-
1
-
-
0035099437
-
In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: Correction of neuropathology and protection against learning impairments in affected mice
-
Consiglio, A. (and 11 others) 2001 In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice. Nature Med. 7, 310-316.
-
(2001)
Nature Med.
, vol.7
, pp. 310-316
-
-
Consiglio, A.1
-
2
-
-
0036085409
-
Novel treatment for neuronopathic lysosomal storage diseases - Cell therapy/gene therapy
-
Eto, Y. & Ohashi, T. 2002 Novel treatment for neuronopathic lysosomal storage diseases - cell therapy/gene therapy. Curr. Mol. Med. 2, 83-89.
-
(2002)
Curr. Mol. Med.
, vol.2
, pp. 83-89
-
-
Eto, Y.1
Ohashi, T.2
-
3
-
-
0032931554
-
Adenoviral gene therapy of the Tay-Sachs disease in hexosaminidase A-deficient knock-out mice
-
Guidotti, J. E., Mignon, A., Haase, G., Caillaud, C., McDonell, N., Kahn, A. & Poenaru, L. 1999 Adenoviral gene therapy of the Tay-Sachs disease in hexosaminidase A-deficient knock-out mice. Hum. Mol. Genet. 8, 831-838.
-
(1999)
Hum. Mol. Genet.
, vol.8
, pp. 831-838
-
-
Guidotti, J.E.1
Mignon, A.2
Haase, G.3
Caillaud, C.4
McDonell, N.5
Kahn, A.6
Poenaru, L.7
-
4
-
-
10544235699
-
Phenotype of arylsulfatase A deficient mice: Relationship to human metachromatic leukodystrophy
-
Hess, B. (and 12 others) 1996 Phenotype of arylsulfatase A deficient mice: relationship to human metachromatic leukodystrophy. Proc. Natl Acad. Sci. USA 93, 14 821-14 826.
-
(1996)
Proc. Natl. Acad. Sci. USA
, vol.93
, pp. 14821-14826
-
-
Hess, B.1
-
5
-
-
0035956882
-
Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice
-
Jung, S.-C. (and 10 others) 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.
-
(2001)
Proc. Natl. Acad. Sci. USA
, vol.98
, pp. 2676-2681
-
-
Jung, S.-C.1
-
6
-
-
0030787644
-
Kinetics of central nervous system microglial and macrophage engraftment: Analysis using a transgenic bone marrow transplantation model
-
Kennedy, D. W. & Abkowitz, J. L. 1997 Kinetics of central nervous system microglial and macrophage engraftment: analysis using a transgenic bone marrow transplantation model. Blood 90, 986-993.
-
(1997)
Blood
, vol.90
, pp. 986-993
-
-
Kennedy, D.W.1
Abkowitz, J.L.2
-
7
-
-
0026637316
-
Structure and function of the mannose-6-phosphate/insulin like growth factor II receptors
-
Kornfeld, S. 1992 Structure and function of the mannose-6-phosphate/insulin like growth factor II receptors. A. Rev. Biochem. 61, 307-330.
-
(1992)
A. Rev. Biochem.
, vol.61
, pp. 307-330
-
-
Kornfeld, S.1
-
8
-
-
0029898457
-
Expression of human β-hexosaminidase α-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells
-
Lacorazza, H. D., Flax, J. D., Snyder, E. Y. & Jendoubi, M. 1996 Expression of human β-hexosaminidase α-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells. Nature Med. 2, 424-429.
-
(1996)
Nature Med.
, vol.2
, pp. 424-429
-
-
Lacorazza, H.D.1
Flax, J.D.2
Snyder, E.Y.3
Jendoubi, M.4
-
9
-
-
0036566195
-
Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cells
-
Leimig, T. (and 10 others) 2002 Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cells. Blood 99, 3169-3178.
-
(2002)
Blood
, vol.99
, pp. 3169-3178
-
-
Leimig, T.1
-
10
-
-
0035989018
-
Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease
-
Li, C., Ziegler, R. J., Cherry, M., Lukason, M., Desnick, R. J., Yew, N. S. & Cheng, S. H. 2002 Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease. Mol. Ther. 5, 745-754.
-
(2002)
Mol. Ther.
, vol.5
, pp. 745-754
-
-
Li, C.1
Ziegler, R.J.2
Cherry, M.3
Lukason, M.4
Desnick, R.J.5
Yew, N.S.6
Cheng, S.H.7
-
11
-
-
0033943326
-
Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector
-
Matzner, U., Harzer, K., Learish, R. D., Barranger, J. A. & Gieselmann, V. 2000 Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector. Gene Ther. 14, 1250-1257.
-
(2000)
Gene Ther.
, vol.14
, pp. 1250-1257
-
-
Matzner, U.1
Harzer, K.2
Learish, R.D.3
Barranger, J.A.4
Gieselmann, V.5
-
12
-
-
85047699059
-
Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: Effects on visceral and nervous system disease manifestations
-
Matzner, U., Hartmann, D., Lullmann-Rauch, R., Coenen, R., Rothert, F., Mansson, J. E., Fredman, P., D'Hooge, R., De Deyn, P. P. & Gieselmann, V. 2002 Bone marrow stem cell-based gene transfer in a mouse model for metachromatic leukodystrophy: effects on visceral and nervous system disease manifestations. Gene Ther. 9, 53-63.
-
(2002)
Gene Ther.
, vol.9
, pp. 53-63
-
-
Matzner, U.1
Hartmann, D.2
Lullmann-Rauch, R.3
Coenen, R.4
Rothert, F.5
Mansson, J.E.6
Fredman, P.7
D'Hooge, R.8
De Deyn, P.P.9
Gieselmann, V.10
-
13
-
-
0033758952
-
Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease
-
Miranda, S. R., Erlich, S., Friedrich Jr, V. L., Gatt, S. & Schuchman, E. H. 2000 Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease. Gene Ther. 7, 768-776.
-
(2000)
Gene Ther.
, vol.7
, pp. 768-776
-
-
Miranda, S.R.1
Erlich, S.2
Friedrich V.L., Jr.3
Gatt, S.4
Schuchman, E.H.5
-
14
-
-
0034799137
-
Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements
-
Shen, J. S., Watabe, K., Ohashi, T. & Eto, Y. 2001 Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements. Gene Ther. 8, 1081-1087.
-
(2001)
Gene Ther.
, vol.8
, pp. 1081-1087
-
-
Shen, J.S.1
Watabe, K.2
Ohashi, T.3
Eto, Y.4
-
16
-
-
12944269059
-
Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells
-
Takenaka, T., Murray, G. J., Qin, G., Quirk, J. M., Ohshima, T., Qasba, P., Clark, K., Kulkarni, A. B., Brady, R. O. & Medin, J. A. 2000 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 97, 7515-7520.
-
(2000)
Proc. Natl. Acad. Sci. USA
, vol.97
, pp. 7515-7520
-
-
Takenaka, T.1
Murray, G.J.2
Qin, G.3
Quirk, J.M.4
Ohshima, T.5
Qasba, P.6
Clark, K.7
Kulkarni, A.B.8
Brady, R.O.9
Medin, J.A.10
-
17
-
-
0033166317
-
Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer
-
Ziegler, R. J., Yew, N. S., Li, C., Cherry, M., Berthelette, P., Romanczuk, H., Ioannou, Y. A., Zeidner, K. M., Desnick, R. J. & Cheng, S. H. 1999 Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer. Hum. Gene Ther. 10, 1667-1682.
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 1667-1682
-
-
Ziegler, R.J.1
Yew, N.S.2
Li, C.3
Cherry, M.4
Berthelette, P.5
Romanczuk, H.6
Ioannou, Y.A.7
Zeidner, K.M.8
Desnick, R.J.9
Cheng, S.H.10
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