","source":"Crossref","is-referenced-by-count":42,"title":["Developing treatment options for metachromatic leukodystrophy"],"prefix":"10.1016","volume":"105","author":[{"given":"Spyros P.","family":"Batzios","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dimitrios I.","family":"Zafeiriou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymgme.2011.10.002_bb0005","doi-asserted-by":"crossref","first-page":"1028","DOI":"10.1002\/biot.200800322","article-title":"Molecular facets of sphingolipids: mediators of diseases","volume":"4","author":"Ozbayraktar","year":"2009","journal-title":"Biotechnol. J."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0010","doi-asserted-by":"crossref","first-page":"372","DOI":"10.1002\/glia.20292","article-title":"Sulfatide is essential for the maintenance of CNS myelin and axon structure","volume":"53","author":"Marcus","year":"2006","journal-title":"Glia"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0015","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1055\/s-0030-1253412","article-title":"Metachromatic leukodystrophy \u2014 an update","volume":"41","author":"Gieselmann","year":"2010","journal-title":"Neuropediatrics"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0020","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1111\/j.1651-2227.2008.00648.x","article-title":"Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options","volume":"97","author":"Gieselmann","year":"2008","journal-title":"Acta Paediatr. Suppl."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0025","doi-asserted-by":"crossref","first-page":"2","DOI":"10.1038\/bmt.2008.275","article-title":"Metachromatic leukodystrophy: an overview of current and prospective treatments","volume":"42","author":"Biffi","year":"2008","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0030","doi-asserted-by":"crossref","first-page":"627","DOI":"10.1111\/j.1432-1033.1990.tb19167.x","article-title":"Structure of the arylsulfatase A gene","volume":"191","author":"Kreysing","year":"1990","journal-title":"Eur. J. Biochem."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0035","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. U. S. A."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0040","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.2011.10.002_bb0045","doi-asserted-by":"crossref","first-page":"1775","DOI":"10.1126\/science.290.5497.1775","article-title":"From marrow to brain: expression of neuronal phenotypes in adult mice","volume":"290","author":"Brazelton","year":"2000","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0050","doi-asserted-by":"crossref","first-page":"1779","DOI":"10.1126\/science.290.5497.1779","article-title":"Turning blood into brain: cells bearing neuronal antigens generated in vivo from bone marrow","volume":"290","author":"Mezey","year":"2000","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0055","doi-asserted-by":"crossref","first-page":"1364","DOI":"10.1073\/pnas.0336479100","article-title":"Transplanted bone marrow generates new neurons in human brains","volume":"100","author":"Mezey","year":"2003","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0060","doi-asserted-by":"crossref","first-page":"2088","DOI":"10.1073\/pnas.0337659100","article-title":"Contribution of transplanted bone marrow cells to Purkinje cells in human adult brains","volume":"100","author":"Weimann","year":"2003","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0065","doi-asserted-by":"crossref","first-page":"3557","DOI":"10.1073\/pnas.0306431101","article-title":"Human CD34+ cells differentiate into microglia and express recombinant therapeutic protein","volume":"101","author":"Asheuer","year":"2004","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0070","doi-asserted-by":"crossref","first-page":"1263","DOI":"10.1126\/science.172.3989.1263","article-title":"Correction of abnormal cerebroside sulfate metabolism in cultured metachromatic leukodystrophy fibroblasts","volume":"172","author":"Porter","year":"1971","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0075","doi-asserted-by":"crossref","first-page":"471","DOI":"10.1016\/S0140-6736(85)90402-7","article-title":"Bone-marrow transplantation for metachromatic leucodystrophy","volume":"2","author":"Bayever","year":"1985","journal-title":"Lancet"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0080","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.2011.10.002_bb0085","doi-asserted-by":"crossref","first-page":"119","DOI":"10.1007\/s00281-004-0166-2","article-title":"Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases","volume":"26","author":"Krivit","year":"2004","journal-title":"Springer Semin. Immunopathol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0090","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1001\/archneur.55.1.98","article-title":"Long-term stabilization after bone marrow transplantation in juvenile metachromatic leukodystrophy","volume":"55","author":"Kidd","year":"1998","journal-title":"Arch. Neurol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0095","doi-asserted-by":"crossref","first-page":"1139","DOI":"10.1177\/0883073807306256","article-title":"Stabilization of juvenile metachromatic leukodystrophy after bone marrow transplantation: a 13-year follow-up","volume":"22","author":"G\u00f6rg","year":"2007","journal-title":"J. Child Neurol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0100","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1212\/WNL.46.1.254","article-title":"Late juvenile metachromatic leukodystrophy treated with bone marrow transplantation; a 4-year follow-up study","volume":"46","author":"Navarro","year":"1996","journal-title":"Neurology"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0105","first-page":"1003","article-title":"Clinical outcome in four children with metachromatic leukodystrophy treated by bone marrow transplantation","volume":"17","author":"Malm","year":"1996","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0110","doi-asserted-by":"crossref","first-page":"1119","DOI":"10.1038\/sj.bmt.1701485","article-title":"Improved peripheral nerve conduction, EEG and verbal IQ after bone marrow transplantation for adult metachromatic leukodystrophy","volume":"22","author":"Solders","year":"1998","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0115","doi-asserted-by":"crossref","first-page":"718","DOI":"10.1097\/01.tp.0000181457.43146.36","article-title":"Allogeneic hematopoietic stem cell transplantation for inherited disorders: experience in a single center","volume":"81","author":"Ringd\u00e9n","year":"2006","journal-title":"Transplantation"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0120","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.2011.10.002_bb0125","doi-asserted-by":"crossref","DOI":"10.1007\/s10545-010-9240-1","article-title":"Haematopoietic stem cell transplantation does not retard disease progression in the psycho-cognitive variant of late-onset metachromatic leukodystrophy","author":"Smith","year":"2010","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0130","doi-asserted-by":"crossref","first-page":"222","DOI":"10.1177\/088307389901400402","article-title":"Slow progression of juvenile metachromatic leukodystrophy 6years after bone marrow transplantation","volume":"14","author":"Kapaun","year":"1999","journal-title":"J. Child Neurol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0135","doi-asserted-by":"crossref","first-page":"755","DOI":"10.1016\/S0022-3476(06)80177-0","article-title":"Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation","volume":"125","author":"Pridjian","year":"1994","journal-title":"J. Pediatr."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0140","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1007\/BF00710053","article-title":"Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation","volume":"18","author":"Shapiro","year":"1995","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0145","doi-asserted-by":"crossref","first-page":"398","DOI":"10.1007\/BF00710052","article-title":"The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome","volume":"18","author":"Krivit","year":"1995","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0150","doi-asserted-by":"crossref","first-page":"80","DOI":"10.1097\/00000441-198708000-00004","article-title":"Prevention of deterioration in metachromatic leukodystrophy by bone marrow transplantation","volume":"294","author":"Krivit","year":"1987","journal-title":"Am. J. Med. Sci."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0155","doi-asserted-by":"crossref","first-page":"28","DOI":"10.1056\/NEJM199001043220106","article-title":"Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation","volume":"322","author":"Krivit","year":"1990","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0160","doi-asserted-by":"crossref","first-page":"404","DOI":"10.1111\/j.1442-200X.1993.tb03081.x","article-title":"Bone marrow transplantation for late infantile metachromatic leukodystrophy: pathogenic investigation for graft rejection","volume":"35","author":"Nagai","year":"1993","journal-title":"Acta Paediatr. Jpn."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0165","doi-asserted-by":"crossref","first-page":"309","DOI":"10.1038\/sj.bmt.1705581","article-title":"Early marrow transplantation in a pre-symptomatic neonate with late infantile metachromatic leukodystrophy does not halt disease progression","volume":"39","author":"Bredius","year":"2007","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0170","doi-asserted-by":"crossref","first-page":"857","DOI":"10.1038\/sj.bmt.1704024","article-title":"Allogeneic bone marrow stem cell transplantation following CD34+ immunomagnetic enrichment in patients with inherited metabolic storage diseases","volume":"31","author":"Gaipa","year":"2003","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0175","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1089\/15258160260194767","article-title":"Fetal hematopoietic stem cell transplantation: a challenge for the twenty-first century","volume":"11","author":"Shields","year":"2002","journal-title":"J. Hematother. Stem Cell Res."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0180","first-page":"189","article-title":"Clinical application of intrauterine bone marrow transplantation for treatment of genetic diseases \u2014 feasibility studies","volume":"9","author":"Slavin","year":"1992","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0200","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1016\/j.earlhumdev.2009.08.005","article-title":"Stem cells and the frontiers of neonatology","volume":"85","author":"Lanfranchi","year":"2009","journal-title":"Early Hum. Dev."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0205","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1056\/NEJM199607183350303","article-title":"Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients","volume":"335","author":"Kurtzberg","year":"1996","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0210","doi-asserted-by":"crossref","first-page":"795","DOI":"10.1182\/blood.V88.3.795.795","article-title":"Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: analysis of engraftment and acute graft-versus-host disease","volume":"88","author":"Wagner","year":"1996","journal-title":"Blood"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0215","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1097\/00062752-199704060-00009","article-title":"Cord blood stem cells","volume":"n4","author":"Wagner","year":"1997","journal-title":"Curr. Opin. Hematol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0220","doi-asserted-by":"crossref","first-page":"184","DOI":"10.1016\/j.bbmt.2005.09.016","article-title":"Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases","volume":"12","author":"Martin","year":"2006","journal-title":"Biol. Blood Marrow Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0225","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1007\/s10545-005-0258-8","article-title":"Trends in haematopoietic cell transplantation for inborn errors of metabolism","volume":"29","author":"Boelens","year":"2006","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0230","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1016\/S1246-7820(01)00132-X","article-title":"Results of unrelated umbilical cord blood hematopoietic stem cell transplant","volume":"8","author":"Gluckman","year":"2001","journal-title":"Transfus. Clin. Biol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0235","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1038\/sj.bmt.1702185","article-title":"Measurements from normal umbilical cord blood of four lysosomal enzymatic activities: alpha-l-iduronidase (Hurler), galactocerebrosidase (globoid cell leukodystrophy), arylsulfatase A (metachromatic leukodystrophy), arylsulfatase B (Maroteaux\u2013Lamy)","volume":"25","author":"deGasperi","year":"2000","journal-title":"Bone Marrow Transplant"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0240","doi-asserted-by":"crossref","first-page":"487","DOI":"10.1038\/sj.bmt.1703405","article-title":"Arylsulfatase-A in umbilical cord blood: gestational age and mode of delivery do not influence enzyme activity","volume":"29","author":"Georgeson","year":"2002","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0245","doi-asserted-by":"crossref","first-page":"583","DOI":"10.1002\/ana.21522","article-title":"Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy","volume":"64","author":"Pierson","year":"2008","journal-title":"Ann. Neurol."},{"issue":"Suppl. S","key":"10.1016\/j.ymgme.2011.10.002_bb0250","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1016\/j.bbmt.2006.12.172","article-title":"Neurodevelopmental outcomes of unrelated umbilical cord transplantation for metachromatic leukodystrophy","volume":"13","author":"Martin","year":"2007","journal-title":"Biol. Blood Marrow Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0255","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1016\/j.ymgme.2010.10.002","article-title":"Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: a 5-year follow-up in three affected siblings","volume":"102","author":"Cable","year":"2011","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0260","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":"10.1016\/j.ymgme.2011.10.002_bb0265","doi-asserted-by":"crossref","first-page":"672","DOI":"10.1111\/j.1399-3046.2007.00876.x","article-title":"Umbilical cord-blood transplantations from unrelated donors in patients with inherited metabolic diseases: single-institute experience","volume":"12","author":"Tokimasa","year":"2008","journal-title":"Pediatr. Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0275","doi-asserted-by":"crossref","first-page":"3722","DOI":"10.1182\/blood-2002-07-2104","article-title":"Bone marrow mesenchymal stem cells inhibit the response of naive and memory antigen-specific T cells to their cognate peptide","volume":"101","author":"Krampera","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0280","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1002\/jcp.20959","article-title":"From the laboratory bench to the patient's bedside: an update on clinical trials with mesenchymal stem cells","volume":"211","author":"Giordano","year":"2007","journal-title":"J. Cell. Physiol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0285","doi-asserted-by":"crossref","first-page":"645","DOI":"10.1002\/jcb.20315","article-title":"Molecular pathways involved in neural in vitro differentiation of marrow stromal stem cells","volume":"94","author":"Jori","year":"2005","journal-title":"J. Cell. Biochem."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0290","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1007\/3-540-31265-X_11","article-title":"Mesenchymal stem cells: isolation, in vitro expansion and characterization","volume":"174","author":"Beyer Nardi","year":"2006","journal-title":"Handb. Exp. Pharmacol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0295","doi-asserted-by":"crossref","first-page":"10711","DOI":"10.1073\/pnas.96.19.10711","article-title":"Marrow stromal cells migrate throughout forebrain and cerebellum, and they differentiate into astrocytes after injection into neonatal mouse brains","volume":"96","author":"Kopen","year":"1999","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0300","doi-asserted-by":"crossref","first-page":"2868","DOI":"10.1634\/stemcells.2005-0636","article-title":"Functional neuronal differentiation of bone marrow-derived mesenchymal stem cells","volume":"24","author":"Tropel","year":"2006","journal-title":"Stem Cells"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0305","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1111\/j.1432-0436.2004.07207003.x","article-title":"Bone marrow-derived mesenchymal stem cells already express specific neural proteins before any differentiation","volume":"72","author":"Tondreau","year":"2004","journal-title":"Differentiation"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0310","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1126\/science.284.5411.143","article-title":"Multilineage potential of adult human mesenchymal stem cells","volume":"284","author":"Pittenger","year":"1999","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0315","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1016\/j.bcmd.2003.09.018","article-title":"A large animal noninjury model for study of human stem cell plasticity","volume":"32","author":"Almeida-Porada","year":"2004","journal-title":"Blood Cells Mol. Dis."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0320","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1080\/14653240310000038","article-title":"Quantifying levels of transplanted murine and human mesenchymal stem cells in vivo by real-time PCR","volume":"5","author":"McBride","year":"2003","journal-title":"Cytotherapy"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0325","doi-asserted-by":"crossref","first-page":"831","DOI":"10.1016\/S0301-472X(02)00829-9","article-title":"Ischemic cerebral tissue and MCP-1 enhance rat bone marrow stromal cell migration in interface culture","volume":"30","author":"Wang","year":"2002","journal-title":"Exp. Hematol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0330","doi-asserted-by":"crossref","first-page":"1020","DOI":"10.1634\/stemcells.2005-0260","article-title":"Local irradiation not only induces homing of human mesenchymal stem cells at exposed sites but promotes their widespread engraftment to multiple organs: a study of their quantitative distribution after irradiation damage","volume":"24","author":"Fran\u00e7ois","year":"2006","journal-title":"Stem Cells"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0335","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1126\/science.276.5309.71","article-title":"Marrow stromal cells as stem cells for nonhematopoietic tissues","volume":"276","author":"Prockop","year":"1997","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0340","doi-asserted-by":"crossref","first-page":"1413","DOI":"10.1016\/j.exphem.2006.06.007","article-title":"In vitro analysis of multipotent mesenchymal stromal cells as potential cellular therapeutics in neurometabolic diseases in pediatric patients","volume":"34","author":"M\u00fcller","year":"2006","journal-title":"Exp. Hematol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0345","doi-asserted-by":"crossref","first-page":"1675","DOI":"10.1016\/S0301-472X(99)00101-0","article-title":"Bone marrow-derived mesenchymal stem cells remain host-derived despite successful hematopoietic engraftment after allogeneic transplantation in patients with lysosomal and peroxisomal storage diseases","volume":"27","author":"Ko\u00e7","year":"1999","journal-title":"Exp. Hematol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0350","doi-asserted-by":"crossref","first-page":"11","DOI":"10.3324\/haematol.11802","article-title":"Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report","volume":"93","author":"Meuleman","year":"2008","journal-title":"Haematologica"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0355","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1038\/sj.bmt.1703650","article-title":"Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH)","volume":"30","author":"Ko\u00e7","year":"2002","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0360","doi-asserted-by":"crossref","first-page":"1006","DOI":"10.1089\/hum.2005.16.1006","article-title":"Neurodegeneration augments the ability of bone marrow-derived mesenchymal stem cells to fuse with Purkinje neurons in Niemann-Pick type C mice","volume":"16","author":"Bae","year":"2005","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0365","doi-asserted-by":"crossref","first-page":"876","DOI":"10.1016\/j.ymthe.2003.07.008","article-title":"Ex vivo gene therapy using bone marrow-derived cells: combined effects of intracerebral and intravenous transplantation in a mouse model of Niemann-Pick disease","volume":"8","author":"Jin","year":"2003","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0370","doi-asserted-by":"crossref","first-page":"6967","DOI":"10.1073\/pnas.78.11.6967","article-title":"Morphologic study of the internalization of a lysosomal enzyme by the mannose 6-phosphate receptor in cultured Chinese hamster ovary cells","volume":"78","author":"Willingham","year":"1981","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0375","doi-asserted-by":"crossref","first-page":"202","DOI":"10.1038\/nrm1050","article-title":"Mannose 6-phosphate receptors: new twists in the tale","volume":"4","author":"Ghosh","year":"2003","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0380","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1002\/iub.284","article-title":"Therapy for lysosomal storage disorders","volume":"62","author":"Beck","year":"2010","journal-title":"IUBMB Life"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0385","doi-asserted-by":"crossref","first-page":"175","DOI":"10.1007\/s10545-007-0540-z","article-title":"Enzyme, cell and gene-based therapies for metachromatic leukodystrophy","volume":"30","author":"Sevin","year":"2007","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0390","doi-asserted-by":"crossref","first-page":"1979","DOI":"10.1093\/hmg\/ddh220","article-title":"Efficacy of enzyme replacement therapy in alpha-mannosidosis mice: a preclinical animal study","volume":"13","author":"Roces","year":"2004","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0395","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.2011.10.002_bb0400","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.2011.10.002_bb0405","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.2011.10.002_bb0410","doi-asserted-by":"crossref","first-page":"433","DOI":"10.1007\/s00109-008-0309-3","article-title":"Non-inhibitory antibodies impede lysosomal storage reduction during enzyme replacement therapy of a lysosomal storage disease","volume":"86","author":"Matzner","year":"2008","journal-title":"J. Mol. Med. (Berl.)"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0415","doi-asserted-by":"crossref","first-page":"471","DOI":"10.2119\/2007-00063Matzner","article-title":"Induction of tolerance to human arylsulfatase A in a mouse model of metachromatic leukodystrophy","volume":"13","author":"Matzner","year":"2007","journal-title":"Mol. Med."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0420","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.2011.10.002_bb0425","doi-asserted-by":"crossref","first-page":"2760","DOI":"10.1093\/hmg\/ddr175","article-title":"Intracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy","volume":"20","author":"Stroobants","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0430","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."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0435","doi-asserted-by":"crossref","first-page":"17487","DOI":"10.1074\/jbc.M110.189381","article-title":"Transport of arylsulfatase A across the blood\u2013brain barrier in vitro","volume":"286","author":"Matthes","year":"2011","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0440","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1001\/archneur.1969.00480080047005","article-title":"Metachromatic leukodystrophy. Treatment with arylsulfatase-A","volume":"20","author":"Greene","year":"1969","journal-title":"Arch. Neurol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0460","series-title":"ACMG Meeting","article-title":"Intravenous enzyme replacement therapy for metachromatic leukodystrophy (MLD)","author":"Dali","year":"2009"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0465","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1038\/sj.gt.3302883","article-title":"Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer","volume":"14","author":"Sevin","year":"2007","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0470","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":"10.1016\/j.ymgme.2011.10.002_bb0475","doi-asserted-by":"crossref","first-page":"188","DOI":"10.1272\/jnms.74.188","article-title":"Recombinant adeno-associated virus-mediated gene delivery to the central nervous system","volume":"74","author":"Kurai","year":"2007","journal-title":"J. Nippon Med. Sch."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0480","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1038\/sj.mt.6300012","article-title":"AAV1 mediated co-expression of formylglycine-generating enzyme and arylsulfatase a efficiently corrects sulfatide storage in a mouse model of metachromatic leukodystrophy","volume":"15","author":"Kurai","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0485","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":"2010","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0490","doi-asserted-by":"crossref","first-page":"2208","DOI":"10.1093\/hmg\/ddq099","article-title":"Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models","volume":"19","author":"Lattanzi","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0495","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":"10.1016\/j.ymgme.2011.10.002_bb0500","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":"10.1016\/j.ymgme.2011.10.002_bb0505","doi-asserted-by":"crossref","first-page":"805","DOI":"10.1038\/sj.gt.3301150","article-title":"Retrovirally expressed human arylsulfatase A corrects the metabolic defect of arylsulfatase A-deficient mouse cells","volume":"7","author":"Matzner","year":"2000","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0510","doi-asserted-by":"crossref","first-page":"578","DOI":"10.1177\/08830738030180090401","article-title":"Leukodystrophies: pathogenesis, diagnosis, strategies, therapies, and future research directions","volume":"18","author":"Maria","year":"2003","journal-title":"J. Child Neurol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0515","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1016\/S0304-3940(99)00647-3","article-title":"Neuromotor alterations and cerebellar deficits in aged arylsulfatase A-deficient transgenic mice","volume":"273","author":"D'Hooge","year":"1999","journal-title":"Neurosci. Lett."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0520","doi-asserted-by":"crossref","first-page":"261","DOI":"10.1007\/s00401-004-0883-6","article-title":"Lysosomal sulfatide storage in the brain of arylsulfatase A-deficient mice: cellular alterations and topographic distribution","volume":"108","author":"Wittke","year":"2004","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0525","doi-asserted-by":"crossref","first-page":"1267","DOI":"10.1038\/mt.2008.111","article-title":"Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain","volume":"16","author":"Cunningham","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0530","doi-asserted-by":"crossref","first-page":"498","DOI":"10.1002\/jgm.1325","article-title":"Global diffuse distribution in the brain and efficient gene delivery to the dorsal root ganglia by intrathecal injection of adeno-associated viral vector serotype 1","volume":"11","author":"Iwamoto","year":"2009","journal-title":"J. Gene Med."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0535","doi-asserted-by":"crossref","first-page":"445","DOI":"10.1016\/S0092-8674(03)00348-9","article-title":"The multiple sulfatase deficiency gene encodes an essential and limiting factor for the activity of sulfatases","volume":"113","author":"Cosma","year":"2003","journal-title":"Cell"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0540","doi-asserted-by":"crossref","first-page":"929","DOI":"10.1089\/hum.2005.16.929","article-title":"Coexpression of formylglycine-generating enzyme is essential for synthesis and secretion of functional arylsulfatase A in a mouse model of metachromatic leukodystrophy","volume":"16","author":"Takakusaki","year":"2005","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0545","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."},{"issue":"Suppl. 1","key":"10.1016\/j.ymgme.2011.10.002_bb0550","doi-asserted-by":"crossref","first-page":"S50","DOI":"10.1016\/S1090-3798(11)70168-4","article-title":"Brain gene therapy for metachromatic leukodystrophy: from bench to bedside","volume":"15","author":"Sevin","year":"2011","journal-title":"Eur. J. Paediatr. Neurol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0555","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1007\/s100380050004","article-title":"Transduction of fibroblasts and CD34+ progenitors using a selectable retroviral vector containing cDNAs encoding arylsulfatase A and CD24","volume":"45","author":"Tsutsuda-Asano","year":"2000","journal-title":"J. Hum. Genet."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0560","doi-asserted-by":"crossref","first-page":"881","DOI":"10.1007\/BF00714283","article-title":"Correction of enzyme deficiency in metachromatic leukodystrophy fibroblasts by retroviral-mediated transfer of the human arylsulphatase A gene","volume":"16","author":"Ohashi","year":"1993","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0565","doi-asserted-by":"crossref","first-page":"2111","DOI":"10.1089\/hum.1998.9.14-2111","article-title":"Transduced fibroblasts and metachromatic leukodystrophy lymphocytes transfer arylsulfatase A to myelinating glia and deficient cells in vitro","volume":"9","author":"Sangalli","year":"1998","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0570","first-page":"343","article-title":"Retroviral gene transfer and sustained expression of human arylsulfatase A","volume":"3","author":"Learish","year":"1996","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0575","first-page":"443","article-title":"Successful transduction of oligodendrocytes and restoration of arylsulfatase A deficiency in metachromatic leukodystrophy fibroblasts using an adenovirus vector","volume":"2","author":"Ohashi","year":"1995","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0580","doi-asserted-by":"crossref","first-page":"1250","DOI":"10.1038\/sj.gt.3301232","article-title":"Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector","volume":"7","author":"Matzner","year":"2000","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0585","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1038\/sj.gt.3301593","article-title":"Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations","volume":"9","author":"Matzner","year":"2002","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0590","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":"10.1016\/j.ymgme.2011.10.002_bb0595","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.2011.10.002_bb0600","doi-asserted-by":"crossref","first-page":"1373","DOI":"10.1038\/mt.2010.74","article-title":"Successful treatment of metachromatic leukodystrophy using bone marrow transplantation of HoxB4 overexpressing cells","volume":"18","author":"Miyake","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0605","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.2011.10.002_bb0615","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1016\/j.jns.2007.01.010","article-title":"Metabolic correction in oligodendrocytes derived from metachromatic leukodystrophy mouse model by using encapsulated recombinant myoblasts","volume":"255","author":"Consiglio","year":"2007","journal-title":"J. Neurol. Sci."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0620","doi-asserted-by":"crossref","first-page":"469","DOI":"10.1007\/s11011-008-9107-4","article-title":"In vitro correction of ARSA deficiency in human skin fibroblasts from metachromatic leukodystrophy patients after treatment with microencapsulated recombinant cells","volume":"23","author":"Lagranha","year":"2008","journal-title":"Metab. Brain Dis."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0625","doi-asserted-by":"crossref","first-page":"869","DOI":"10.1016\/S0896-6273(00)80469-9","article-title":"Oligodendrocyte population dynamics and the role of PDGF in vivo","volume":"20","author":"Calver","year":"1998","journal-title":"Neuron"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0630","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1002\/(SICI)1097-4695(19981115)37:3<413::AID-NEU7>3.0.CO;2-8","article-title":"In vivo proliferation of oligodendrocyte progenitors expressing PDGFalphaR during early remyelination","volume":"37","author":"Redwine","year":"1998","journal-title":"J. Neurobiol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0635","doi-asserted-by":"crossref","first-page":"3109","DOI":"10.1523\/JNEUROSCI.4366-05.2006","article-title":"Oligodendroglial progenitor cell therapy limits central neurological deficits in mice with metachromatic leukodystrophy","volume":"26","author":"Givogri","year":"2006","journal-title":"J. Neurosci."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0640","doi-asserted-by":"crossref","first-page":"1707","DOI":"10.1126\/science.1553558","article-title":"Generation of neurons and astrocytes from isolated cells of the adult mammalian central nervous system","volume":"255","author":"Reynolds","year":"1992","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2011.10.002_bb0645","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1016\/j.brainres.2006.03.116","article-title":"Ex vivo cell-mediated gene therapy for metachromatic leukodystrophy using neurospheres","volume":"1094","author":"Kawabata","year":"2006","journal-title":"Brain Res."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0650","doi-asserted-by":"crossref","first-page":"340","DOI":"10.1159\/000150127","article-title":"Multipotential neural precursors transplanted into the metachromatic leukodystrophy brain fail to generate oligodendrocytes but contribute to limit brain dysfunction","volume":"30","author":"Givogri","year":"2008","journal-title":"Dev. Neurosci."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0655","doi-asserted-by":"crossref","first-page":"208","DOI":"10.1111\/j.1750-3639.2005.tb00522.x","article-title":"ES cell-derived glial precursors contribute to remyelination in acutely demyelinated spinal cord lesions","volume":"15","author":"Perez-Bouza","year":"2005","journal-title":"Brain Pathol."},{"key":"10.1016\/j.ymgme.2011.10.002_bb0660","doi-asserted-by":"crossref","first-page":"1686","DOI":"10.1038\/sj.gt.3302834","article-title":"Embryonic stem cell-based reduction of central nervous system sulfatide storage in an animal model of metachromatic leukodystrophy","volume":"13","author":"Klein","year":"2006","journal-title":"Gene Ther."}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719211003714?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719211003714?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,4,13]],"date-time":"2024-04-13T22:44:56Z","timestamp":1713048296000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719211003714"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2012,1]]},"references-count":124,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2012,1]]}},"alternative-id":["S1096719211003714"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2011.10.002","relation":{},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2012,1]]}}}