메뉴 건너뛰기




Volumn 47, Issue SUPPL. 1, 2009, Pages

Anderson-Fabry disease: Developments in diagnosis and treatment

Author keywords

Anderson Fabry disease; Biomarkers; Enzyme replacement therapy; Late onset; galactosidase A

Indexed keywords

ALPHA GALACTOSIDASE; BIOLOGICAL MARKER;

EID: 77649212988     PISSN: 09461965     EISSN: None     Source Type: Journal    
DOI: 10.5414/cpp47066     Document Type: Conference Paper
Times cited : (21)

References (82)
  • 7
    • 31544473757 scopus 로고    scopus 로고
    • Fabry's disease as a differential diagnosis of MS
    • Callegaro D, Kaimen-Maciel DR. Fabry's disease as a differential diagnosis of MS. Int MS J. 2006; 13: 27- 30.
    • (2006) Int MS J , vol.13 , pp. 27-30
    • Callegaro, D.1    Kaimen-Maciel, D.R.2
  • 8
    • 34548033303 scopus 로고    scopus 로고
    • The pharmacology of multiple regimens of agalsidase alfa enzyme replacement therapy for Fabry disease
    • DOI 10.1097/GIM.0b013e318133fb1b, PII 0012581720070800000003
    • Clarke JT, West ML, Bultas J, Schiffmann R. The pharmacology of multiple regimens of agalsidase alfa enzyme replacement therapy for Fabry disease. Genet Med. 2007; 9: 504-509. (Pubitemid 47282038)
    • (2007) Genetics in Medicine , vol.9 , Issue.8 , pp. 504-509
    • Clarke, J.T.R.1    West, M.L.2    Bultas, J.3    Schiffmann, R.4
  • 10
    • 33745684367 scopus 로고    scopus 로고
    • Asystematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type I
    • Connock M, Juarez-Garcia A, Frew E, Mans A, Dretzke J, Fry-Smith A et al. Asystematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type I. Health Technol Assess. 2006; 10: iii-iv, ix-113.
    • (2006) Health Technol Assess , vol.10
    • Connock, M.1    Juarez-Garcia, A.2    Frew, E.3    Mans, A.4    Dretzke, J.5    Fry-Smith, A.6
  • 11
    • 16844374890 scopus 로고    scopus 로고
    • Biomarkers in lysosomal storage diseases: A review
    • Cox TM. Biomarkers in lysosomal storage diseases: a review. Acta Paediatr. 2005; 94 (Suppl 447): 39-42.
    • (2005) Acta Paediatr , vol.94 , Issue.SUPPL. 447 , pp. 39-42
    • Cox, T.M.1
  • 15
    • 0037806518 scopus 로고    scopus 로고
    • Fabry disease: Unrecognized ESRD patients and effectiveness of enzyme replacement therapy on renal pathology and function
    • Desnick RJ. Fabry disease: unrecognized ESRD patients and effectiveness of enzyme replacement therapy on renal pathology and function. J Inherit Metab Dis. 2002; 25: 116.
    • (2002) J Inherit Metab Dis , vol.25 , pp. 116
    • Desnick, R.J.1
  • 16
    • 0036895451 scopus 로고    scopus 로고
    • Enzyme replacement and enhancement therapies: Lessons from lysosomal disorders
    • DOI 10.1038/nrg963
    • Desnick RJ, Schuchman EH. Enzyme replacement and enhancement therapies: lessons from lysosomal disorders. Nat Rev Genet. 2002; 3: 954-966. (Pubitemid 35423507)
    • (2002) Nature Reviews Genetics , vol.3 , Issue.12 , pp. 954-966
    • Desnick, R.J.1    Schuchman, E.H.2
  • 20
    • 34748866532 scopus 로고    scopus 로고
    • Active-site-specific chaperone therapy for Fabry disease: Yin and Yang of enzyme inhibitors
    • DOI 10.1111/j.1742-4658.2007.06041.x
    • Fan JQ, Ishii S. Active-site-specific chaperone therapy for Fabry disease. Yin and Yang of enzyme inhibitors. FEBS J. 2007; 274: 4962-4971. (Pubitemid 47481192)
    • (2007) FEBS Journal , vol.274 , Issue.19 , pp. 4962-4971
    • Fan, J.-Q.1    Ishii, S.2
  • 21
    • 34548681011 scopus 로고    scopus 로고
    • Images in cardiovascular medicine. Cryptogenic ventricular arrhythmias and sudden death by Fabry disease: Prominent infiltration of cardiac conduction tissue
    • Frustaci A, Chimenti C. Images in cardiovascular medicine. Cryptogenic ventricular arrhythmias and sudden death by Fabry disease: prominent infiltration of cardiac conduction tissue. Circulation. 2007; 116: 350- 351.
    • (2007) Circulation , vol.116 , pp. 350-351
    • Frustaci, A.1    Chimenti, C.2
  • 23
    • 20944449722 scopus 로고    scopus 로고
    • Osteopenia and osteoporosis: Previously unrecognized manifestations of Fabry disease [3]
    • DOI 10.1111/j.1399-0004.2005.00457.x
    • Germain DP, Benistan K, Boutouyrie P, Mutschler C. Osteopenia and osteoporosis: previously unrecognized manifestations of Fabry disease. Clin Genet. 2005; 68: 93-95. (Pubitemid 40867545)
    • (2005) Clinical Genetics , vol.68 , Issue.1 , pp. 93-95
    • Germain, D.P.1    Benistan, K.2    Boutouyrie, P.3    Mutschler, C.4
  • 25
    • 0028220472 scopus 로고
    • Marked elevation of plasma chitotriosidase activity. A novel hallmark of Gaucher disease
    • Hollak CE, vanWeely S, van Oers MH, Aerts JM. Marked elevation of plasma chitotriosidase activity. A novel hallmark of Gaucher disease. J Clin Invest. 1994; 93: 1288-1292.
    • (1994) J Clin Invest , vol.93 , pp. 1288-1292
    • Hollak, C.E.1    VanWeely, S.2    Van Oers, M.H.3    Aerts, J.M.4
  • 26
    • 41049102218 scopus 로고    scopus 로고
    • Early therapeutic intervention in females with Fabry disease?
    • DOI 10.1111/j.1651-2227.2008.00649.x
    • Hughes DA. Early therapeutic intervention in females with Fabry disease? Acta Paediatr. 2008; 97 (Suppl 457): 41-47. (Pubitemid 351421292)
    • (2008) Acta Paediatrica, International Journal of Paediatrics , vol.97 , Issue.SUPPL. 457 , pp. 41-47
    • Hughes, D.A.1
  • 27
    • 38749130497 scopus 로고    scopus 로고
    • Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: A randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa
    • DOI 10.1136/hrt.2006.104026
    • Hughes DA, Elliott PM, Shah J, Zuckerman J, Coghlan G, Brookes J et al. Effects of enzyme replacement therapy on the cardiomyopathy ofAnderson-Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa. Heart. 2008; 94: 153-158. (Pubitemid 351211719)
    • (2008) Heart , vol.94 , Issue.2 , pp. 153-158
    • Hughes, D.A.1    Elliott, P.M.2    Shah, J.3    Zuckerman, J.4    Coghlan, G.5    Brookes, J.6    Mehta, A.B.7
  • 28
    • 54449100137 scopus 로고    scopus 로고
    • Acase of Fabry disease with central nervous system (CNS) demyelinating lesions: A double trouble?
    • Invernizzi P, Bonometti M, Turri E, Benedetti M, Salviati A.Acase of Fabry disease with central nervous system (CNS) demyelinating lesions: a double trouble? Mult Scler. 2008; 14: 1003-1006.
    • (2008) Mult Scler , vol.14 , pp. 1003-1006
    • Invernizzi, P.1    Bonometti, M.2    Turri, E.3    Benedetti, M.4    Salviati, A.5
  • 29
    • 34548145120 scopus 로고    scopus 로고
    • Mutant α-galactosidase A enzymes identified in Fabry disease patients with residual enzyme activity: Biochemical characterization and restoration of normal intracellular processing by 1-deoxygalactonojirimycin
    • DOI 10.1042/BJ20070479
    • Ishii S, Chang HH, Kawasaki K, Yasuda K, Wu HL, Garman SC et al. Mutant α-galactosidase A enzymes identified in Fabry disease patients with residual enzyme activity: biochemical characterization and restoration of normal intracellular processing by 1-deoxygalactonojirimycin. Biochem J. 2007; 406: 285-295. (Pubitemid 47310959)
    • (2007) Biochemical Journal , vol.406 , Issue.2 , pp. 285-295
    • Ishii, S.1    Chang, H.-H.2    Kawasaki, K.3    Yasuda, K.4    Wu, H.-L.5    Garman, S.C.6    Fan, J.-Q.7
  • 30
    • 33845698287 scopus 로고    scopus 로고
    • Myeloperoxidase predicts risk of vasculopathic events in hemizgygous males with Fabry disease
    • DOI 10.1212/01.wnl.0000247278.88077.09, PII 0000611420061212000031
    • Kaneski CR, Moore DF, Ries M, Zirzow GC, Schiffmann R. Myeloperoxidase predicts risk of vasculopathic events in hemizgygous males with Fabry disease. Neurology. 2006; 67: 2045-2047. (Pubitemid 44967388)
    • (2006) Neurology , vol.67 , Issue.11 , pp. 2045-2047
    • Kaneski, C.R.1    Moore, D.F.2    Ries, M.3    Zirzow, G.C.4    Schiffmann, R.5
  • 31
    • 44449096508 scopus 로고    scopus 로고
    • Replacement of α-galactosidase A in Fabry disease: Effect on fibroblast cultures compared with biopsied tissues of treated patients
    • Keslova-Veselikova J, Hulkova H, Dobrovolny R, Asfaw B, Poupetova H, Berna L et al. Replacement of α-galactosidase A in Fabry disease: effect on fibroblast cultures compared with biopsied tissues of treated patients.Virchows Arch. 2008; 452: 651-665.
    • (2008) Virchows Arch , vol.452 , pp. 651-665
    • Keslova-Veselikova, J.1    Hulkova, H.2    Dobrovolny, R.3    Asfaw, B.4    Poupetova, H.5    Berna, L.6
  • 34
    • 33747191418 scopus 로고    scopus 로고
    • Hyperhidrosis: A new and often early symptom in Fabry disease. International experience and data from the Fabry Outcome Survey
    • Lidove O, Ramaswami U, Jaussaud R, Barbey F, Maisonobe T, Caillaud C et al. Hyperhidrosis: a new and often early symptom in Fabry disease. International experience and data from the Fabry Outcome Survey. Int J Clin Pract. 2006; 60: 1053-1059.
    • (2006) Int J Clin Pract , vol.60 , pp. 1053-1059
    • Lidove, O.1    Ramaswami, U.2    Jaussaud, R.3    Barbey, F.4    Maisonobe, T.5    Caillaud, C.6
  • 35
    • 33847050214 scopus 로고    scopus 로고
    • Clinical results of enzyme replacement therapy in Fabry disease: A comprehensive review of literature
    • Lidove O, Joly D, Barbey F, Bekri S, Alexandra JF, Peigne V et al. Clinical results of enzyme replacement therapy in Fabry disease: a comprehensive review of literature. Int J Clin Pract. 2007; 61: 293-302.
    • (2007) Int J Clin Pract , vol.61 , pp. 293-302
    • Lidove, O.1    Joly, D.2    Barbey, F.3    Bekri, S.4    Alexandra, J.F.5    Peigne, V.6
  • 36
    • 4644316602 scopus 로고    scopus 로고
    • Enzyme therapy for Fabry disease: Neutralizing antibodies toward agalsidase alpha and beta
    • DOI 10.1111/j.1523-1755.2004.00924.x
    • Linthorst GE, Hollak CE, Donker-Koopman WE, Strijland A, Aerts JM. Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alfa and beta. Kidney Int. 2004; 66: 1589-1595. (Pubitemid 39298394)
    • (2004) Kidney International , vol.66 , Issue.4 , pp. 1589-1595
    • Linthorst, G.E.1    Hollak, C.E.M.2    Donker-Koopman, W.E.3    Strijland, A.4    Aerts, J.M.F.G.5
  • 43
    • 34848895305 scopus 로고    scopus 로고
    • Is there a neutralizing effect of antibodies against agalsidase alfa and agalsidase beta?
    • Mengel E, Baron K, Kalkum G, Kampmann C, Beck M. Is there a neutralizing effect of antibodies against agalsidase alfa and agalsidase beta? Acta Paediatr. 2007; 96 (Suppl 455): 108.
    • (2007) Acta Paediatr , vol.96 , Issue.SUPPL. 455 , pp. 108
    • Mengel, E.1    Baron, K.2    Kalkum, G.3    Kampmann, C.4    Beck, M.5
  • 44
    • 33646845939 scopus 로고    scopus 로고
    • Intravenous enzyme replacement therapy: Better in home or hospital?
    • Milligan A, Hughes D, Goodwin S, Richfield L, Mehta A. Intravenous enzyme replacement therapy: better in home or hospital? Br J Nurs. 2006; 15: 330-333.
    • (2006) Br J Nurs , vol.15 , pp. 330-333
    • Milligan, A.1    Hughes, D.2    Goodwin, S.3    Richfield, L.4    Mehta, A.5
  • 46
    • 33846896857 scopus 로고    scopus 로고
    • Cellular and tissue distribution of intravenously administered agalsidase alfa
    • DOI 10.1016/j.ymgme.2006.11.008, PII S1096719206003611
    • Murray GJ, Anver MR, Kennedy MA, Quirk JM, Schiffmann R. Cellular and tissue distribution of intravenously administered agalsidase alfa. Mol Genet Metab. 2007; 90: 307-312. (Pubitemid 46241908)
    • (2007) Molecular Genetics and Metabolism , vol.90 , Issue.3 SPEC. ISS. , pp. 307-312
    • Murray, G.J.1    Anver, M.R.2    Kennedy, M.A.3    Quirk, J.M.4    Schiffmann, R.5
  • 47
    • 0029023150 scopus 로고
    • An atypical variant of Fabry's disease in men with left ventricular hypertrophy
    • Nakao S, Takenaka T, Maeda M, Kodama C, Tanaka A, Tahara M et al. An atypical variant of Fabry's disease in men with left ventricular hypertrophy. N Engl J Med. 1995; 333: 288-293.
    • (1995) N Engl J Med , vol.333 , pp. 288-293
    • Nakao, S.1    Takenaka, T.2    Maeda, M.3    Kodama, C.4    Tanaka, A.5    Tahara, M.6
  • 49
    • 84860145185 scopus 로고    scopus 로고
    • Available from
    • National Institute for Health and Clinical Excellence. Appraising orphan drugs.Available from: http://www. nice.org.uk/niceMedia/pdf/smt/120705item4.pdf; 2008.
    • (2008) Appraising Orphan Drugs
  • 50
    • 34848819423 scopus 로고    scopus 로고
    • Influence of antibody formation on reduction of globotriaosylceramide (GL-3) in urine from Fabry patients during agalsidase beta therapy
    • DOI 10.1016/j.ymgme.2007.06.013, PII S1096719207002089
    • Ohashi T, Sakuma M, Kitagawa T, Suzuki K, Ishige N, Eto Y. Influence of antibody formation on reduction of globotriaosylceramide (GL-3) in urine from Fabry patients during agalsidase beta therapy. Mol Genet Metab. 2007; 92: 271-273. (Pubitemid 47513559)
    • (2007) Molecular Genetics and Metabolism , vol.92 , Issue.3 , pp. 271-273
    • Ohashi, T.1    Sakuma, M.2    Kitagawa, T.3    Suzuki, K.4    Ishige, N.5    Eto, Y.6
  • 52
    • 49549102039 scopus 로고    scopus 로고
    • Enzyme replacement therapy with agalsidase alfa in a cohort of Italian patients with Anderson-Fabry disease: Testing the effects with the Mainz Severity Score Index
    • Parini R, Rigoldi M, Santus F, Furlan F, De Lorenzo P, Valsecchi G et al. Enzyme replacement therapy with agalsidase alfa in a cohort of Italian patients with Anderson-Fabry disease: testing the effects with the Mainz Severity Score Index. Clin Genet. 2008; 74: 260-266.
    • (2008) Clin Genet , vol.74 , pp. 260-266
    • Parini, R.1    Rigoldi, M.2    Santus, F.3    Furlan, F.4    De Lorenzo, P.5    Valsecchi, G.6
  • 54
    • 41049103114 scopus 로고    scopus 로고
    • Fabry disease during childhood: Clinical manifestations and treatment with agalsidase alfa
    • DOI 10.1111/j.1651-2227.2008.00658.x
    • Ramaswami U. Fabry disease during childhood: clinical manifestations and treatment with agalsidase alfa. Acta Paediatr. 2008; 97 (Suppl 457): 38-40. (Pubitemid 351421299)
    • (2008) Acta Paediatrica, International Journal of Paediatrics , vol.97 , Issue.SUPPL. 457 , pp. 38-40
    • Ramaswami, U.1
  • 58
    • 0037177166 scopus 로고    scopus 로고
    • Prevalence of Anderson-Fabry disease in male patients with late onset hypertrophic cardiomyopathy
    • DOI 10.1161/01.CIR.0000012626.81324.38
    • Sachdev B, Takenaka T, Teraguchi H, Tei C, Lee P, McKenna WJ et al. Prevalence of Anderson-Fabry disease in male patients with late onset hypertrophic cardiomyopathy. Circulation. 2002; 105: 1407-1411. (Pubitemid 34263268)
    • (2002) Circulation , vol.105 , Issue.12 , pp. 1407-1411
    • Sachdev, B.1    Takenaka, T.2    Teraguchi, H.3    Tei, C.4    Lee, P.5    McKenna, W.J.6    Elliott, P.M.7
  • 59
    • 33947327728 scopus 로고    scopus 로고
    • Fabry disease mimicking multiple sclerosis
    • DOI 10.1016/j.clineuro.2006.12.006, PII S0303846706002095
    • Saip S, Uluduz D, ErkolG. Fabry disease mimicking multiple sclerosis. Clin Neurol Neurosurg. 2007; 109: 361-363. (Pubitemid 46441632)
    • (2007) Clinical Neurology and Neurosurgery , vol.109 , Issue.4 , pp. 361-363
    • Saip, S.1    Uluduz, D.2    Erkol, G.3
  • 60
    • 16844370666 scopus 로고    scopus 로고
    • Genotype and phenotype in Fabry disease: Analysis of the Fabry Outcome Survey
    • Schaefer E, Mehta A, Gal A. Genotype and phenotype in Fabry disease: analysis of the Fabry Outcome Survey. Acta Paediatr. 2005; 94 (Suppl 451): 87-92.
    • (2005) Acta Paediatr , vol.94 , Issue.SUPPL. 451 , pp. 87-92
    • Schaefer, E.1    Mehta, A.2    Gal, A.3
  • 61
    • 33644830752 scopus 로고    scopus 로고
    • Pathological findings in a patient with Fabry disease who died after 2.5 years of enzyme replacement
    • DOI 10.1007/s00428-005-0089-x
    • Schiffmann R, Rapkiewicz A, Abu-Asab M, Ries M, Askari H, Tsokos M et al. Pathological findings in a patient with Fabry disease who died after 2.5 years of enzyme replacement. Virchows Arch. 2006a; 448: 337-343. (Pubitemid 43357651)
    • (2006) Virchows Archiv , vol.448 , Issue.3 , pp. 337-343
    • Schiffmann, R.1    Rapkiewicz, A.2    Abu-Asab, M.3    Ries, M.4    Askari, K.5    Tsokos, M.6    Quezado, M.7
  • 62
    • 31544456336 scopus 로고    scopus 로고
    • Long-term therapy with agalsidase alfa for Fabry disease: Safety and effects on renal function in a home infusion setting
    • DOI 10.1093/ndt/gfi152
    • Schiffmann R, Ries M, Timmons M, Flaherty JT, Brady RO. Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting. Nephrol Dial Transplant. 2006b; 21: 345-354. (Pubitemid 43159987)
    • (2006) Nephrology Dialysis Transplantation , vol.21 , Issue.2 , pp. 345-354
    • Schiffmann, R.1    Ries, M.2    Timmons, M.3    Flaherty, J.T.4    Brady, R.O.5
  • 63
    • 34248138939 scopus 로고    scopus 로고
    • Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing
    • DOI 10.1681/ASN.2006111263
    • Schiffmann R, Askari H, Timmons M, Robinson C, Benko W, Brady RO et al. Weekly enzyme replacement therapy mayslow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing. J Am Soc Nephrol. 2007; 18: 1576-1583. (Pubitemid 46717525)
    • (2007) Journal of the American Society of Nephrology , vol.18 , Issue.5 , pp. 1576-1583
    • Schiffmann, R.1    Askari, H.2    Timmons, M.3    Robinson, C.4    Benko, W.5    Brady, R.O.6    Ries, M.7
  • 65
    • 36148980467 scopus 로고    scopus 로고
    • Chronic meningitis and lacunar stroke in Fabry disease [3]
    • DOI 10.1007/s00415-007-0533-8
    • Schreiber W, Udvardi A, Kristoferitsch W. Chronic meningitis and lacunar stroke in Fabry disease. J Neurol. 2007; 254: 1447-1449. (Pubitemid 350112316)
    • (2007) Journal of Neurology , vol.254 , Issue.10 , pp. 1447-1449
    • Schreiber, W.1    Udvardi, A.2    Kristoferitsch, W.3
  • 68
  • 70
    • 39549116083 scopus 로고    scopus 로고
    • Treatment of Fabry disease: Outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg
    • Vedder AC, Linthorst GE, Houge G, Groener JE, Ormel EE, Bouma BJ et al. Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. PLoS ONE. 2007a; 2: 598.
    • (2007) PLoS ONE , vol.2 , pp. 598
    • Vedder, A.C.1    Linthorst, G.E.2    Houge, G.3    Groener, J.E.4    Ormel, E.E.5    Bouma, B.J.6
  • 72
    • 44949248124 scopus 로고    scopus 로고
    • Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3
    • Vedder AC, Breunig F, Donker-Koopman WE, Mills K, Young E, Winchester B et al. Treatment of Fabry disease with different dosing regimens of agalsidase: effects on antibody formation and GL-3. Mol Genet Metab. 2008; 94: 319-325.
    • (2008) Mol Genet Metab , vol.94 , pp. 319-325
    • Vedder, A.C.1    Breunig, F.2    Donker-Koopman, W.E.3    Mills, K.4    Young, E.5    Winchester, B.6
  • 76
    • 0036303882 scopus 로고    scopus 로고
    • Lysosomal disorders
    • DOI 10.1053/siny.2001.0088
    • Wraith JE. Lysosomal disorders. Semin Neonatol. 2002; 7: 75-83. (Pubitemid 34742221)
    • (2002) Seminars in Neonatology , vol.7 , Issue.1 , pp. 75-83
    • Wraith, J.E.1
  • 77
    • 16844377475 scopus 로고    scopus 로고
    • Is globotriaosylceramide a useful biomarker in Fabry disease?
    • Young E, Mills K, Morris P, Vellodi A, Lee P, Waldek S et al. Is globotriaosylceramide a useful biomarker in Fabry disease? Acta Paediatr. 2005; 94 (Suppl 447): 51-54.
    • (2005) Acta Paediatr , vol.94 , Issue.SUPPL. 447 , pp. 51-54
    • Young, E.1    Mills, K.2    Morris, P.3    Vellodi, A.4    Lee, P.5    Waldek, S.6
  • 81
    • 33847213924 scopus 로고    scopus 로고
    • Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of α-galactosidase A
    • Ziegler RJ, Cherry M, Barbon CM, Li C, Bercury SD, Armentano D et al. Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of α-galactosidase A. Mol Ther. 2007; 15: 492-500.
    • (2007) Mol Ther , vol.15 , pp. 492-500
    • Ziegler, R.J.1    Cherry, M.2    Barbon, C.M.3    Li, C.4    Bercury, S.D.5    Armentano, D.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.