-
1
-
-
0000884121
-
Glucose-6-phosphatase of the liver in glycogen storage disease
-
Cori GT, Cori CF. Glucose-6-phosphatase of the liver in glycogen storage disease. J Biol Chem 1952; 199: 661-667.
-
(1952)
J Biol Chem
, vol.199
, pp. 661-667
-
-
Cori, G.T.1
Cori, C.F.2
-
2
-
-
0018198963
-
A new variant of glycogen storage disease type I probably due to a defect in the glucose-6-phosphate transport system
-
Narisawa K, Igarashi Y, Otomo H, Tada K. A new variant of glycogen storage disease type I probably due to a defect in the glucose-6-phosphate transport system. Biochem Biophys Res Commun 1978; 83: 1360-1364.
-
(1978)
Biochem Biophys Res Commun
, vol.83
, pp. 1360-1364
-
-
Narisawa, K.1
Igarashi, Y.2
Otomo, H.3
Tada, K.4
-
3
-
-
0036086034
-
Type I glycogen storage diseases: disorders of the glucose-6-phosphatase complex
-
Chou JY, Matern D, Mansfield BC, Chen YT. Type I glycogen storage diseases: disorders of the glucose-6-phosphatase complex. Curr Mol Med 2002; 2: 121-143.
-
(2002)
Curr Mol Med
, vol.2
, pp. 121-143
-
-
Chou, J.Y.1
Matern, D.2
Mansfield, B.C.3
Chen, Y.T.4
-
4
-
-
0030063963
-
Glucose-6-phosphatase dependent substrate transport in the glycogen storage disease type-1a mouse
-
Lei KJ, Chen H, Pan CJ, Ward JM, Mosinger B, Jr., Lee EJ, et al. Glucose-6-phosphatase dependent substrate transport in the glycogen storage disease type-1a mouse. Nat Genet 1996; 13: 203-209.
-
(1996)
Nat Genet
, vol.13
, pp. 203-209
-
-
Lei, K.J.1
Chen, H.2
Pan, C.J.3
Ward, J.M.4
Mosinger Jr., B.5
Lee, E.J.6
-
5
-
-
0035235572
-
Canine model and genomic structural organization of glycogen storage disease type Ia (GSD Ia)
-
Kishnani PS, Faulkner E, VanCamp S, Jackson M, Brown T, Boney A, Koeberl D, et al. Canine model and genomic structural organization of glycogen storage disease type Ia (GSD Ia). Vet Pathol 2001; 38: 83-91.
-
(2001)
Vet Pathol
, vol.38
, pp. 83-91
-
-
Kishnani, P.S.1
Faulkner, E.2
VanCamp, S.3
Jackson, M.4
Brown, T.5
Boney, A.6
Koeberl, D.7
-
6
-
-
0036053159
-
Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors
-
Beaty RM, Jackson M, Peterson D, Bird A, Brown T, Benjamin DK, Jr., et al. Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors. Gene Ther 2002; 9: 1015-1022.
-
(2002)
Gene Ther
, vol.9
, pp. 1015-1022
-
-
Beaty, R.M.1
Jackson, M.2
Peterson, D.3
Bird, A.4
Brown, T.5
Benjamin Jr., D.K.6
-
7
-
-
0036712798
-
Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in mice
-
Sun MS, Pan CJ, Shieh JJ, Ghosh A, Chen LY, Mansfield BC, et al. Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in mice. Hum Mol Genet 2002; 11: 2155-2164.
-
(2002)
Hum Mol Genet
, vol.11
, pp. 2155-2164
-
-
Sun, M.S.1
Pan, C.J.2
Shieh, J.J.3
Ghosh, A.4
Chen, L.Y.5
Mansfield, B.C.6
-
8
-
-
0037015049
-
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
-
Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A 2002; 99: 11854-11859.
-
(2002)
Proc Natl Acad Sci U S A
, vol.99
, pp. 11854-11859
-
-
Gao, G.P.1
Alvira, M.R.2
Wang, L.3
Calcedo, R.4
Johnston, J.5
Wilson, J.M.6
-
9
-
-
0034882826
-
Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
-
McCarty DM, Monahan PE, Samulski RJ. Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 2001; 8: 1248-1254.
-
(2001)
Gene Ther
, vol.8
, pp. 1248-1254
-
-
McCarty, D.M.1
Monahan, P.E.2
Samulski, R.J.3
-
10
-
-
32444436226
-
Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer
-
Ghosh A, Allamarvdasht M, Pan CJ, Sun MS, Mansfield BC, Byrne BJ, Chou JY. Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer. Gene Ther 2006; 13: 321-329.
-
(2006)
Gene Ther
, vol.13
, pp. 321-329
-
-
Ghosh, A.1
Allamarvdasht, M.2
Pan, C.J.3
Sun, M.S.4
Mansfield, B.C.5
Byrne, B.J.6
Chou, J.Y.7
-
11
-
-
33747600886
-
Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia
-
Koeberl DD, Sun BD, Damodaran TV, Brown T, Millington DS, Benjamin DK, Jr., et al. Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia. Gene Ther 2006; 13: 1281-1289.
-
(2006)
Gene Ther
, vol.13
, pp. 1281-1289
-
-
Koeberl, D.D.1
Sun, B.D.2
Damodaran, T.V.3
Brown, T.4
Millington, D.S.5
Benjamin Jr., D.K.6
-
12
-
-
41149109650
-
AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia
-
Koeberl DD, Pinto C, Sun B, Li S, Kozink DM, Benjamin DK, Jr., et al. AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia. Mol Ther 2008; 16: 665-672.
-
(2008)
Mol Ther
, vol.16
, pp. 665-672
-
-
Koeberl, D.D.1
Pinto, C.2
Sun, B.3
Li, S.4
Kozink, D.M.5
Benjamin Jr., D.K.6
-
13
-
-
80455174006
-
Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector
-
Luo X, Hall G, Li S, Bird A, Lavin PJ, Winn MP, et al. Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector. Mol Ther 2011; 19: 1961-1970.
-
(2011)
Mol Ther
, vol.19
, pp. 1961-1970
-
-
Luo, X.1
Hall, G.2
Li, S.3
Bird, A.4
Lavin, P.J.5
Winn, M.P.6
-
14
-
-
77953136183
-
Complete normalization of hepatic G6PC deficiency in murine glycogen storage disease type Ia using gene therapy
-
Yiu WH, Lee YM, Peng WT, Pan CJ, Mead PA, Mansfield BC, Chou JY. Complete normalization of hepatic G6PC deficiency in murine glycogen storage disease type Ia using gene therapy. Mol Ther 2010; 18: 1076-1084.
-
(2010)
Mol Ther
, vol.18
, pp. 1076-1084
-
-
Yiu, W.H.1
Lee, Y.M.2
Peng, W.T.3
Pan, C.J.4
Mead, P.A.5
Mansfield, B.C.6
Chou, J.Y.7
-
15
-
-
84860155178
-
Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type ia
-
Demaster A, Luo X, Curtis S, Williams KD, Landau DJ, Drake EJ, et al. Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type ia. Hum Gene Ther 2012; 23: 407-418.
-
(2012)
Hum Gene Ther
, vol.23
, pp. 407-418
-
-
Demaster, A.1
Luo, X.2
Curtis, S.3
Williams, K.D.4
Landau, D.J.5
Drake, E.J.6
-
16
-
-
0030987931
-
Hepatocellular adenomas in glycogen storage disease type I and III: a series of 43 patients and review of the literature
-
Labrune P, Trioche P, Duvaltier I, Chevalier P, Odievre M. Hepatocellular adenomas in glycogen storage disease type I and III: a series of 43 patients and review of the literature. J Pediatr Gastroenterol Nutr 1997; 24: 276-279.
-
(1997)
J Pediatr Gastroenterol Nutr
, vol.24
, pp. 276-279
-
-
Labrune, P.1
Trioche, P.2
Duvaltier, I.3
Chevalier, P.4
Odievre, M.5
-
17
-
-
0036387417
-
Glycogen storage disease type I: diagnosis, management, clinical course, and outcome. Results of the European Study on Glycogen Storage Disease Type I (ESGSD I)
-
Rake JP, Visser G, Labrune P, Leonard JV, Ullrich K, Smit GP. Glycogen storage disease type I: diagnosis, management, clinical course, and outcome. Results of the European Study on Glycogen Storage Disease Type I (ESGSD I). Eur J Pediatr 2002; 161( Suppl 1): S20-S34.
-
(2002)
Eur J Pediatr
, vol.161
, Issue.SUPPL. 1
-
-
Rake, J.P.1
Visser, G.2
Labrune, P.3
Leonard, J.V.4
Ullrich, K.5
Smit, G.P.6
-
18
-
-
79951672283
-
Targeted deletion of liver glucose-6 phosphatase mimics glycogen storage disease type 1a including development of multiple adenomas
-
Mutel E, Abdul-Wahed A, Ramamonjisoa N, Stefanutti A, Houberdon I, Cavassila S, et al. Targeted deletion of liver glucose-6 phosphatase mimics glycogen storage disease type 1a including development of multiple adenomas. J Hepatol 2011; 54: 529-537.
-
(2011)
J Hepatol
, vol.54
, pp. 529-537
-
-
Mutel, E.1
Abdul-Wahed, A.2
Ramamonjisoa, N.3
Stefanutti, A.4
Houberdon, I.5
Cavassila, S.6
-
19
-
-
84868210119
-
Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapy
-
Lee YM, Jun HS, Pan C-J, Lin SR, Wilson LH, Mansfield BC, et al. Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapy. HEPATOLOGY 2012; 55: 1719-1729.
-
(2012)
HEPATOLOGY
, vol.55
, pp. 1719-1729
-
-
Lee, Y.M.1
Jun, H.S.2
Pan, C.-J.3
Lin, S.R.4
Wilson, L.H.5
Mansfield, B.C.6
-
20
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
-
Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011; 365: 2357-2365.
-
(2011)
N Engl J Med
, vol.365
, pp. 2357-2365
-
-
Nathwani, A.C.1
Tuddenham, E.G.2
Rangarajan, S.3
Rosales, C.4
McIntosh, J.5
Linch, D.C.6
|