{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,3]],"date-time":"2026-06-03T02:43:46Z","timestamp":1780454626397,"version":"3.54.1"},"reference-count":81,"publisher":"Springer Science and Business Media LLC","issue":"3","license":[{"start":{"date-parts":[[2013,4,11]],"date-time":"2013-04-11T00:00:00Z","timestamp":1365638400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["BioDrugs"],"published-print":{"date-parts":[[2013,6]]},"DOI":"10.1007\/s40259-013-0032-7","type":"journal-article","created":{"date-parts":[[2013,4,10]],"date-time":"2013-04-10T08:21:27Z","timestamp":1365582087000},"page":"237-246","source":"Crossref","is-referenced-by-count":26,"title":["Gene Therapy for Fabry Disease: A Review of the Literature"],"prefix":"10.1007","volume":"27","author":[{"given":"Aritz Perez","family":"Ruiz de Garibay","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mar\u00eda \u00c1ngeles","family":"Solin\u00eds","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alicia","family":"Rodr\u00edguez-Gasc\u00f3n","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2013,4,11]]},"reference":[{"issue":"21","key":"32_CR1","doi-asserted-by":"crossref","first-page":"1163","DOI":"10.1056\/NEJM196705252762101","volume":"276","author":"RO Brady","year":"1967","unstructured":"Brady RO, Gal AE, Bradley RM, et al. Enzymatic defect in Fabry\u2019s disease. N Engl J Med. 1967;276(21):1163\u20137.","journal-title":"N Engl J Med"},{"key":"32_CR2","doi-asserted-by":"crossref","first-page":"3148","DOI":"10.1016\/S0021-9258(18)51888-3","volume":"238","author":"CC Sweeley","year":"1963","unstructured":"Sweeley CC, Klionsky B. Fabry\u2019s disease: classification as a sphingolipidosis and partial characterization of a novel glycolipid. J Biol Chem. 1963;238:3148\u201350.","journal-title":"J Biol Chem"},{"issue":"7","key":"32_CR3","doi-asserted-by":"crossref","first-page":"1559","DOI":"10.1161\/01.STR.32.7.1559","volume":"32","author":"G Altarescu","year":"2001","unstructured":"Altarescu G, Moore DF, Pursley R, et al. Enhanced endothelium-dependent vasodilation in Fabry disease. Stroke. 2001;32(7):1559\u201362.","journal-title":"Stroke"},{"key":"32_CR4","first-page":"3733","volume-title":"The metabolic and molecular basis of inherited disease","author":"RJ Desnick","year":"2001","unstructured":"Desnick RJ, Ioannou YA, Eng CM. Alpha-galactosidase A deficiency: Fabry disease. In: Scriver CR, Beudet AL, Sly WS, Valle D, editors. The metabolic and molecular basis of inherited disease. 8th ed. NewYork: McGraw-Hill; 2001. p. 3733\u201374.","edition":"8"},{"issue":"1","key":"32_CR5","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1086\/504601","volume":"79","author":"M Spada","year":"2006","unstructured":"Spada M, Pagliardini S, Yasuda M, et al. High incidence of later-onset Fabry disease revealed by newborn screening. Am J Hum Genet. 2006;79(1):31\u201340.","journal-title":"Am J Hum Genet"},{"issue":"5","key":"32_CR6","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1161\/CIRCGENETICS.109.862920","volume":"2","author":"HY Lin","year":"2009","unstructured":"Lin HY, Chong KW, Hsu JH, et al. High incidence of the cardiac variant of Fabry disease revealed by newborn screening in the Taiwan Chinese population. Circ Cardiovasc Genet. 2009;2(5):450\u20136.","journal-title":"Circ Cardiovasc Genet"},{"issue":"10","key":"32_CR7","doi-asserted-by":"crossref","first-page":"1397","DOI":"10.1002\/humu.21074","volume":"30","author":"WL Hwu","year":"2009","unstructured":"Hwu WL, Chien YH, Lee NC, et al. Newborn screening for Fabry disease in Taiwan reveals a high incidence of the later-onset GLA mutation c.936+919G>A (IVS4+919G>A). Hum Mutat. 2009;30(10):1397\u2013405.","journal-title":"Hum Mutat"},{"issue":"11","key":"32_CR8","doi-asserted-by":"crossref","first-page":"750","DOI":"10.1136\/jmg.38.11.750","volume":"38","author":"KD MacDermot","year":"2001","unstructured":"MacDermot KD, Holmes A, Miners AH. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males. J Med Genet. 2001;38(11):750\u201360.","journal-title":"J Med Genet"},{"key":"32_CR9","doi-asserted-by":"crossref","first-page":"155","DOI":"10.2147\/DDDT.S11985","volume":"5","author":"U Ramaswami","year":"2011","unstructured":"Ramaswami U. Update on role of agalsidase alfa in management of Fabry disease. Drug Des Devel Ther. 2011;5:155\u201373.","journal-title":"Drug Des Devel Ther"},{"issue":"1","key":"32_CR10","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1097\/GIM.0b013e31802d8321","volume":"9","author":"RY Wang","year":"2007","unstructured":"Wang RY, Lelis A, Mirocha J, et al. Heterozygous Fabry women are not just carriers, but have a significant burden of disease and impaired quality of life. Genet Med. 2007;9(1):34\u201345.","journal-title":"Genet Med"},{"issue":"2","key":"32_CR11","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1016\/j.ymgme.2007.09.013","volume":"93","author":"WR Wilcox","year":"2008","unstructured":"Wilcox WR, Oliveira JP, Hopkin RJ, et al. Females with Fabry disease frequently have major organ involvement: lessons from the Fabry Registry. Mol Genet Metab. 2008;93(2):112\u201328.","journal-title":"Mol Genet Metab"},{"key":"32_CR12","doi-asserted-by":"crossref","unstructured":"Hoffmann B, Garcia de Lorenzo A, Mehta A, et al. Effects of enzyme replacement therapy on pain and health related quality of life in patients with Fabry disease: data from FOS (Fabry Outcome Survey). J Med Genet. 2005;42(3):247\u201352.","DOI":"10.1136\/jmg.2004.025791"},{"key":"32_CR13","unstructured":"Schiffmann R, Martin RA, Reimschisel T, et al. Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease. J Pediatr. 2010;156(5):832\u20137, 837.e1."},{"issue":"5","key":"32_CR14","doi-asserted-by":"crossref","first-page":"485","DOI":"10.1111\/j.1399-0004.2011.01671.x","volume":"81","author":"U Ramaswami","year":"2012","unstructured":"Ramaswami U, Parini R, Pintos-Morell G, et al. Fabry disease in children and response to enzyme replacement therapy: results from the Fabry Outcome Survey. Clin Genet. 2012;81(5):485\u201390.","journal-title":"Clin Genet"},{"issue":"4","key":"32_CR15","doi-asserted-by":"crossref","first-page":"524","DOI":"10.1161\/CIRCULATIONAHA.108.794529","volume":"119","author":"F Weidemann","year":"2009","unstructured":"Weidemann F, Niemann M, Breunig F, et al. Long-term effects of enzyme replacement therapy on Fabry cardiomyopathy: evidence for a better outcome with early treatment. Circulation. 2009;119(4):524\u20139.","journal-title":"Circulation"},{"issue":"3","key":"32_CR16","doi-asserted-by":"crossref","first-page":"1042","DOI":"10.1093\/ndt\/gfr420","volume":"27","author":"DG Warnock","year":"2012","unstructured":"Warnock DG, Ortiz A, Mauer M, et al. Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation. Nephrol Dial Transplant. 2012;27(3):1042\u20139.","journal-title":"Nephrol Dial Transplant"},{"issue":"3","key":"32_CR17","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1016\/j.ymgme.2011.12.006","volume":"105","author":"WR Wilcox","year":"2012","unstructured":"Wilcox WR, Linthorst GE, Germain DP, et al. Anti-alpha-galactosidase A antibody response to agalsidase beta treatment: data from the Fabry Registry. Mol Genet Metab. 2012;105(3):443\u20139.","journal-title":"Mol Genet Metab"},{"issue":"7","key":"32_CR18","doi-asserted-by":"crossref","first-page":"e598","DOI":"10.1371\/journal.pone.0000598","volume":"2","author":"AC Vedder","year":"2007","unstructured":"Vedder AC, Linthorst GE, Houge G, et al. Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2\u00a0mg\/kg. PLoS One. 2007;2(7):e598.","journal-title":"PLoS One"},{"issue":"1","key":"32_CR19","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1038\/4801","volume":"5","author":"JQ Fan","year":"1999","unstructured":"Fan JQ, Ishii S, Asano N, Suzuki Y. Accelerated transport and maturation of lysosomal alpha-galactosidase A in Fabry lymphoblasts by an enzyme inhibitor. Nat Med. 1999;5(1):112\u20135.","journal-title":"Nat Med"},{"issue":"6","key":"32_CR20","doi-asserted-by":"crossref","first-page":"408","DOI":"10.1016\/j.bbadis.2008.03.001","volume":"1782","author":"R Hamanaka","year":"2008","unstructured":"Hamanaka R, Shinohara T, Yano S, et al. Rescue of mutant alpha-galactosidase A in the endoplasmic reticulum by 1-deoxygalactonojirimycin leads to trafficking to lysosomes. Biochim Biophys Acta. 2008;1782(6):408\u201313.","journal-title":"Biochim Biophys Acta"},{"issue":"2","key":"32_CR21","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1093\/jb\/mvq125","volume":"149","author":"C Shiozuka","year":"2011","unstructured":"Shiozuka C, Taguchi A, Matsuda J, et al. Increased globotriaosylceramide levels in a transgenic mouse expressing human alpha1,4-galactosyltransferase and a mouse model for treating Fabry disease. J Biochem. 2011;149(2):161\u201370.","journal-title":"J Biochem"},{"issue":"1","key":"32_CR22","doi-asserted-by":"crossref","first-page":"18","DOI":"10.2183\/pjab.88.18","volume":"88","author":"S Ishii","year":"2012","unstructured":"Ishii S. Pharmacological chaperone therapy for Fabry disease. Proc Jpn Acad Ser B Phys Biol Sci. 2012;88(1):18\u201330.","journal-title":"Proc Jpn Acad Ser B Phys Biol Sci"},{"issue":"5","key":"32_CR23","doi-asserted-by":"crossref","first-page":"839","DOI":"10.1016\/j.ymthe.2006.01.006","volume":"13","author":"MS Sands","year":"2006","unstructured":"Sands MS, Davidson BL. Gene therapy for lysosomal storage diseases. Mol Ther. 2006;13(5):839\u201349.","journal-title":"Mol Ther"},{"issue":"7","key":"32_CR24","doi-asserted-by":"crossref","first-page":"905","DOI":"10.1089\/hum.1995.6.7-905","volume":"6","author":"Y Sugimoto","year":"1995","unstructured":"Sugimoto Y, Aksentijevich I, Murray GJ, et al. Retroviral coexpression of a multidrug resistance gene (MDR1) and human alpha-galactosidase A for gene therapy of Fabry disease. Hum Gene Ther. 1995;6(7):905\u201315.","journal-title":"Hum Gene Ther"},{"issue":"15","key":"32_CR25","doi-asserted-by":"crossref","first-page":"7917","DOI":"10.1073\/pnas.93.15.7917","volume":"93","author":"JA Medin","year":"1996","unstructured":"Medin JA, Tudor M, Simovitch R, et al. Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector. Proc Natl Acad Sci USA. 1996;93(15):7917\u201322.","journal-title":"Proc Natl Acad Sci USA"},{"issue":"6","key":"32_CR26","doi-asserted-by":"crossref","first-page":"2540","DOI":"10.1073\/pnas.94.6.2540","volume":"94","author":"T Ohshima","year":"1997","unstructured":"Ohshima T, Murray GJ, Swaim WD, et al. Alpha-Galactosidase A deficient mice: a model of Fabry disease. Proc Natl Acad Sci USA. 1997;94(6):2540\u20134.","journal-title":"Proc Natl Acad Sci USA"},{"issue":"7","key":"32_CR27","doi-asserted-by":"crossref","first-page":"1149","DOI":"10.1016\/S0301-472X(99)00050-8","volume":"27","author":"T Takenaka","year":"1999","unstructured":"Takenaka T, Hendrickson CS, Tworek DM, et al. Enzymatic and functional correction along with long-term enzyme secretion from transduced bone marrow hematopoietic stem\/progenitor and stromal cells derived from patients with Fabry disease. Exp Hematol. 1999;27(7):1149\u201359.","journal-title":"Exp Hematol"},{"issue":"18","key":"32_CR28","doi-asserted-by":"crossref","first-page":"2881","DOI":"10.1089\/10430349950016302","volume":"10","author":"N Takiyama","year":"1999","unstructured":"Takiyama N, Dunigan JT, Vallor MJ, et al. Retrovirus-mediated transfer of human alpha-galactosidase A gene to human CD34+ hematopoietic progenitor cells. Hum Gene Ther. 1999;10(18):2881\u20139.","journal-title":"Hum Gene Ther"},{"issue":"12","key":"32_CR29","doi-asserted-by":"crossref","first-page":"1931","DOI":"10.1089\/10430349950017293","volume":"10","author":"T Takenaka","year":"1999","unstructured":"Takenaka T, Qin G, Brady RO, et al. Circulating alpha-galactosidase A derived from transduced bone marrow cells: relevance for corrective gene transfer for Fabry disease. Hum Gene Ther. 1999;10(12):1931\u20139.","journal-title":"Hum Gene Ther"},{"issue":"13","key":"32_CR30","doi-asserted-by":"crossref","first-page":"7515","DOI":"10.1073\/pnas.120177997","volume":"97","author":"T Takenaka","year":"2000","unstructured":"Takenaka T, Murray GJ, Qin G, et al. Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells. Proc Natl Acad Sci USA. 2000;97(13):7515\u201320.","journal-title":"Proc Natl Acad Sci USA"},{"issue":"6","key":"32_CR31","doi-asserted-by":"crossref","first-page":"3428","DOI":"10.1073\/pnas.061020598","volume":"98","author":"G Qin","year":"2001","unstructured":"Qin G, Takenaka T, Telsch K, et al. Preselective gene therapy for Fabry disease. Proc Natl Acad Sci USA. 2001;98(6):3428\u201333.","journal-title":"Proc Natl Acad Sci USA"},{"key":"32_CR32","unstructured":"Gene Therapy Clinical Trials Worldwide. Provided by the Journal of Gene Medicine. Jon Wiley and Sons Ltd, Oxford. 2012. http:\/\/www.wiley.co.k\/genmed\/clinical . Accessed 02 Nov 2012."},{"issue":"2","key":"32_CR33","doi-asserted-by":"crossref","first-page":"173","DOI":"10.1002\/jmv.10467","volume":"71","author":"J D\u2019Costa","year":"2003","unstructured":"D\u2019Costa J, Harvey-White J, Qasba P, et al. HIV-2 derived lentiviral vectors: gene transfer in Parkinson\u2019s and Fabry disease models in vitro. J Med Virol. 2003;71(2):173\u201382.","journal-title":"J Med Virol"},{"issue":"48","key":"32_CR34","doi-asserted-by":"crossref","first-page":"16909","DOI":"10.1073\/pnas.0407572101","volume":"101","author":"M Yoshimitsu","year":"2004","unstructured":"Yoshimitsu M, Sato T, Tao K, et al. Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors. Proc Natl Acad Sci USA. 2004;101(48):16909\u201314.","journal-title":"Proc Natl Acad Sci USA"},{"issue":"3","key":"32_CR35","doi-asserted-by":"crossref","first-page":"256","DOI":"10.1038\/sj.gt.3302839","volume":"14","author":"M Yoshimitsu","year":"2007","unstructured":"Yoshimitsu M, Higuchi K, Ramsubir S, et al. Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem\/progenitor cells. Gene Ther. 2007;14(3):256\u201365.","journal-title":"Gene Ther"},{"issue":"11","key":"32_CR36","doi-asserted-by":"crossref","first-page":"1503","DOI":"10.1253\/circj.70.1503","volume":"70","author":"M Yoshimitsu","year":"2006","unstructured":"Yoshimitsu M, Higuchi K, Dawood F, et al. Correction of cardiac abnormalities in Fabry mice by direct intraventricular injection of a recombinant lentiviral vector that engineers expression of alpha-galactosidase A. Circ J. 2006;70(11):1503\u20138.","journal-title":"Circ J"},{"issue":"1","key":"32_CR37","doi-asserted-by":"crossref","first-page":"115","DOI":"10.1016\/j.jjcc.2010.08.003","volume":"57","author":"CJ Lee","year":"2011","unstructured":"Lee CJ, Fan X, Guo X, et al. Promoter-specific lentivectors for long-term, cardiac-directed therapy of Fabry disease. J Cardiol. 2011;57(1):115\u201322.","journal-title":"J Cardiol"},{"key":"32_CR38","doi-asserted-by":"crossref","unstructured":"Siatskas C, Medin JA. Gene therapy for Fabry disease. J Inherit Metab Dis. 2001;24(Suppl. 2):25\u201341; discussion 11-2.","DOI":"10.1023\/A:1012455421014"},{"issue":"2","key":"32_CR39","first-page":"137","volume":"3","author":"Y Yang","year":"1996","unstructured":"Yang Y, Jooss KU, Su Q, et al. Immune responses to viral antigens versus transgene product in the elimination of recombinant adenovirus-infected hepatocytes in vivo. Gene Ther. 1996;3(2):137\u201344.","journal-title":"Gene Ther"},{"issue":"10","key":"32_CR40","doi-asserted-by":"crossref","first-page":"1667","DOI":"10.1089\/10430349950017671","volume":"10","author":"RJ Ziegler","year":"1999","unstructured":"Ziegler RJ, Yew NS, Li C, et al. Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer. Hum Gene Ther. 1999;10(10):1667\u201382.","journal-title":"Hum Gene Ther"},{"issue":"8","key":"32_CR41","doi-asserted-by":"crossref","first-page":"935","DOI":"10.1089\/10430340252939041","volume":"13","author":"RJ Ziegler","year":"2002","unstructured":"Ziegler RJ, Li C, Cherry M, et al. Correction of the nonlinear dose response improves the viability of adenoviral vectors for gene therapy of Fabry disease. Hum Gene Ther. 2002;13(8):935\u201345.","journal-title":"Hum Gene Ther"},{"issue":"6","key":"32_CR42","doi-asserted-by":"crossref","first-page":"745","DOI":"10.1006\/mthe.2002.0605","volume":"5","author":"C Li","year":"2002","unstructured":"Li C, Ziegler RJ, Cherry M, et al. Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease. Mol Ther. 2002;5(6):745\u201354.","journal-title":"Mol Ther"},{"issue":"3","key":"32_CR43","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1089\/hum.2010.069","volume":"22","author":"MJ Passineau","year":"2011","unstructured":"Passineau MJ, Fahrenholz T, Machen L, et al. alpha-Galactosidase A expressed in the salivary glands partially corrects organ biochemical deficits in the Fabry mouse through endocrine trafficking. Hum Gene Ther. 2011;22(3):293\u2013301.","journal-title":"Hum Gene Ther"},{"issue":"4","key":"32_CR44","doi-asserted-by":"crossref","first-page":"583","DOI":"10.1128\/CMR.00008-08","volume":"21","author":"S Daya","year":"2008","unstructured":"Daya S, Berns KI. Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev. 2008;21(4):583\u201393.","journal-title":"Clin Microbiol Rev"},{"key":"32_CR45","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1186\/1743-422X-2-43","volume":"2","author":"MA Goncalves","year":"2005","unstructured":"Goncalves MA. Adeno-associated virus: from defective virus to effective vector. Virol J. 2005;2:43.","journal-title":"Virol J"},{"issue":"20","key":"32_CR46","doi-asserted-by":"crossref","first-page":"9451","DOI":"10.1128\/JVI.74.20.9451-9463.2000","volume":"74","author":"H Nakai","year":"2000","unstructured":"Nakai H, Storm TA, Kay MA. Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo. J Virol. 2000;74(20):9451\u201363.","journal-title":"J Virol"},{"issue":"5","key":"32_CR47","doi-asserted-by":"crossref","first-page":"2676","DOI":"10.1073\/pnas.051634498","volume":"98","author":"SC Jung","year":"2001","unstructured":"Jung SC, Han IP, Limaye A, et al. Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice. Proc Natl Acad Sci USA. 2001;98(5):2676\u201381.","journal-title":"Proc Natl Acad Sci USA"},{"issue":"21","key":"32_CR48","doi-asserted-by":"crossref","first-page":"13777","DOI":"10.1073\/pnas.222221899","volume":"99","author":"H Takahashi","year":"2002","unstructured":"Takahashi H, Hirai Y, Migita M, et al. Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer. Proc Natl Acad Sci USA. 2002;99(21):13777\u201382.","journal-title":"Proc Natl Acad Sci USA"},{"issue":"6","key":"32_CR49","doi-asserted-by":"crossref","first-page":"3450","DOI":"10.1073\/pnas.0537900100","volume":"100","author":"J Park","year":"2003","unstructured":"Park J, Murray GJ, Limaye A, et al. Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer. Proc Nat Acad Sci. 2003;100(6):3450\u20134.","journal-title":"Proc Nat Acad Sci"},{"issue":"2","key":"32_CR50","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1016\/j.ymthe.2003.11.015","volume":"9","author":"RJ Ziegler","year":"2004","unstructured":"Ziegler RJ, Lonning SM, Armentano D, et al. AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of [alpha]-galactosidase A and the induction of immune tolerance in Fabry mice. Mol Ther. 2004;9(2):231\u201340.","journal-title":"Mol Ther"},{"issue":"9","key":"32_CR51","doi-asserted-by":"crossref","first-page":"1574","DOI":"10.1038\/sj.gt.3300994","volume":"6","author":"N Chirmule","year":"1999","unstructured":"Chirmule N, Propert K, Magosin S, et al. Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther. 1999;6(9):1574\u201383.","journal-title":"Gene Ther"},{"issue":"3","key":"32_CR52","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1038\/sj.mt.6300066","volume":"15","author":"RJ Ziegler","year":"2007","unstructured":"Ziegler RJ, Cherry M, Barbon CM, et al. Correction of the biochemical and functional deficits in Fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A. Mol Ther. 2007;15(3):492\u2013500.","journal-title":"Mol Ther"},{"issue":"3","key":"32_CR53","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1016\/j.ymgme.2008.10.017","volume":"96","author":"K Ogawa","year":"2009","unstructured":"Ogawa K, Hirai Y, Ishizaki M, et al. Long-term inhibition of glycosphingolipid accumulation in Fabry model mice by a single systemic injection of AAV1 vector in the neonatal period. Mol Genet Metab. 2009;96(3):91\u20136.","journal-title":"Mol Genet Metab"},{"issue":"9","key":"32_CR54","doi-asserted-by":"crossref","first-page":"1677","DOI":"10.1038\/sj.mt.6300219","volume":"15","author":"DE Sabatino","year":"2007","unstructured":"Sabatino DE, Mackenzie TC, Peranteau W, et al. Persistent expression of hF. IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice. Mol Ther. 2007;15(9):1677\u201385.","journal-title":"Mol Ther"},{"key":"32_CR55","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1186\/1423-0127-17-26","volume":"17","author":"JO Choi","year":"2010","unstructured":"Choi JO, Lee MH, Park HY, et al. Characterization of Fabry mice treated with recombinant adeno-associated virus 2\/8-mediated gene transfer. J Biomed Sci. 2010;17:26.","journal-title":"J Biomed Sci"},{"issue":"1","key":"32_CR56","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1016\/j.ymthe.2005.08.017","volume":"13","author":"G Gao","year":"2006","unstructured":"Gao G, Lu Y, Calcedo R, et al. Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. Mol Ther. 2006;13(1):77\u201387.","journal-title":"Mol Ther"},{"issue":"11","key":"32_CR57","doi-asserted-by":"crossref","first-page":"1983","DOI":"10.1038\/mt.2010.175","volume":"18","author":"GD Hurlbut","year":"2010","unstructured":"Hurlbut GD, Ziegler RJ, Nietupski JB, et al. Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy. Mol Ther. 2010;18(11):1983\u201394.","journal-title":"Mol Ther"},{"issue":"11","key":"32_CR58","doi-asserted-by":"crossref","first-page":"1999","DOI":"10.1038\/mt.2011.119","volume":"19","author":"JB Nietupski","year":"2011","unstructured":"Nietupski JB, Hurlbut GD, Ziegler RJ, et al. Systemic administration of AAV-8-a-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression. Mol Ther. 2011;19(11):1999\u20132011.","journal-title":"Mol Ther"},{"issue":"2","key":"32_CR59","doi-asserted-by":"crossref","first-page":"197","DOI":"10.2174\/092986712803414141","volume":"19","author":"M Jafari","year":"2012","unstructured":"Jafari M, Soltani M, Naahidi S, et al. Nonviral approach for targeted nucleic acid delivery. Curr Med Chem. 2012;19(2):197\u2013208.","journal-title":"Curr Med Chem"},{"issue":"5","key":"32_CR60","doi-asserted-by":"crossref","first-page":"488","DOI":"10.1038\/sj.gt.3300410","volume":"4","author":"FJ Novo","year":"1997","unstructured":"Novo FJ, Gorecki DC, Goldspink G, et al. Gene transfer and expression of human alpha-galactosidase from mouse muscle in vitro and in vivo. Gene Ther. 1997;4(5):488\u201392.","journal-title":"Gene Ther"},{"key":"32_CR61","doi-asserted-by":"crossref","unstructured":"Estruch EJ, Hart SL, Kinnon C, et al. Non-viral, integrin-mediated gene transfer into fibroblasts from patients with lysosomal storage diseases. J Gene Med. 2001;3(5):488\u201397.","DOI":"10.1002\/jgm.214"},{"issue":"1","key":"32_CR62","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1002\/jgm.468","volume":"6","author":"M Przybylska","year":"2004","unstructured":"Przybylska M, Wu IH, Zhao H, et al. Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors. J Gene Med. 2004;6(1):85\u201392.","journal-title":"J Gene Med"},{"issue":"1","key":"32_CR63","doi-asserted-by":"crossref","first-page":"14","DOI":"10.1086\/316953","volume":"68","author":"YA Ioannou","year":"2001","unstructured":"Ioannou YA, Zeidner KM, Gordon RE, et al. Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice. Am J Hum Genet. 2001;68(1):14\u201325.","journal-title":"Am J Hum Genet"},{"issue":"5","key":"32_CR64","doi-asserted-by":"crossref","first-page":"985","DOI":"10.1016\/j.ymthe.2005.02.032","volume":"12","author":"MD Lavigne","year":"2005","unstructured":"Lavigne MD, Pohlschmidt M, Novo JF, et al. Promoter dependence of plasmid-pluronics targeted alpha galactosidase A expression in skeletal muscle of Fabry mice. Mol Ther. 2005;12(5):985\u201390.","journal-title":"Mol Ther"},{"issue":"11","key":"32_CR65","doi-asserted-by":"crossref","first-page":"986","DOI":"10.1038\/sj.gt.3301189","volume":"7","author":"P Lemieux","year":"2000","unstructured":"Lemieux P, Guerin N, Paradis G, et al. A combination of poloxamers increases gene expression of plasmid DNA in skeletal muscle. Gene Ther. 2000;7(11):986\u201391.","journal-title":"Gene Ther"},{"issue":"2","key":"32_CR66","doi-asserted-by":"crossref","first-page":"264","DOI":"10.1016\/j.ymthe.2005.04.007","volume":"12","author":"Q Chu","year":"2005","unstructured":"Chu Q, Joseph M, Przybylska M, et al. Transient siRNA-mediated attenuation of liver expression from an alpha-galactosidase A plasmid reduces subsequent humoral immune responses to the transgene product in mice. Mol Ther. 2005;12(2):264\u201373.","journal-title":"Mol Ther"},{"issue":"6","key":"32_CR67","doi-asserted-by":"crossref","first-page":"2097","DOI":"10.1096\/fj.07-093765","volume":"22","author":"MD Lavigne","year":"2008","unstructured":"Lavigne MD, Yates L, Coxhead P, et al. Nuclear-targeted chimeric vector enhancing nonviral gene transfer into skeletal muscle of Fabry mice in vivo. FASEB J. 2008;22(6):2097\u2013107.","journal-title":"FASEB J"},{"issue":"2","key":"32_CR68","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1007\/s12033-007-9008-5","volume":"38","author":"G Nakamura","year":"2008","unstructured":"Nakamura G, Maruyama H, Ishii S, et al. Naked plasmid DNA-based alpha-galactosidase A gene transfer partially reduces systemic accumulation of globotriaosylceramide in Fabry mice. Mol Biotechnol. 2008;38(2):109\u201319.","journal-title":"Mol Biotechnol"},{"key":"32_CR69","doi-asserted-by":"crossref","unstructured":"Ruiz de Garibay AP, Delgado D, Del Pozo-Rodriguez A, et al. Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy. Drug Des Devel Ther. 2012;6:303\u201310.","DOI":"10.2147\/DDDT.S36131"},{"issue":"1\u20132","key":"32_CR70","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1016\/j.ijpharm.2009.10.020","volume":"385","author":"A Pozo-Rodriguez del","year":"2010","unstructured":"del Pozo-Rodriguez A, Delgado D, Solinis MA, et al. Solid lipid nanoparticles as potential tools for gene therapy: in vivo protein expression after intravenous administration. Int J Pharm. 2010;385(1\u20132):157\u201362.","journal-title":"Int J Pharm"},{"issue":"1\u20132","key":"32_CR71","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1016\/j.ijpharm.2011.12.052","volume":"425","author":"D Delgado","year":"2012","unstructured":"Delgado D, Gasc\u00f3n AR, del Pozo-Rodr\u00edguez A, et al. Dextran\u2013protamine\u2013solid lipid nanoparticles as a non-viral vector for gene therapy: in vitro characterization and in vivo transfection after intravenous administration to mice. Int J Pharm. 2012;425(1\u20132):35\u201343.","journal-title":"Int J Pharm"},{"key":"32_CR72","doi-asserted-by":"crossref","unstructured":"Shen JS, Meng XL, Schiffmann R, et al. Establishment and characterization of Fabry disease endothelial cells with an extended lifespan. Mol Genet Metab. 2007;92(1\u20132):137\u201344.","DOI":"10.1016\/j.ymgme.2007.06.003"},{"key":"32_CR73","doi-asserted-by":"crossref","unstructured":"Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 2000;288(5466):669\u201372.","DOI":"10.1126\/science.288.5466.669"},{"issue":"9452","key":"32_CR74","doi-asserted-by":"crossref","first-page":"2181","DOI":"10.1016\/S0140-6736(04)17590-9","volume":"364","author":"HB Gaspar","year":"2004","unstructured":"Gaspar HB, Parsley KL, Howe S, et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet. 2004;364(9452):2181\u20137.","journal-title":"Lancet"},{"issue":"4","key":"32_CR75","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1056\/NEJMoa1000164","volume":"363","author":"S Hacein-Bey-Abina","year":"2010","unstructured":"Hacein-Bey-Abina S, Hauer J, Lim A, et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2010;363(4):355\u201364.","journal-title":"N Engl J Med"},{"issue":"3","key":"32_CR76","doi-asserted-by":"crossref","first-page":"603","DOI":"10.1042\/BJ20120146","volume":"443","author":"T Sakuma","year":"2012","unstructured":"Sakuma T, Barry MA, Ikeda Y. Lentiviral vectors: basic to translational. Biochem J. 2012;443(3):603\u201318.","journal-title":"Biochem J"},{"issue":"3","key":"32_CR77","doi-asserted-by":"crossref","first-page":"618","DOI":"10.1038\/sj.mt.6300075","volume":"15","author":"SB Liang","year":"2007","unstructured":"Liang SB, Yoshimitsu M, Poeppl A, et al. Multiple reduced-intensity conditioning regimens facilitate correction of Fabry mice after transplantation of transduced cells. Mol Ther. 2007;15(3):618\u201327.","journal-title":"Mol Ther"},{"issue":"1","key":"32_CR78","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1038\/gt.2011.210","volume":"20","author":"M Scaife","year":"2013","unstructured":"Scaife M, Pacienza N, Au BC, et al. Engineered human Tmpk fused with truncated cell-surface markers: versatile cell-fate control safety cassettes. Gene Ther. 2013;20(1):24\u201334.","journal-title":"Gene Ther"},{"issue":"13","key":"32_CR79","doi-asserted-by":"crossref","first-page":"5071","DOI":"10.1002\/j.1460-2075.1992.tb05614.x","volume":"11","author":"RM Kotin","year":"1992","unstructured":"Kotin RM, Linden RM, Berns KI. Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination. EMBO J. 1992;11(13):5071\u20138.","journal-title":"EMBO J"},{"issue":"5","key":"32_CR80","doi-asserted-by":"crossref","first-page":"517","DOI":"10.1165\/ajrcmb.11.5.7946381","volume":"11","author":"TR Flotte","year":"1994","unstructured":"Flotte TR, Afione SA, Zeitlin PL. Adeno-asociated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am J Respir Cell Mol Biol. 1994;11(5):517\u201321.","journal-title":"Am J Respir Cell Mol Biol"},{"key":"32_CR81","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1016\/B978-0-12-386509-0.00012-0","volume":"507","author":"JC Grieger","year":"2012","unstructured":"Grieger JC, Samulski RJ. Adeno-associated virus vectorology, manufacturing, and clinical applications. Methods Enzymol. 2012;507:229\u201354.","journal-title":"Methods Enzymol"}],"container-title":["BioDrugs"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s40259-013-0032-7.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1007\/s40259-013-0032-7\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s40259-013-0032-7","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,2,13]],"date-time":"2022-02-13T12:53:30Z","timestamp":1644756810000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/s40259-013-0032-7"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,4,11]]},"references-count":81,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2013,6]]}},"alternative-id":["32"],"URL":"https:\/\/doi.org\/10.1007\/s40259-013-0032-7","relation":{},"ISSN":["1173-8804","1179-190X"],"issn-type":[{"value":"1173-8804","type":"print"},{"value":"1179-190X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2013,4,11]]}}}