-
1
-
-
0000869162
-
The Mucopolysaccharidoses
-
C.R. Scriver A.L. Beaudet W.S. Sly D. Valle McGraw-Hill New York
-
E.F. Neufeld, and J. Muenzer The Mucopolysaccharidoses C.R. Scriver A.L. Beaudet W.S. Sly D. Valle Metabolic and Molecular Basis of Inherited Disease 2001 McGraw-Hill New York 3421 3452
-
(2001)
Metabolic and Molecular Basis of Inherited Disease
, pp. 3421-3452
-
-
Neufeld, E.F.1
Muenzer, J.2
-
4
-
-
0015582263
-
Beta glucuronidase deficiency: Report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis
-
W.S. Sly, B.A. Quinton, W.H. McAlister, and D.L. Rimoin Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis J. Pediatr. 82 1973 249 257
-
(1973)
J. Pediatr.
, vol.82
, pp. 249-257
-
-
Sly, W.S.1
Quinton, B.A.2
McAlister, W.H.3
Rimoin, D.L.4
-
5
-
-
0024524979
-
Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency
-
E.H. Birkenmeier, M.T. Davisson, W.G. Beamer, R.E. Ganschow, C.A. Vogler, B. Gwynn, K.A. Lyford, L.M. Maltais, and C.J. Wawrzyniak Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency J. Clin. Invest. 83 1989 1258 1266
-
(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
-
6
-
-
0021213609
-
Beta-glucuronidase deficiency in a dog: A model of human mucopolysaccharidosis VII
-
M.E. Haskins, R.J. Desnick, N. DiFerrante, P.F. Jezyk, and D.F. Patterson Beta-glucuronidase deficiency in a dog: a model of human mucopolysaccharidosis VII Pediatr. Res. 18 1984 980 984
-
(1984)
Pediatr. Res.
, vol.18
, pp. 980-984
-
-
Haskins, M.E.1
Desnick, R.J.2
Diferrante, N.3
Jezyk, P.F.4
Patterson, D.F.5
-
7
-
-
0033153522
-
Molecular basis of feline beta-glucuronidase deficiency: An animal model of mucopolysaccharidosis VII
-
J.C. Fyfe, R.L. Kurzhals, M.E. Lassaline, P.S. Henthorn, P.R. Alur, P. Wang, J.H. Wolfe, U. Giger, M.E. Haskins, D.F. Patterson, H. Sun, S. Jain, and N. Yuhki Molecular basis of feline beta-glucuronidase deficiency: an animal model of mucopolysaccharidosis VII Genomics 58 1999 121 128
-
(1999)
Genomics
, vol.58
, pp. 121-128
-
-
Fyfe, J.C.1
Kurzhals, R.L.2
Lassaline, M.E.3
Henthorn, P.S.4
Alur, P.R.5
Wang, P.6
Wolfe, J.H.7
Giger, U.8
Haskins, M.E.9
Patterson, D.F.10
Sun, H.11
Jain, S.12
Yuhki, N.13
-
8
-
-
0034741927
-
Murine mucopolysaccharidosis VII: Impact of therapies on the phenotype, clinical course, and pathology in a model of a lysosomal storage disease
-
C. Vogler, J. Barker, M.S. Sands, B. Levy, N. Galvin, and W.S. Sly Murine mucopolysaccharidosis VII: impact of therapies on the phenotype, clinical course, and pathology in a model of a lysosomal storage disease Pediatr. Dev. Pathol. 4 2001 421 433
-
(2001)
Pediatr. Dev. Pathol.
, vol.4
, pp. 421-433
-
-
Vogler, C.1
Barker, J.2
Sands, M.S.3
Levy, B.4
Galvin, N.5
Sly, W.S.6
-
9
-
-
0027258694
-
Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates
-
M.S. Sands, J.E. Barker, C.A. Vogler, B. Levy, B. Gwynn, N. Galvin, W.S. Sly, and E.H. Birkenmeier Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates Lab. Invest. 68 1993 676 686
-
(1993)
Lab. Invest.
, vol.68
, pp. 676-686
-
-
Sands, M.S.1
Barker, J.E.2
Vogler, C.A.3
Levy, B.4
Gwynn, B.5
Galvin, N.6
Sly, W.S.7
Birkenmeier, E.H.8
-
10
-
-
0035283136
-
Nonablative neonatal marrow transplantation attenuates functional and physical defects of β-glucuronidase deficiency
-
B.W. Soper, M.D. Lessard, C.A. Vogler, B. Levy, W.G. Beamer, W.S. Sly, and J.E. Barker Nonablative neonatal marrow transplantation attenuates functional and physical defects of β-glucuronidase deficiency Blood 97 2001 1496 1504
-
(2001)
Blood
, vol.97
, pp. 1496-1504
-
-
Soper, B.W.1
Lessard, M.D.2
Vogler, C.A.3
Levy, B.4
Beamer, W.G.5
Sly, W.S.6
Barker, J.E.7
-
11
-
-
0031907997
-
Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation
-
Y. Yamada, K. Kato, K. Sukegawa, S. Tomatsu, S. Fukuda, S. Emura, S. Kojima, T. Matsuyama, W.S. Sly, N. Kondo, and T. Orii Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation Bone Marrow Transplant. 21 1998 629 634
-
(1998)
Bone Marrow Transplant.
, vol.21
, pp. 629-634
-
-
Yamada, Y.1
Kato, K.2
Sukegawa, K.3
Tomatsu, S.4
Fukuda, S.5
Emura, S.6
Kojima, S.7
Matsuyama, T.8
Sly, W.S.9
Kondo, N.10
Orii, T.11
-
12
-
-
2342535103
-
Cord-blood transplants from unrelated donors in patients with Hurler's syndrome
-
S.L. Staba, M.L. Escolar, M. Poe, Y. Kim, P.L. Martin, P. Szabolcs, J. Allison-Thacker, S. Wood, D.A. Wenger, P. Rubinstein, J.J. Hopwood, W. Krivit, and L. Kurtzberg Cord-blood transplants from unrelated donors in patients with Hurler's syndrome N. Engl. J. Med. 350 2004 1960 1969
-
(2004)
N. Engl. J. Med.
, vol.350
, pp. 1960-1969
-
-
Staba, S.L.1
Escolar, M.L.2
Poe, M.3
Kim, Y.4
Martin, P.L.5
Szabolcs, P.6
Allison-Thacker, J.7
Wood, S.8
Wenger, D.A.9
Rubinstein, P.10
Hopwood, J.J.11
Krivit, W.12
Kurtzberg, L.13
-
13
-
-
0026637316
-
Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors
-
S. Kornfeld Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors Annu. Rev. Biochem. 61 1992 307 330
-
(1992)
Annu. Rev. Biochem.
, vol.61
, pp. 307-330
-
-
Kornfeld, S.1
-
14
-
-
2942588994
-
Enzyme replacement and enhancement therapies for lysosomal diseases
-
R.J. Desnick Enzyme replacement and enhancement therapies for lysosomal diseases J. Inherit. Metab. Dis. 27 2004 385 410
-
(2004)
J. Inherit. Metab. Dis.
, vol.27
, pp. 385-410
-
-
Desnick, R.J.1
-
15
-
-
0030898926
-
Murine mucopolysaccharidosis type VII: Long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation
-
M.S. Sands, C.A. Vogler, A. Torrey, B. Levy, B. Gwynn, J. Grubb, W.S. Sly, and E.H. Birkenmeier Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation J. Clin. Invest. 99 1997 1596 1605
-
(1997)
J. Clin. Invest.
, vol.99
, pp. 1596-1605
-
-
Sands, M.S.1
Vogler, C.A.2
Torrey, A.3
Levy, B.4
Gwynn, B.5
Grubb, J.6
Sly, W.S.7
Birkenmeier, E.H.8
-
16
-
-
0035905889
-
Enzyme-replacement therapy in mucopolysaccharidosis I
-
E.D. Kakkis, J. Muenzer, G.E. Tiller, L. Waber, J. Belmont, M. Passage, B. Izykowski, J. Phillips, R. Doroshow, I. Walot, R. Hoft, and E.F. Neufeld Enzyme-replacement therapy in mucopolysaccharidosis I N. Engl. J. Med. 344 2001 182 188
-
(2001)
N. Engl. J. Med.
, vol.344
, pp. 182-188
-
-
Kakkis, E.D.1
Muenzer, J.2
Tiller, G.E.3
Waber, L.4
Belmont, J.5
Passage, M.6
Izykowski, B.7
Phillips, J.8
Doroshow, R.9
Walot, I.10
Hoft, R.11
Neufeld, E.F.12
-
17
-
-
0042526080
-
Gene therapy progress and prospects: Gene therapy of lysosomal storage disorders
-
S.H. Cheng, and A.E. Smith Gene therapy progress and prospects: gene therapy of lysosomal storage disorders Gene Ther. 10 2003 1275 1281
-
(2003)
Gene Ther.
, vol.10
, pp. 1275-1281
-
-
Cheng, S.H.1
Smith, A.E.2
-
18
-
-
3042731837
-
+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease
-
+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease Mol. Ther. 9 2004 856 865
-
(2004)
Mol. Ther.
, vol.9
, pp. 856-865
-
-
Hofling, A.A.1
Devine, S.2
Vogler, C.3
Sands, M.S.4
-
19
-
-
0027178735
-
Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells
-
V. Marechal, N. Naffakh, O. Danos, and J.M. Heard Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells Blood 82 1993 1358 1365
-
(1993)
Blood
, vol.82
, pp. 1358-1365
-
-
Marechal, V.1
Naffakh, N.2
Danos, O.3
Heard, J.M.4
-
20
-
-
0033559722
-
Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-l-iduronidase deficiency
-
C. Lutzko, S. Kruth, A.C. Abrams-Ogg, K. Lau, L. Li, B.R. Clark, C. Ruedy, S. Nanji, R. Foster, D. Kohn, R. Shull, and I.D. Dube Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-l-iduronidase deficiency Blood 93 1999 1895 1905
-
(1999)
Blood
, vol.93
, pp. 1895-1905
-
-
Lutzko, C.1
Kruth, S.2
Abrams-Ogg, A.C.3
Lau, K.4
Li, L.5
Clark, B.R.6
Ruedy, C.7
Nanji, S.8
Foster, R.9
Kohn, D.10
Shull, R.11
Dube, I.D.12
-
21
-
-
0032922212
-
Autologous transplantation of retrovirally transduced bone marrow or neonatal blood cells into cats can lead to long-term engraftment in the absence of myeloablation
-
C.M. Simonaro, M.E. Haskins, J.L. Abkowitz, D.A. Brooks, J.J. Hopwood, J. Zhang, and E.H. Schuchman Autologous transplantation of retrovirally transduced bone marrow or neonatal blood cells into cats can lead to long-term engraftment in the absence of myeloablation Gene Ther. 6 1999 107 113
-
(1999)
Gene Ther.
, vol.6
, pp. 107-113
-
-
Simonaro, C.M.1
Haskins, M.E.2
Abkowitz, J.L.3
Brooks, D.A.4
Hopwood, J.J.5
Zhang, J.6
Schuchman, E.H.7
-
22
-
-
0033515007
-
Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease
-
T.M. Daly, C. Vogler, B. Levy, M.E. Haskins, and M.S. Sands Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease Proc. Natl. Acad. Sci. 96 1999 2296 2300
-
(1999)
Proc. Natl. Acad. Sci.
, vol.96
, pp. 2296-2300
-
-
Daly, T.M.1
Vogler, C.2
Levy, B.3
Haskins, M.E.4
Sands, M.S.5
-
23
-
-
4444229470
-
Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse
-
T.J. Sferra, K. Backstrom, C. Wang, R. Rennard, M. Miller, and Y. Hu Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse Mol. Ther. 10 2004 478 491
-
(2004)
Mol. Ther.
, vol.10
, pp. 478-491
-
-
Sferra, T.J.1
Backstrom, K.2
Wang, C.3
Rennard, R.4
Miller, M.5
Hu, Y.6
-
24
-
-
0037344452
-
Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII
-
Y. Kamata, A. Tanabe, A. Kanaji, M. Kosuga, Y. Fukuhara, X.K. Li, S. Suzuki, M. Yamada, N. Azuma, and T. Okuyama Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII Gene Ther. 10 2003 406 414
-
(2003)
Gene Ther.
, vol.10
, pp. 406-414
-
-
Kamata, Y.1
Tanabe, A.2
Kanaji, A.3
Kosuga, M.4
Fukuhara, Y.5
Li, X.K.6
Suzuki, S.7
Yamada, M.8
Azuma, N.9
Okuyama, T.10
-
25
-
-
0034960875
-
In vivo treatment of hemophilia a and mucopolysaccharidosis type VII using nonprimate lentiviral vectors
-
C.S. Stein, Y. Kang, S.L. Sauter, K. Townsend, P. Staber, T.A. Derksen, I. Martins, J. Qian, B.L. Davidson, and P.B. McCray In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors Mol. Ther. 3 2001 850 856
-
(2001)
Mol. Ther.
, vol.3
, pp. 850-856
-
-
Stein, C.S.1
Kang, Y.2
Sauter, S.L.3
Townsend, K.4
Staber, P.5
Derksen, T.A.6
Martins, I.7
Qian, J.8
Davidson, B.L.9
McCray, P.B.10
-
26
-
-
0036932904
-
Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy
-
L. Xu, R.L. Mango, M.S. Sands, M.E. Haskins, N.M. Ellinwood, and K.P. Ponder Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy Mol. Ther. 6 2002 745 758
-
(2002)
Mol. Ther.
, vol.6
, pp. 745-758
-
-
Xu, L.1
Mango, R.L.2
Sands, M.S.3
Haskins, M.E.4
Ellinwood, N.M.5
Ponder, K.P.6
-
27
-
-
0032033199
-
Cloning of the canine beta-glucuronidase cDNA, mutation identification in canine MPS VII, and retroviral vector-mediated correction of MPS VII cells
-
J. Ray, A. Bouvet, C. DeSanto, J.C. Fyfe, D. Xu, J.H. Wolfe, G.D. Aguirre, D.F. Patterson, M.E. Haskins, and P.S. Henthorn Cloning of the canine beta-glucuronidase cDNA, mutation identification in canine MPS VII, and retroviral vector-mediated correction of MPS VII cells Genomics 48 1998 248 253
-
(1998)
Genomics
, vol.48
, pp. 248-253
-
-
Ray, J.1
Bouvet, A.2
Desanto, C.3
Fyfe, J.C.4
Xu, D.5
Wolfe, J.H.6
Aguirre, G.D.7
Patterson, D.F.8
Haskins, M.E.9
Henthorn, P.S.10
-
28
-
-
0034567124
-
Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII
-
J.H. Wolfe, M.S. Sands, N. Harel, M.A. Weil, M.K. Parente, A.C. Polesky, J.J. Reilly, C. Hasson, S. Weimelt, and M.E. Haskins Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII Mol. Ther. 2 2000 552 561
-
(2000)
Mol. Ther.
, vol.2
, pp. 552-561
-
-
Wolfe, J.H.1
Sands, M.S.2
Harel, N.3
Weil, M.A.4
Parente, M.K.5
Polesky, A.C.6
Reilly, J.J.7
Hasson, C.8
Weimelt, S.9
Haskins, M.E.10
-
29
-
-
0036792035
-
Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs
-
K.P. Ponder, J.R. Melniczek, L. Xu, M.A. Weil, T.M. O'Malley, P.A. O'Donnell, V.W. Knox, G.D. Aguirre, H. Mazrier, N.M. Ellinwood, M. Sleeper, A.M. Maguire, S.W. Volk, R.L. Mango, J. Zweigle, J.H. Wolfe, and M.E. Haskins Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs Proc. Natl. Acad. Sci. USA 99 2002 13102 13107
-
(2002)
Proc. Natl. Acad. Sci. USA
, vol.99
, pp. 13102-13107
-
-
Ponder, K.P.1
Melniczek, J.R.2
Xu, L.3
Weil, M.A.4
O'Malley, T.M.5
O'Donnell, P.A.6
Knox, V.W.7
Aguirre, G.D.8
Mazrier, H.9
Ellinwood, N.M.10
Sleeper, M.11
Maguire, A.M.12
Volk, S.W.13
Mango, R.L.14
Zweigle, J.15
Wolfe, J.H.16
Haskins, M.E.17
-
30
-
-
1942521291
-
Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs
-
R.L. Mango, L. Xu, M.S. Sands, C. Vogler, G. Seiler, T. Schwarz, M.E. Haskins, and K.P. Ponder Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs Mol. Genet. Metab. 82 2004 4 19
-
(2004)
Mol. Genet. Metab.
, vol.82
, pp. 4-19
-
-
Mango, R.L.1
Xu, L.2
Sands, M.S.3
Vogler, C.4
Seiler, G.5
Schwarz, T.6
Haskins, M.E.7
Ponder, K.P.8
-
31
-
-
4143134061
-
Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII
-
M.M. Sleeper, B. Fornasari, N.M. Ellinwood, M.A. Weil, J. Melniczek, T.M. O'Malley, C.D. Sammarco, L. Xu, K.P. Ponder, and M.E. Haskins Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII Circulation 110 2004 815 820
-
(2004)
Circulation
, vol.110
, pp. 815-820
-
-
Sleeper, M.M.1
Fornasari, B.2
Ellinwood, N.M.3
Weil, M.A.4
Melniczek, J.5
O'Malley, T.M.6
Sammarco, C.D.7
Xu, L.8
Ponder, K.P.9
Haskins, M.E.10
-
32
-
-
0036168867
-
Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs
-
L. Xu, M.E. Haskins, J.R. Melniczek, C. Gao, M.A. Weil, T.M. O'Malley, P.A. O'Donnell, H. Mazrier, N.M. Ellinwood, J. Zweigle, J.H. Wolfe, and K.P. Ponder Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs Mol. Ther. 5 2002 141 153
-
(2002)
Mol. Ther.
, vol.5
, pp. 141-153
-
-
Xu, L.1
Haskins, M.E.2
Melniczek, J.R.3
Gao, C.4
Weil, M.A.5
O'Malley, T.M.6
O'Donnell, P.A.7
Mazrier, H.8
Ellinwood, N.M.9
Zweigle, J.10
Wolfe, J.H.11
Ponder, K.P.12
-
33
-
-
3042815635
-
In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice
-
L. Xu, T. O'Malley, M.S. Sands, B. Wang, T. Myerose, M.E. Haskins, and K.P. Ponder In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice Mol. Ther. 10 2004 37 44
-
(2004)
Mol. Ther.
, vol.10
, pp. 37-44
-
-
Xu, L.1
O'Malley, T.2
Sands, M.S.3
Wang, B.4
Myerose, T.5
Haskins, M.E.6
Ponder, K.P.7
-
34
-
-
0001351056
-
Murine mucopolysaccharidosis type VII: A model system for somatic gene therapy of the central nervous system
-
P. Lowenstein L. Enquist Essex Wiley
-
J. Wolfe, and M.S. Sands Murine mucopolysaccharidosis type VII: a model system for somatic gene therapy of the central nervous system P. Lowenstein L. Enquist Gene Transfer into Neurones, towards Gene Therapy of Neurological Disorders 1996 Essex Wiley 263 274
-
(1996)
Gene Transfer into Neurones, Towards Gene Therapy of Neurological Disorders
, pp. 263-274
-
-
Wolfe, J.1
Sands, M.S.2
-
35
-
-
0031055741
-
Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy
-
M. Le, T. Okuyama, S.R. Cai, S.C. Kennedy, W.M. Bowling, M.W. Flye, and K.P. Ponder Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy Blood 89 1997 1254 1259
-
(1997)
Blood
, vol.89
, pp. 1254-1259
-
-
Le, M.1
Okuyama, T.2
Cai, S.R.3
Kennedy, S.C.4
Bowling, W.M.5
Flye, M.W.6
Ponder, K.P.7
-
36
-
-
0042389570
-
The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells
-
D.L. Haas, C. Lutzko, A.C. Logan, G.J. Cho, D. Skelton, X. Jin Yu, K.A. Pepper, and D.B. Kohn The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells J. Virol. 77 2003 9439 9450
-
(2003)
J. Virol.
, vol.77
, pp. 9439-9450
-
-
Haas, D.L.1
Lutzko, C.2
Logan, A.C.3
Cho, G.J.4
Skelton, D.5
Jin Yu, X.6
Pepper, K.A.7
Kohn, D.B.8
-
37
-
-
4344630950
-
Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier
-
A. Urayama, J.H. Grubb, W.S. Sly, and W.A. Banks Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier Proc. Natl. Acad. Sci. USA 101 2004 12658 12663
-
(2004)
Proc. Natl. Acad. Sci. USA
, vol.101
, pp. 12658-12663
-
-
Urayama, A.1
Grubb, J.H.2
Sly, W.S.3
Banks, W.A.4
-
38
-
-
4043174204
-
Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB
-
Y. Zheng, S. Ryazantsev, K. Ohmi, H.Z. Zhao, N. Rozengurt, D.B. Kohn, and E.F. Neufeld Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB Mol. Genet. Metab. 82 2004 286 295
-
(2004)
Mol. Genet. Metab.
, vol.82
, pp. 286-295
-
-
Zheng, Y.1
Ryazantsev, S.2
Ohmi, K.3
Zhao, H.Z.4
Rozengurt, N.5
Kohn, D.B.6
Neufeld, E.F.7
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