-
1
-
-
0024524979
-
Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency
-
10.1172/JCI114010 1:CAS:528:DyaL1MXitFSgsLo%3D 2495302
-
E.H. Birkenmeier M.T. Davisson W.G. Beamer R.E. Ganschow C.A. Vogler B. Gwynn K.A. Lyford L.M. Maltais C.J. Wawrzyniak 1989 Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency J. Clin. Invest. 83 1258 1266 10.1172/JCI114010 1:CAS:528:DyaL1MXitFSgsLo%3D 2495302
-
(1989)
J. Clin. Invest.
, vol.83
, pp. 1258-1266
-
-
Birkenmeier, E.H.1
Davisson, M.T.2
Beamer, W.G.3
Ganschow, R.E.4
Vogler, C.A.5
Gwynn, B.6
Lyford, K.A.7
Maltais, L.M.8
Wawrzyniak, C.J.9
-
2
-
-
0027138235
-
Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase
-
DOI 10.1073/pnas.90.24.11830
-
S.E. Braun E.L. Aronovich R.A. Anderson P.L. Crotty R.S. McIvor C.B. Whitley 1993 Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase Proc. Natl. Acad. Sci. USA 90 11830 11834 10.1073/pnas.90.24.11830 1:CAS:528:DyaK2cXht1Kgsr8%3D 8265633 (Pubitemid 24008733)
-
(1993)
Proceedings of the National Academy of Sciences of the United States of America
, vol.90
, Issue.24
, pp. 11830-11834
-
-
Braun, S.E.1
Aronovich, E.L.2
Anderson, R.A.3
Crotty, P.L.4
McIvor, R.S.5
Whitley, C.B.6
-
3
-
-
0142153023
-
Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder
-
DOI 10.1016/j.molmed.2003.08.004
-
D.A. Brooks R. Kakavanos J.J. Hopwood 2003 Significance of immune response to enzyme-replacement therapy for patients with a lysosomal storage disorder Trends Mol. Med. 9 450 453 10.1016/j.molmed.2003.08.004 1:CAS:528:DC%2BD3sXnvVKku7w%3D 14557058 (Pubitemid 37309647)
-
(2003)
Trends in Molecular Medicine
, vol.9
, Issue.10
, pp. 450-453
-
-
Brooks, D.A.1
Kakavanos, R.2
Hopwood, J.J.3
-
4
-
-
33645110474
-
Correction of Hunter syndrome in the MPS II mouse model by AAV2/8-mediated gene delivery
-
10.1093/hmg/ddl038 1:CAS:528:DC%2BD28Xis1Shs7s%3D 16505002
-
M. Cardone V.A. Polito S. Pepe L. Mann A. D'Azzo A. Auricchio A. Ballabio M.P. Cosma 2006 Correction of Hunter syndrome in the MPS II mouse model by AAV2/8-mediated gene delivery Hum. Mol. Genet. 15 1225 1236 10.1093/hmg/ddl038 1:CAS:528:DC%2BD28Xis1Shs7s%3D 16505002
-
(2006)
Hum. Mol. Genet.
, vol.15
, pp. 1225-1236
-
-
Cardone, M.1
Polito, V.A.2
Pepe, S.3
Mann, L.4
D'Azzo, A.5
Auricchio, A.6
Ballabio, A.7
Cosma, M.P.8
-
5
-
-
0030899669
-
Murine mucopolysaccharidosis type I: Targeted disruption of the murine α-L-iduronidase gene
-
DOI 10.1093/hmg/6.4.503
-
L.A. Clarke C.S. Russell S. Pownall C.L. Warrington A. Borowski J.E. Dimmick J. Toone F.R. Jirik 1997 Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene Hum. Mol. Genet. 6 503 511 10.1093/hmg/6.4.503 1:CAS:528:DyaK2sXisFOmt7s%3D 9097952 (Pubitemid 27142098)
-
(1997)
Human Molecular Genetics
, vol.6
, Issue.4
, pp. 503-511
-
-
Clarke, L.A.1
Russell, C.S.2
Pownall, S.3
Warrington, C.L.4
Borowski, A.5
Dimmick, J.E.6
Toone, J.7
Jirik, F.R.8
-
6
-
-
0029125545
-
Bone marrow transplantation in Hunter syndrome (mucopolysaccharidosis type II): Two-year follow-up of the first Italian patient and review of the literature
-
1:STN:280:DyaK28%2FktFSlsA%3D%3D 7567644
-
G.V. Coppa O. Gabrielli L. Zampini P. Pierani P.L. Giorgi A.M. Jezequel F. Orlandi R. Miniero A. Busca T. De Luca, et al. 1995 Bone marrow transplantation in Hunter syndrome (mucopolysaccharidosis type II): two-year follow-up of the first Italian patient and review of the literature Pediatr. Med. Chir. 17 227 235 1:STN:280:DyaK28%2FktFSlsA%3D%3D 7567644
-
(1995)
Pediatr. Med. Chir.
, vol.17
, pp. 227-235
-
-
Coppa, G.V.1
Gabrielli, O.2
Zampini, L.3
Pierani, P.4
Giorgi, P.L.5
Jezequel, A.M.6
Orlandi, F.7
Miniero, R.8
Busca, A.9
De Luca, T.10
-
7
-
-
0037069736
-
Uptake of recombinant iduronate-2-sulfatase into neuronal and glial cells in vitro
-
1:CAS:528:DC%2BD38XotVKqurc%3D 12393174
-
A. Daniele R. Tomanin G.R. Villani F. Zacchello M. Scarpa P. Di Natale 2002 Uptake of recombinant iduronate-2-sulfatase into neuronal and glial cells in vitro Biochim. Biophys. Acta 1588 203 209 1:CAS:528:DC%2BD38XotVKqurc%3D 12393174
-
(2002)
Biochim. Biophys. Acta
, vol.1588
, pp. 203-209
-
-
Daniele, A.1
Tomanin, R.2
Villani, G.R.3
Zacchello, F.4
Scarpa, M.5
Di Natale, P.6
-
8
-
-
54249126862
-
Gene therapy using adeno-associated virus vectors
-
10.1128/CMR.00008-08 1:CAS:528:DC%2BD1cXhtlClurfN 18854481
-
S. Daya K.I. Berns 2008 Gene therapy using adeno-associated virus vectors Clin. Microbiol. Rev. 21 583 593 10.1128/CMR.00008-08 1:CAS:528: DC%2BD1cXhtlClurfN 18854481
-
(2008)
Clin. Microbiol. Rev.
, vol.21
, pp. 583-593
-
-
Daya, S.1
Berns, K.I.2
-
9
-
-
8244241749
-
In vitro correction of iduronate-2-sulfatase deficiency by adenovirus-mediated gene transfer
-
10.1038/sj.gt.3300411 9274721
-
C. Di Francesco C. Cracco R. Tomanin L. Picci L. Ventura E. Zacchello P. Di Natale D.S. Anson J.J. Hopwood F.L. Graham, et al. 1997 In vitro correction of iduronate-2-sulfatase deficiency by adenovirus-mediated gene transfer Gene Ther. 4 442 448 10.1038/sj.gt.3300411 9274721
-
(1997)
Gene Ther.
, vol.4
, pp. 442-448
-
-
Di Francesco, C.1
Cracco, C.2
Tomanin, R.3
Picci, L.4
Ventura, L.5
Zacchello, E.6
Di Natale, P.7
Anson, D.S.8
Hopwood, J.J.9
Graham, F.L.10
-
10
-
-
0029738625
-
Targeted disruption of the arylsulfatase B gene results in mice resembling the phenotype of mucopolysaccharidosis VI
-
DOI 10.1073/pnas.93.16.8214
-
M. Evers P. Saftig P. Schmidt A. Hafner D.B. McLoghlin W. Schmahl B. Hess K. von Figura C. Peters 1996 Targeted disruption of the arylsulfatase B gene results in mice resembling the phenotype of mucopolysaccharidosis VI Proc. Natl. Acad. Sci. USA 93 8214 8219 10.1073/pnas.93.16.8214 1:CAS:528: DyaK28XkvFyltro%3D 8710849 (Pubitemid 26269536)
-
(1996)
Proceedings of the National Academy of Sciences of the United States of America
, vol.93
, Issue.16
, pp. 8214-8219
-
-
Evers, M.1
Saftig, P.2
Schmidt, P.3
Hafner, A.4
McLoghlin, D.B.5
Schmahl, W.6
Hess, B.7
Von Figura, K.8
Peters, C.9
-
11
-
-
37249043762
-
The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome)
-
DOI 10.1007/s10545-007-0641-8
-
A.R. Garcia J. Pan J.C. Lamsa J. Muenzer 2007 The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome) J. Inherit. Metab. Dis. 30 924 934 10.1007/s10545-007-0641-8 1:CAS:528:DC%2BD2sXhtl2ks7nM 17876721 (Pubitemid 350263133)
-
(2007)
Journal of Inherited Metabolic Disease
, vol.30
, Issue.6
, pp. 924-934
-
-
Garcia, A.R.1
Pan, J.2
Lamsa, J.C.3
Muenzer, J.4
-
12
-
-
33645992763
-
Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype
-
10.1128/JVI.80.1.426-439.2006 1:CAS:528:DC%2BD28XjslaltbY%3D 16352567
-
D. Grimm K. Pandey H. Nakai T.A. Storm M.A. Kay 2006 Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype J. Virol. 80 426 439 10.1128/JVI.80.1.426-439.2006 1:CAS:528:DC%2BD28XjslaltbY%3D 16352567
-
(2006)
J. Virol.
, vol.80
, pp. 426-439
-
-
Grimm, D.1
Pandey, K.2
Nakai, H.3
Storm, T.A.4
Kay, M.A.5
-
13
-
-
0037448352
-
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency [1]
-
DOI 10.1056/NEJM200301163480314
-
S. Hacein-Bey-Abina C. von Kalle M. Schmidt F. Le Deist N. Wulffraat E. McIntyre I. Radford J.L. Villeval C.C. Fraser M. Cavazzana-Calvo, et al. 2003 A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency N. Engl. J. Med. 348 255 256 10.1056/NEJM200301163480314 12529469 (Pubitemid 36077924)
-
(2003)
New England Journal of Medicine
, vol.348
, Issue.3
, pp. 255-256
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
Le Deist, F.4
Wulffraat, N.5
McIntyre, E.6
Radford, I.7
Villeval, J.-L.8
Fraser, C.C.9
Cavazzana-Calvo, M.10
Fischer, A.11
-
14
-
-
0035956882
-
Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice
-
DOI 10.1073/pnas.051634498
-
S.C. Jung I.P. Han A. Limaye R. Xu M.P. Gelderman P. Zerfas K. Tirumalai G.J. Murray M.J. During R.O. Brady, et al. 2001 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 98 2676 2681 10.1073/pnas.051634498 1:CAS:528:DC%2BD3MXhslKmtr4%3D 11226298 (Pubitemid 32209541)
-
(2001)
Proceedings of the National Academy of Sciences of the United States of America
, vol.98
, Issue.5
, pp. 2676-2681
-
-
Jung, S.-C.1
Han, I.P.2
Limaye, A.3
Xu, R.4
Gelderman, M.P.5
Zerfas, P.6
Tirumalai, K.7
Murray, G.J.8
During, M.J.9
Brady, R.O.10
Qasba, P.11
-
15
-
-
0037928717
-
Angiogenesis and white blood cell proliferation induced in mice by injection of a prolactin-expressing plasmid into muscle
-
1:CAS:528:DC%2BD3sXkvVeqsLg%3D 12803491
-
J.Y. Ko Y.L. Ahn B.N. Cho 2003 Angiogenesis and white blood cell proliferation induced in mice by injection of a prolactin-expressing plasmid into muscle Mol. Cells 15 262 270 1:CAS:528:DC%2BD3sXkvVeqsLg%3D 12803491
-
(2003)
Mol. Cells
, vol.15
, pp. 262-270
-
-
Ko, J.Y.1
Ahn, Y.L.2
Cho, B.N.3
-
16
-
-
0029837349
-
Biochemical and molecular analysis in a patient with the severe form of Hunter syndrome after bone marrow transplantation
-
DOI 10.1002/(SICI)1096-8628(19960906)64:4<531::AID-AJMG1>3.0.CO;2-S
-
P. Li J.N. Thompson G. Hug P. Huffman G. Chuck 1996 Biochemical and molecular analysis in a patient with the severe form of Hunter syndrome after bone marrow transplantation Am. J. Med. Genet. 64 531 535 10.1002/(SICI)1096- 8628(19960906)64:4<531::AID-AJMG1>3.0.CO;2-S 1:STN:280: DyaK2s%2FisVKqsg%3D%3D 8870917 (Pubitemid 26296097)
-
(1996)
American Journal of Medical Genetics
, vol.64
, Issue.4
, pp. 531-535
-
-
Li, P.1
Thompson, J.N.2
Hug, G.3
Huffman, P.4
Chuck, G.5
-
17
-
-
0013451138
-
Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding α-N-acetylglucosaminidase
-
DOI 10.1073/pnas.96.25.14505
-
H.H. Li W.H. Yu N. Rozengurt H.Z. Zhao K.M. Lyons S. Anagnostaras M.S. Fanselow K. Suzuki M.T. Vanier E.F. Neufeld 1999 Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase Proc. Natl. Acad. Sci. USA 96 14505 14510 10.1073/pnas.96.25.14505 1:CAS:528:DyaK1MXnvFKks7w%3D 10588735 (Pubitemid 30000680)
-
(1999)
Proceedings of the National Academy of Sciences of the United States of America
, vol.96
, Issue.25
, pp. 14505-14510
-
-
Li, H.H.1
Yu, W.-H.2
Rozengurt, N.3
Zhao, H.-Z.4
Lyons, K.M.5
Anagnostaras, S.6
Fanselow, M.S.7
Suzuki, K.8
Vanier, M.T.9
Neufeld, E.F.10
-
18
-
-
0037790710
-
Molecular basis of the inflammatory response to adenovirus vectors
-
DOI 10.1038/sj.gt.3302036
-
Q. Liu D.A. Muruve 2003 Molecular basis of the inflammatory response to adenovirus vectors Gene Ther. 10 935 940 10.1038/sj.gt.3302036 1:CAS:528:DC%2BD3sXjvVagt74%3D 12756413 (Pubitemid 36722427)
-
(2003)
Gene Therapy
, vol.10
, Issue.11 SPEC.
, pp. 935-940
-
-
Liu, Q.1
Muruve, D.A.2
-
19
-
-
0346995457
-
The status of hematopoietic stem cell transplantation in lysosomal storage disease
-
DOI 10.1016/j.pediatrneurol.2003.09.003
-
J.J. Malatack D.M. Consolini E. Bayever 2003 The status of hematopoietic stem cell transplantation in lysosomal storage disease Pediatr. Neurol. 29 391 403 10.1016/j.pediatrneurol.2003.09.003 14684234 (Pubitemid 37543674)
-
(2003)
Pediatric Neurology
, vol.29
, Issue.5
, pp. 391-403
-
-
Malatack, J.J.1
Consolini, D.M.2
Bayever, E.3
-
20
-
-
38849176942
-
Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome)
-
DOI 10.1542/peds.2007-1350
-
R. Martin M. Beck C. Eng R. Giugliani P. Harmatz V. Munoz J. Muenzer 2008 Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome) Pediatrics 121 e377 386 10.1542/peds.2007-1350 18245410 (Pubitemid 351198459)
-
(2008)
Pediatrics
, vol.121
, Issue.2
-
-
Martin, R.1
Beck, M.2
Eng, C.3
Giugliani, R.4
Harmatz, P.5
Munoz, V.6
Muenzer, J.7
-
21
-
-
0036984005
-
Enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome): A preliminary report
-
10.1080/080352502762458012 1:STN:280:DC%2BD3s%2Fmt1Wntw%3D%3D
-
J. Muenzer J.C. Lamsa A. Garcia J. Dacosta J. Garcia D.A. Treco 2002 Enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome): a preliminary report Acta Paediatr. 91 98 99 10.1080/080352502762458012 1:STN:280:DC%2BD3s%2Fmt1Wntw%3D%3D
-
(2002)
Acta Paediatr.
, vol.91
, pp. 98-99
-
-
Muenzer, J.1
Lamsa, J.C.2
Garcia, A.3
Dacosta, J.4
Garcia, J.5
Treco, D.A.6
-
22
-
-
11144248909
-
The innate immune response to adenovirus vectors
-
10.1089/hum.2004.15.1157 1:CAS:528:DC%2BD2MXktVWhug%3D%3D 15684693
-
D.A. Muruve 2004 The innate immune response to adenovirus vectors Hum. Gene Ther. 15 1157 1166 10.1089/hum.2004.15.1157 1:CAS:528: DC%2BD2MXktVWhug%3D%3D 15684693
-
(2004)
Hum. Gene Ther.
, vol.15
, pp. 1157-1166
-
-
Muruve, D.A.1
-
23
-
-
0025826050
-
Lysosomal storage diseases
-
10.1146/annurev.bi.60.070191.001353 1:CAS:528:DyaK3MXmtVCmurg%3D 1883197
-
E.F. Neufeld 1991 Lysosomal storage diseases Annu. Rev. Biochem. 60 257 280 10.1146/annurev.bi.60.070191.001353 1:CAS:528:DyaK3MXmtVCmurg%3D 1883197
-
(1991)
Annu. Rev. Biochem.
, vol.60
, pp. 257-280
-
-
Neufeld, E.F.1
-
24
-
-
3242778717
-
Long-term enzymatic and phenotypic correction in the phenylketonuria mouse model by adeno-associated virus vector-mediated gene transfer
-
DOI 10.1203/01.PDR.0000132837.29067.0E
-
H.J. Oh E.S. Park S. Kang I. Jo S.C. Jung 2004 Longterm enzymatic and phenotypic correction in the phenylketonuria mouse model by adeno-associated virus vectormediated gene transfer Pediatr. Res. 56 278 284 10.1203/01.PDR. 0000132837.29067.0E 1:CAS:528:DC%2BD2cXlsV2jtbc%3D 15181195 (Pubitemid 38971824)
-
(2004)
Pediatric Research
, vol.56
, Issue.2
, pp. 278-284
-
-
Oh, H.-J.1
Park, E.-S.2
Kang, S.3
Jo, I.4
Jung, S.-C.5
-
25
-
-
12644284502
-
α-Galactosidase A deficient mice: A model of fabry disease
-
DOI 10.1073/pnas.94.6.2540
-
T. Ohshima G.J. Murray W.D. Swaim G. Longenecker J.M. Quirk C.O. Cardarelli Y. Sugimoto I. Pastan M.M. Gottesman R.O. Brady, et al. 1997 alpha-Galactosidase A deficient mice: a model of Fabry disease Proc. Natl. Acad. Sci. USA 94 2540 2544 10.1073/pnas.94.6.2540 1:CAS:528:DyaK2sXhvF2ksL8%3D 9122231 (Pubitemid 27136911)
-
(1997)
Proceedings of the National Academy of Sciences of the United States of America
, vol.94
, Issue.6
, pp. 2540-2544
-
-
Ohshima, T.1
Murray, G.J.2
Swaim, W.D.3
Longenecker, G.4
Quirk, J.M.5
Cardarelli, C.O.6
Sugimoto, Y.7
Pastan, I.8
Gottesman, M.M.9
Brady, R.O.10
Kulkarni, A.B.11
-
26
-
-
0034183048
-
Hematopoietic cell transplantation for mucopolysaccharidosis IIB (Hunter syndrome)
-
10.1038/sj.bmt.1702398 1:STN:280:DC%2BD3czptFyqtg%3D%3D 10828872
-
C. Peters W. Krivit 2000 Hematopoietic cell transplantation for mucopolysaccharidosis IIB (Hunter syndrome) Bone Marrow Transplant. 25 1097 1099 10.1038/sj.bmt.1702398 1:STN:280:DC%2BD3czptFyqtg%3D%3D 10828872
-
(2000)
Bone Marrow Transplant.
, vol.25
, pp. 1097-1099
-
-
Peters, C.1
Krivit, W.2
-
27
-
-
33750079559
-
Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors
-
DOI 10.1073/pnas.0607053103
-
W.S. Sly C. Vogler J.H. Grubb B. Levy N. Galvin Y. Tan T. Nishioka S. Tomatsu 2006 Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors Proc. Natl. Acad. Sci. USA 103 15172 15177 10.1073/pnas.0607053103 1:CAS:528: DC%2BD28XhtFais7bP 17015822 (Pubitemid 44583183)
-
(2006)
Proceedings of the National Academy of Sciences of the United States of America
, vol.103
, Issue.41
, pp. 15172-15177
-
-
Sly, W.S.1
Vogler, C.2
Grubb, J.H.3
Levy, B.4
Galvin, N.5
Tan, Y.6
Nishioka, T.7
Tomatsu, S.8
-
28
-
-
8844269115
-
Why do we need new gene therapy viral vectors? Characteristics, limitations and future perspectives of viral vector transduction
-
1:CAS:528:DC%2BD2cXhtVWitLfP 15578987
-
R. Tomanin M. Scarpa 2004 Why do we need new gene therapy viral vectors? Characteristics, limitations and future perspectives of viral vector transduction Curr. Gene Ther. 4 357 372 1:CAS:528:DC%2BD2cXhtVWitLfP 15578987
-
(2004)
Curr. Gene Ther.
, vol.4
, pp. 357-372
-
-
Tomanin, R.1
Scarpa, M.2
-
29
-
-
0346752134
-
-/-) produced by targeted disruption of the gene defective in Morquio A disease
-
DOI 10.1093/hmg/ddg366
-
S. Tomatsu K.O. Orii C. Vogler J. Nakayama B. Levy J.H. Grubb M.A. Gutierrez S. Shim S. Yamaguchi T. Nishioka, et al. 2003 Mouse model of N-acetylgalactosamine-6-sulfate sulfatase deficiency (Galns-/-) produced by targeted disruption of the gene defective in Morquio A disease Hum. Mol. Genet. 12 3349 3358 10.1093/hmg/ddg366 1:CAS:528:DC%2BD3sXpsl2mtbs%3D 14583446 (Pubitemid 37541080)
-
(2003)
Human Molecular Genetics
, vol.12
, Issue.24
, pp. 3349-3358
-
-
Tomatsu, S.1
Orii, K.O.2
Vogler, C.3
Nakayama, J.4
Levy, B.5
Grubb, J.H.6
Gutierrez, M.A.7
Shim, S.8
Yamaguchi, S.9
Nishioka, T.10
Montano, A.M.11
Noguchi, A.12
Orii, T.13
Kondo, N.14
Sly, W.S.15
-
31
-
-
0035194407
-
A fluorimetric enzyme assay for the diagnosis of MPS II (hunter disease)
-
DOI 10.1023/A:1012763026526
-
Y.V. Voznyi J.L. Keulemans O.P. van Diggelen 2001 A fluorimetric enzyme assay for the diagnosis of MPS II (Hunter disease) J. Inherit. Metab. Dis. 24 675 680 10.1023/A:1012763026526 1:CAS:528:DC%2BD38XltVSisA%3D%3D 11768586 (Pubitemid 33130088)
-
(2001)
Journal of Inherited Metabolic Disease
, vol.24
, Issue.6
, pp. 675-680
-
-
Voznyi, Y.V.1
Keulemans, J.L.M.2
Van Diggelen, O.P.3
-
32
-
-
0029965750
-
Retroviral-mediated transfer of the iduronate-2-sulfatase gene into lymphocytes for treatment of mild Hunter syndrome (mucopolysaccharidosis type II)
-
10.1089/hum.1996.7.4-537 1:CAS:528:DyaK28Xit12htrc%3D 8800749
-
C.B. Whitley R.S. McIvor E.L. Aronovich S.A. Berry B.R. Blazar S.R. Burger J.H. Kersey R.A. King A.J. Faras R.E. Latchaw, et al. 1996 Retroviral-mediated transfer of the iduronate-2-sulfatase gene into lymphocytes for treatment of mild Hunter syndrome (mucopolysaccharidosis type II) Hum. Gene Ther. 7 537 549 10.1089/hum.1996.7.4-537 1:CAS:528:DyaK28Xit12htrc%3D 8800749
-
(1996)
Hum. Gene Ther.
, vol.7
, pp. 537-549
-
-
Whitley, C.B.1
McIvor, R.S.2
Aronovich, E.L.3
Berry, S.A.4
Blazar, B.R.5
Burger, S.R.6
Kersey, J.H.7
King, R.A.8
Faras, A.J.9
Latchaw, R.E.10
|