|
|
 Previous Article | Table of Contents | Next Article 
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 JM Heard
Laboratoire Retrovirus et Transfert Genetique, Institut Pasteur, URA 1157,
CNRS, Paris, France.
Mice homozygous for the gusmps allele lack beta-glucuronidase activity and
provide a useful model for human Mucopolysaccharidosis type VII (MPS VII),
also known as Sly syndrome. Bone marrow (BM) transplantation was shown to
correct the metabolic defect and to increase the life span of diseased
animals. We have used this murine model in a preclinical study aimed at
evaluating whether the techniques currently available for gene transfer
into large mammalian and human BM cells will provide efficient enzyme
replacement therapy in MPS patients. Autologous BM was transplanted into
deficient mice after retrovirus-mediated transfer of the human
beta-glucuronidase cDNA. Conditioning of recipients was performed by a
single sublethal irradiation of 4.5 Gy, giving rise to low donor
engraftment. In recipient mice analyzed until 145 days after gene transfer,
the percentage of genetically modified hematopoietic cells was less than
5%. Nevertheless, beta-glucuronidase enzyme activity was detectable in
various organs, including the brain, and disappearance of lysosomal storage
was obvious in the liver and spleen. These results show that the autologous
transplantation of genetically engineered BM cells could be beneficial in
MPS patients.
Volume 82,
Issue 4,
pp. 1358-1365,
08/15/1993
Copyright © 1993 by The American Society of Hematology
 CiteULike  Connotea  Del.icio.us  Digg  Reddit  Technorati What's this?
This article has been cited by other articles:
|
|
|
|
 
A. W. Nienhuis
Development of gene therapy for blood disorders
Blood,
May 1, 2008;
111(9):
4431 - 4444.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
S. Tomatsu, M. Gutierrez, T. Nishioka, M. Yamada, M. Yamada, Y. Tosaka, J. H. Grubb, A. M. Montano, M. B. Vieira, G. G. Trandafirescu, et al.
Development of MPS IVA mouse (Galnstm(hC79S{middle dot}mC76S)slu) tolerant to human N-acetylgalactosamine-6-sulfate sulfatase
Hum. Mol. Genet.,
November 15, 2005;
14(22):
3321 - 3335.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
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,
August 17, 2004;
110(7):
815 - 820.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
S. Tomatsu, K. O. Orii, C. Vogler, J. H. Grubb, E. M. Snella, M. Gutierrez, T. Dieter, C. C. Holden, K. Sukegawa, T. Orii, et al.
Production of MPS VII mouse (Gustm(hE540A{middle dot}mE536A)Sly) doubly tolerant to human and mouse {beta}-glucuronidase
Hum. Mol. Genet.,
May 1, 2003;
12(9):
961 - 973.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
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, et al.
From the Cover: Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs
PNAS,
October 1, 2002;
99(20):
13102 - 13107.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
W. S. Sly, C. Vogler, J. H. Grubb, M. Zhou, J. Jiang, X. Y. Zhou, S. Tomatsu, Y. Bi, and E. M. Snella
Active site mutant transgene confers tolerance to human beta -glucuronidase without affecting the phenotype of MPS VII mice
PNAS,
February 8, 2001;
(2001)
51623698.
[Abstract]
[Full Text]
|
|
|
|
|
|
|
S. Marathe, S. R.P. Miranda, C. Devlin, A. Johns, G. Kuriakose, K. J. Williams, E. H. Schuchman, and I. Tabas
Creation of a mouse model for non-neurological (type B) Niemann-Pick disease by stable, low level expression of lysosomal sphingomyelinase in the absence of secretory sphingomyelinase: relationship between brain intra-lysosomal enzyme activity and central nervous system function
Hum. Mol. Genet.,
August 12, 2000;
9(13):
1967 - 1976.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
B. J. Freeman, M. S. Roberts, C. A. Vogler, A. Nicholes, A. A. Hofling, and M. S. Sands
Behavior and Therapeutic Efficacy of beta -Glucuronidase-Positive Mononuclear Phagocytes in a Murine Model of Mucopolysaccharidosis Type VII
Blood,
September 15, 1999;
94(6):
2142 - 2150.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
C. S. Stein, A. Ghodsi, T. Derksen, and B. L. Davidson
Systemic and Central Nervous System Correction of Lysosomal Storage in Mucopolysaccharidosis Type VII Mice
J. Virol.,
April 1, 1999;
73(4):
3424 - 3429.
[Abstract]
[Full Text]
|
|
|
|
|
|
|
T. Ohashi, K. Watabe, K. Uehara, W. S. Sly, C. Vogler, and Y. Eto
Adenovirus-mediated gene transfer and expression of human beta -glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice
PNAS,
February 18, 1997;
94(4):
1287 - 1292.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
M A Bedell, D A Largaespada, N A Jenkins, and N G Copeland
Mouse models of human disease. Part II: recent progress and future directions.
Genes & Dev.,
January 1, 1997;
11(1):
11 - 43.
[PDF]
|
|
|
|
|
|
|
W. S. Sly, C. Vogler, J. H. Grubb, M. Zhou, J. Jiang, X. Y. Zhou, S. Tomatsu, Y. Bi, and E. M. Snella
Active site mutant transgene confers tolerance to human beta -glucuronidase without affecting the phenotype of MPS VII mice
PNAS,
February 27, 2001;
98(5):
2205 - 2210.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
