-
1
-
-
0034766525
-
Anderson-Fabry disease: Clinical manifestations and impact of disease in a cohort of 98 hemizygous males
-
K.D. MacDermot A. Holmes and A.H. Miners Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males J. Med. Genet. 38 2001 750-760
-
(2001)
J. Med. Genet.
, vol.38
, pp. 750-760
-
-
MacDermot, K.D.1
Holmes, A.2
Miners, A.H.3
-
2
-
-
13044281563
-
Aging accentuates and bone marrow transplantation ameliorates metabolic defects in Fabry disease mice
-
T. Ohshima et al. Aging accentuates and bone marrow transplantation ameliorates metabolic defects in Fabry disease mice Proc. Natl. Acad. Sci. USA 96 1999 6423-6427
-
(1999)
Proc. Natl. Acad. Sci. USA
, vol.96
, pp. 6423-6427
-
-
Ohshima, T.1
-
3
-
-
0033166317
-
Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer
-
R.J. Ziegler et al. Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer Hum. Gene Ther. 10 1999 1667-1682
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 1667-1682
-
-
Ziegler, R.J.1
-
4
-
-
14844288273
-
Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA
-
F. Xu Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA J. Gene. Med. 7 2004 171-178
-
(2004)
J. Gene. Med.
, vol.7
, pp. 171-178
-
-
Xu, F.1
-
5
-
-
0035163539
-
Fabry disease: Preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice
-
Y.A. Ioannou K.M. Zeidner R.E. Gordon and R.J. Desnick Fabry disease: preclinical studies demonstrate the effectiveness of alpha-galactosidase A replacement in enzyme-deficient mice Am. J. Hum. Genet. 68 2001 14-25
-
(2001)
Am. J. Hum. Genet.
, vol.68
, pp. 14-25
-
-
Ioannou, Y.A.1
Zeidner, K.M.2
Gordon, R.E.3
Desnick, R.J.4
-
6
-
-
0035811624
-
Safety and efficacy of recombinant human alpha-galactosidase A-replacement therapy in Fabry's disease
-
C.M. Eng et al. Safety and efficacy of recombinant human alpha-galactosidase A-replacement therapy in Fabry's disease N. Engl. J. Med. 345 2001 9-16
-
(2001)
N. Engl. J. Med.
, vol.345
, pp. 9-16
-
-
Eng, C.M.1
-
7
-
-
12644284502
-
α-Galactosidase A deficient mice: A model of Fabry disease
-
T. Ohshima et al. α-Galactosidase A deficient mice: A model of Fabry disease Proc. Natl. Acad. Sci. USA 94 1997 2540-2544
-
(1997)
Proc. Natl. Acad. Sci. USA
, vol.94
, pp. 2540-2544
-
-
Ohshima, T.1
-
8
-
-
0034044578
-
A combination of poloxamers increases gene expression of plasmid DNA in skeletal muscle
-
P. Lemieux et al. A combination of poloxamers increases gene expression of plasmid DNA in skeletal muscle Gene Ther. 7 2000 986-991
-
(2000)
Gene Ther.
, vol.7
, pp. 986-991
-
-
Lemieux, P.1
-
9
-
-
0024261447
-
A muscle-specific enhancer is located at the 3′ end of the myosin light-chain 1/3 gene locus
-
M. Donoghue H. Ernst B. Wentworth B. Nadal-Ginard and N. Rosenthal A muscle-specific enhancer is located at the 3′ end of the myosin light-chain 1/3 gene locus Genes Dev. 2 1988 1779-1790
-
(1988)
Genes Dev.
, vol.2
, pp. 1779-1790
-
-
Donoghue, M.1
Ernst, H.2
Wentworth, B.3
Nadal-Ginard, B.4
Rosenthal, N.5
-
10
-
-
0022727888
-
Nuclear factor 1 interacts with five DNA elements in the promoter region of the human cytomegalovirus major immediate early gene
-
L. Hennighausen and B. Fleckenstein Nuclear factor 1 interacts with five DNA elements in the promoter region of the human cytomegalovirus major immediate early gene EMBO J. 5 1986 1367-1371
-
(1986)
EMBO J.
, vol.5
, pp. 1367-1371
-
-
Hennighausen, L.1
Fleckenstein, B.2
-
11
-
-
8244264752
-
Efficient muscle-specific transgene expression after adenovirus-mediated gene transfer in mice using a 1.35 kb muscle creatine kinase promoter/ enhancer
-
N. Larochelle et al. Efficient muscle-specific transgene expression after adenovirus-mediated gene transfer in mice using a 1.35 kb muscle creatine kinase promoter/enhancer Gene Ther. 4 1997 465-472
-
(1997)
Gene Ther.
, vol.4
, pp. 465-472
-
-
Larochelle, N.1
-
12
-
-
0034091578
-
Reduction of globotriaosylceramide in Fabry disease mice by substrate deprivation
-
A. Abe et al. Reduction of globotriaosylceramide in Fabry disease mice by substrate deprivation J. Clin. Invest. 105 2000 1563-1571
-
(2000)
J. Clin. Invest.
, vol.105
, pp. 1563-1571
-
-
Abe, A.1
-
13
-
-
0037109052
-
Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer
-
H. Takahashi et al. Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer Proc. Natl. Acad. Sci. USA 99 2002 13777-13782
-
(2002)
Proc. Natl. Acad. Sci. USA
, vol.99
, pp. 13777-13782
-
-
Takahashi, H.1
-
14
-
-
0037453003
-
Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer
-
J. Park et al. Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer Proc. Natl. Acad. Sci. USA 100 2003 3450-3454
-
(2003)
Proc. Natl. Acad. Sci. USA
, vol.100
, pp. 3450-3454
-
-
Park, J.1
-
15
-
-
0037135675
-
Role of transfection in the priming of cytotoxic T-cells by DNA-mediated immunization
-
H. Sbai J. Schneider A.V. Hill and R.G. Whalen Role of transfection in the priming of cytotoxic T-cells by DNA-mediated immunization Vaccine 20 2002 3137-3147
-
(2002)
Vaccine
, vol.20
, pp. 3137-3147
-
-
Sbai, H.1
Schneider, J.2
Hill, A.V.3
Whalen, R.G.4
-
17
-
-
85047691459
-
Developing DNA vaccines that call to dendritic cells
-
M.A. Kutzler and D.B. Weiner Developing DNA vaccines that call to dendritic cells J. Clin. Invest. 114 2004 1241-1244
-
(2004)
J. Clin. Invest.
, vol.114
, pp. 1241-1244
-
-
Kutzler, M.A.1
Weiner, D.B.2
-
18
-
-
12944265457
-
Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease
-
R. Schiffmann et al. Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease Proc. Natl. Acad. Sci. USA 97 2000 365-370
-
(2000)
Proc. Natl. Acad. Sci. USA
, vol.97
, pp. 365-370
-
-
Schiffmann, R.1
-
19
-
-
0037029879
-
Muscle specific versus ubiquitous expression of Gag based HIV-1 DNA vaccines: A comparative analysis
-
A. Bojak D. Hammer H. Wolf and R. Wagner Muscle specific versus ubiquitous expression of Gag based HIV-1 DNA vaccines: A comparative analysis Vaccine 20 2002 1975-1979
-
(2002)
Vaccine
, vol.20
, pp. 1975-1979
-
-
Bojak, A.1
Hammer, D.2
Wolf, H.3
Wagner, R.4
-
20
-
-
0033695299
-
Proteolysis in MHC class II antigen presentation: Who's in charge?
-
J.A. Villadangos and H.L. Ploegh Proteolysis in MHC class II antigen presentation: Who's in charge? Immunity 12 2000 233-239
-
(2000)
Immunity
, vol.12
, pp. 233-239
-
-
Villadangos, J.A.1
Ploegh, H.L.2
-
21
-
-
0037713660
-
Fabry disease: Novel alpha-galactosidase A 3′-terminal mutations result in multiple transcripts due to aberrant 3′-end formation
-
M. Yasuda J. Shabbeer M. Osawa and R.J. Desnick Fabry disease: Novel alpha-galactosidase A 3′-terminal mutations result in multiple transcripts due to aberrant 3′-end formation Am. J. Hum. Genet. 73 2003 162-173
-
(2003)
Am. J. Hum. Genet.
, vol.73
, pp. 162-173
-
-
Yasuda, M.1
Shabbeer, J.2
Osawa, M.3
Desnick, R.J.4
-
22
-
-
0028990407
-
Alpha-galactosidase gene mutations in Fabry disease: Heterogeneous expressions of mutant enzyme proteins
-
T. Okumiya S. Ishii R. Kase S. Kamei H. Sakuraba and Y. Suzuki Alpha-galactosidase gene mutations in Fabry disease: Heterogeneous expressions of mutant enzyme proteins Hum. Genet. 95 1995 557-561
-
(1995)
Hum. Genet.
, vol.95
, pp. 557-561
-
-
Okumiya, T.1
Ishii, S.2
Kase, R.3
Kamei, S.4
Sakuraba, H.5
Suzuki, Y.6
-
25
-
-
0030914425
-
Gene transfer and expression of human alpha-galactosidase from mouse muscle in vitro and in vivo
-
F.J. Novo D.C. Gorecki G. Goldspink and K.D. MacDermot Gene transfer and expression of human alpha-galactosidase from mouse muscle in vitro and in vivo Gene Ther. 4 1997 488-492
-
(1997)
Gene Ther.
, vol.4
, pp. 488-492
-
-
Novo, F.J.1
Gorecki, D.C.2
Goldspink, G.3
MacDermot, K.D.4
-
26
-
-
0031589164
-
Immune responses, not promoter inactivation, are responsible for decreased long-term expression following plasmid gene transfer into skeletal muscle
-
K.E. Wells et al. Immune responses, not promoter inactivation, are responsible for decreased long-term expression following plasmid gene transfer into skeletal muscle FEBS Lett. 407 1997 164-168
-
(1997)
FEBS Lett.
, vol.407
, pp. 164-168
-
-
Wells, K.E.1
-
27
-
-
0033623151
-
Immune responses to dystropin: Implications for gene therapy of Duchenne muscular dystrophy
-
A. Ferrer K.E. Wells and D.J. Wells Immune responses to dystropin: implications for gene therapy of Duchenne muscular dystrophy Gene Ther. 7 2000 1439-1446
-
(2000)
Gene Ther.
, vol.7
, pp. 1439-1446
-
-
Ferrer, A.1
Wells, K.E.2
Wells, D.J.3
|