-
1
-
-
0017368366
-
GM2 ganglioside lysosomal storage disease in cats with beta-hexosaminidase deficiency
-
Cork L.C., Munnell J.F., Lorenz M.D., Murphy J.V., Baker H.J., and Rattazzi M.C. GM2 ganglioside lysosomal storage disease in cats with beta-hexosaminidase deficiency. Science 196 (1977) 1014-1017
-
(1977)
Science
, vol.196
, pp. 1014-1017
-
-
Cork, L.C.1
Munnell, J.F.2
Lorenz, M.D.3
Murphy, J.V.4
Baker, H.J.5
Rattazzi, M.C.6
-
2
-
-
34248525942
-
Clinical and molecular analysis of GM2 gangliosidosis in two apparent littermate kittens of the Japanese domestic cat
-
Hasegawa D., Yamato O., Kobayashi M., Fujita M., Nakamura S., Takahashi K., Satoh H., Shoda T., Hayashi D., Yamasaki M., Maede Y., Arai T., and Orima H. Clinical and molecular analysis of GM2 gangliosidosis in two apparent littermate kittens of the Japanese domestic cat. J. Fel. Med. Surg. 9 (2007) 232-237
-
(2007)
J. Fel. Med. Surg.
, vol.9
, pp. 232-237
-
-
Hasegawa, D.1
Yamato, O.2
Kobayashi, M.3
Fujita, M.4
Nakamura, S.5
Takahashi, K.6
Satoh, H.7
Shoda, T.8
Hayashi, D.9
Yamasaki, M.10
Maede, Y.11
Arai, T.12
Orima, H.13
-
3
-
-
0021919599
-
Characterization of a new model of GM2-gangliosidosis (Sandhoff's disease) in Korat cats
-
Neuwelt E.A., Johnson W.G., Blank N.K., Pagel M.A., Maslen-McClure C., McClure M.J., and Wu P.M. Characterization of a new model of GM2-gangliosidosis (Sandhoff's disease) in Korat cats. J. Clin. Invest. 76 (1985) 482-490
-
(1985)
J. Clin. Invest.
, vol.76
, pp. 482-490
-
-
Neuwelt, E.A.1
Johnson, W.G.2
Blank, N.K.3
Pagel, M.A.4
Maslen-McClure, C.5
McClure, M.J.6
Wu, P.M.7
-
5
-
-
0021783450
-
GM2 gangliosidosis in a Japanese spaniel
-
Cummings J.F., Wood P.A., Walkley S.U., de Lahunta A., and DeForest M.E. GM2 gangliosidosis in a Japanese spaniel. Acta Neuropathol. 67 (1985) 247-253
-
(1985)
Acta Neuropathol.
, vol.67
, pp. 247-253
-
-
Cummings, J.F.1
Wood, P.A.2
Walkley, S.U.3
de Lahunta, A.4
DeForest, M.E.5
-
7
-
-
0029113867
-
Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism
-
Sango K., Yamanaka S., Hoffmann A., Okuda Y., Grinberg A., Westphal H., McDonald M.P., Crawley J.N., Sandhoff K., and Suzuki K. Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism. Nat. Genet. 11 (1995) 170-176
-
(1995)
Nat. Genet.
, vol.11
, pp. 170-176
-
-
Sango, K.1
Yamanaka, S.2
Hoffmann, A.3
Okuda, Y.4
Grinberg, A.5
Westphal, H.6
McDonald, M.P.7
Crawley, J.N.8
Sandhoff, K.9
Suzuki, K.10
-
8
-
-
9044236158
-
Dramatically different phenotypes in mouse models of human Tay-Sachs and Sandhoff diseases
-
Phaneuf D., Wakamatsu N., Huang J.Q., Borowski A., Peterson A.C., Fortunato S.R., Ritter G., Igdoura S.A., Morales C.R., Benoit G., Akerman B.R., Leclerc D., Hanai N., Marth J.D., Trasler J.M., and Gravel R.A. Dramatically different phenotypes in mouse models of human Tay-Sachs and Sandhoff diseases. Hum. Mol. Genet. 5 (1996) 1-14
-
(1996)
Hum. Mol. Genet.
, vol.5
, pp. 1-14
-
-
Phaneuf, D.1
Wakamatsu, N.2
Huang, J.Q.3
Borowski, A.4
Peterson, A.C.5
Fortunato, S.R.6
Ritter, G.7
Igdoura, S.A.8
Morales, C.R.9
Benoit, G.10
Akerman, B.R.11
Leclerc, D.12
Hanai, N.13
Marth, J.D.14
Trasler, J.M.15
Gravel, R.A.16
-
9
-
-
27944484472
-
Mutation of the GM2 activator protein in a feline model of GM2 gangliosidosis
-
Martin D.R., Cox N.R., Morrison N.E., Kennamer D.M., Peck S.L., Dodson A.N., Gentry A.S., Griffin B., Rolsma M.D., and Baker H.J. Mutation of the GM2 activator protein in a feline model of GM2 gangliosidosis. Acta Neuropathol. 110 (2005) 443-450
-
(2005)
Acta Neuropathol.
, vol.110
, pp. 443-450
-
-
Martin, D.R.1
Cox, N.R.2
Morrison, N.E.3
Kennamer, D.M.4
Peck, S.L.5
Dodson, A.N.6
Gentry, A.S.7
Griffin, B.8
Rolsma, M.D.9
Baker, H.J.10
-
10
-
-
1842714246
-
An inversion of 25 base pairs causes feline GM2 gangliosidosis variant 0
-
Martin D.R., Krum B.K., Varadarajan G.S., Hathcock T.L., Smith B.F., and Baker H.J. An inversion of 25 base pairs causes feline GM2 gangliosidosis variant 0. Exp. Neurol. 187 (2004) 30-37
-
(2004)
Exp. Neurol.
, vol.187
, pp. 30-37
-
-
Martin, D.R.1
Krum, B.K.2
Varadarajan, G.S.3
Hathcock, T.L.4
Smith, B.F.5
Baker, H.J.6
-
11
-
-
0028336694
-
Characterization of the molecular defect in a feline model for type II GM2-gangliosidosis (Sandhoff disease)
-
Muldoon L.L., Neuwelt E.A., Pagel M.A., and Weiss D.L. Characterization of the molecular defect in a feline model for type II GM2-gangliosidosis (Sandhoff disease). Am. J. Pathol. 144 (1994) 1109-1118
-
(1994)
Am. J. Pathol.
, vol.144
, pp. 1109-1118
-
-
Muldoon, L.L.1
Neuwelt, E.A.2
Pagel, M.A.3
Weiss, D.L.4
-
12
-
-
33845238476
-
Nonsense mutation of feline beta-hexosaminidase beta-subunit (HEXB) gene causing Sandhoff disease in a family of Japanese domestic cats
-
Kanae Y., Endoh D., Yamato O., Hayashi D., Matsunaga S., Ogawa H., Maede Y., and Hayashi M. Nonsense mutation of feline beta-hexosaminidase beta-subunit (HEXB) gene causing Sandhoff disease in a family of Japanese domestic cats. Res. Vet. Sci. 82 (2007) 54-60
-
(2007)
Res. Vet. Sci.
, vol.82
, pp. 54-60
-
-
Kanae, Y.1
Endoh, D.2
Yamato, O.3
Hayashi, D.4
Matsunaga, S.5
Ogawa, H.6
Maede, Y.7
Hayashi, M.8
-
13
-
-
0024121624
-
Gene encoding the human beta-hexosaminidase beta chain: extensive homology of intron placement in the alpha- and beta-chain genes
-
Proia R.L. Gene encoding the human beta-hexosaminidase beta chain: extensive homology of intron placement in the alpha- and beta-chain genes. Proc. Natl. Acad. Sci. USA 85 (1988) 1883-1887
-
(1988)
Proc. Natl. Acad. Sci. USA
, vol.85
, pp. 1883-1887
-
-
Proia, R.L.1
-
14
-
-
0028173323
-
Characterization of the murine beta-hexosaminidase (HEXB) gene
-
Triggs-Raine B.L., Benoit G., Salo T.J., Trasler J.M., and Gravel R.A. Characterization of the murine beta-hexosaminidase (HEXB) gene. Biochim. Biophys. Acta 1227 (1994) 79-86
-
(1994)
Biochim. Biophys. Acta
, vol.1227
, pp. 79-86
-
-
Triggs-Raine, B.L.1
Benoit, G.2
Salo, T.J.3
Trasler, J.M.4
Gravel, R.A.5
-
15
-
-
0028292563
-
Structure and expression of the mouse beta-hexosaminidase genes, Hexa and Hexb
-
Yamanaka S., Johnson O.N., Norflus F., Boles D.J., and Proia R.L. Structure and expression of the mouse beta-hexosaminidase genes, Hexa and Hexb. Genomics 21 (1994) 588-596
-
(1994)
Genomics
, vol.21
, pp. 588-596
-
-
Yamanaka, S.1
Johnson, O.N.2
Norflus, F.3
Boles, D.J.4
Proia, R.L.5
-
16
-
-
43249114793
-
Molecular consequences of the pathogenic mutation in feline GM1 gangliosidosis
-
Martin D.R., Rigat B.A., Foureman P., Varadarajan G.S., Hwang M., Krum B.K., Smith B.F., Callahan J.W., Mahuran D.J., and Baker H.J. Molecular consequences of the pathogenic mutation in feline GM1 gangliosidosis. Mol. Genet. Metab. 94 (2008) 212-221
-
(2008)
Mol. Genet. Metab.
, vol.94
, pp. 212-221
-
-
Martin, D.R.1
Rigat, B.A.2
Foureman, P.3
Varadarajan, G.S.4
Hwang, M.5
Krum, B.K.6
Smith, B.F.7
Callahan, J.W.8
Mahuran, D.J.9
Baker, H.J.10
-
17
-
-
11844281490
-
GM2-gangliosidosis variant 0 (Sandhoff-like disease) in a family of Japanese domestic cats
-
Yamato O., Matsunaga S., Takata K., Uetsuka K., Satoh H., Shoda T., Baba Y., Yasoshima A., Kato K., Takahashi K., Yamasaki M., Nakayama H., Doi K., Maede Y., and Ogawa H. GM2-gangliosidosis variant 0 (Sandhoff-like disease) in a family of Japanese domestic cats. Vet. Rec. 155 (2004) 739-744
-
(2004)
Vet. Rec.
, vol.155
, pp. 739-744
-
-
Yamato, O.1
Matsunaga, S.2
Takata, K.3
Uetsuka, K.4
Satoh, H.5
Shoda, T.6
Baba, Y.7
Yasoshima, A.8
Kato, K.9
Takahashi, K.10
Yamasaki, M.11
Nakayama, H.12
Doi, K.13
Maede, Y.14
Ogawa, H.15
-
18
-
-
0015146931
-
Neuronal GM1 gangliosidosis in a Siamese cat with beta-galactosidase deficiency
-
Baker Jr. H.J., Lindsey J.R., McKhann G.M., and Farrell D.F. Neuronal GM1 gangliosidosis in a Siamese cat with beta-galactosidase deficiency. Science 174 (1971) 838-839
-
(1971)
Science
, vol.174
, pp. 838-839
-
-
Baker Jr., H.J.1
Lindsey, J.R.2
McKhann, G.M.3
Farrell, D.F.4
-
19
-
-
0031723896
-
Two mutations remote from an exon/intron junction in the beta-hexosaminidase beta-subunit gene affect 3′-splice site selection and cause Sandhoff disease
-
Fujimaru M., Tanaka A., Choeh K., Wakamatsu N., Sakuraba H., and Isshiki G. Two mutations remote from an exon/intron junction in the beta-hexosaminidase beta-subunit gene affect 3′-splice site selection and cause Sandhoff disease. Hum. Genet. 103 (1998) 462-469
-
(1998)
Hum. Genet.
, vol.103
, pp. 462-469
-
-
Fujimaru, M.1
Tanaka, A.2
Choeh, K.3
Wakamatsu, N.4
Sakuraba, H.5
Isshiki, G.6
-
20
-
-
0024566551
-
Genetic cause of a juvenile form of Sandhoff disease. Abnormal splicing of beta-hexosaminidase beta chain gene transcript due to a point mutation within intron 12
-
Nakano T., and Suzuki K. Genetic cause of a juvenile form of Sandhoff disease. Abnormal splicing of beta-hexosaminidase beta chain gene transcript due to a point mutation within intron 12. J. Biol. Chem. 264 (1989) 5155-5158
-
(1989)
J. Biol. Chem.
, vol.264
, pp. 5155-5158
-
-
Nakano, T.1
Suzuki, K.2
-
21
-
-
0026785968
-
A novel exon mutation in the human beta-hexosaminidase beta-subunit gene affects 3′ splice site selection
-
Wakamatsu N., Kobayashi H., Miyatake T., and Tsuji S. A novel exon mutation in the human beta-hexosaminidase beta-subunit gene affects 3′ splice site selection. J. Biol. Chem. 267 (1992) 2406-2413
-
(1992)
J. Biol. Chem.
, vol.267
, pp. 2406-2413
-
-
Wakamatsu, N.1
Kobayashi, H.2
Miyatake, T.3
Tsuji, S.4
-
22
-
-
0037196882
-
Compound heterozygosity with two novel mutations in the HEXB gene produces adult Sandhoff disease presenting as a motor neuron disease phenotype
-
Yoshizawa T., Kohno Y., Nissato S., and Shoji S. Compound heterozygosity with two novel mutations in the HEXB gene produces adult Sandhoff disease presenting as a motor neuron disease phenotype. J. Neurol. Sci. 195 (2002) 129-138
-
(2002)
J. Neurol. Sci.
, vol.195
, pp. 129-138
-
-
Yoshizawa, T.1
Kohno, Y.2
Nissato, S.3
Shoji, S.4
-
23
-
-
0026760912
-
An unusual splicing mutation in the HEXB gene is associated with dramatically different phenotypes in patients from different racial backgrounds
-
McInnes B., Potier M., Wakamatsu N., Melancon S.B., Klavins M.H., Tsuji S., and Mahuran D.J. An unusual splicing mutation in the HEXB gene is associated with dramatically different phenotypes in patients from different racial backgrounds. J. Clin. Invest. 90 (1992) 306-314
-
(1992)
J. Clin. Invest.
, vol.90
, pp. 306-314
-
-
McInnes, B.1
Potier, M.2
Wakamatsu, N.3
Melancon, S.B.4
Klavins, M.H.5
Tsuji, S.6
Mahuran, D.J.7
-
24
-
-
38349138834
-
Regulation of alternative splicing: more than just the ABCs
-
House A.E., and Lynch K.W. Regulation of alternative splicing: more than just the ABCs. J. Biol. Chem. 283 (2008) 1217-1221
-
(2008)
J. Biol. Chem.
, vol.283
, pp. 1217-1221
-
-
House, A.E.1
Lynch, K.W.2
-
25
-
-
42449098125
-
Splicing regulation: from a parts list of regulatory elements to an integrated splicing code
-
Wang Z., and Burge C.B. Splicing regulation: from a parts list of regulatory elements to an integrated splicing code. RNA 14 (2008) 802-813
-
(2008)
RNA
, vol.14
, pp. 802-813
-
-
Wang, Z.1
Burge, C.B.2
-
26
-
-
0033018496
-
Accelerated transport and maturation of lysosomal alpha-galactosidase A in Fabry lymphoblasts by an enzyme inhibitor
-
Fan J.Q., Ishii S., Asano N., and Suzuki Y. Accelerated transport and maturation of lysosomal alpha-galactosidase A in Fabry lymphoblasts by an enzyme inhibitor. Nat. Med. 5 (1999) 112-115
-
(1999)
Nat. Med.
, vol.5
, pp. 112-115
-
-
Fan, J.Q.1
Ishii, S.2
Asano, N.3
Suzuki, Y.4
-
27
-
-
34548145120
-
Mutant alpha-galactosidase A enzymes identified in Fabry disease patients with residual enzyme activity: biochemical characterization and restoration of normal intracellular processing by 1-deoxygalactonojirimycin
-
Ishii S., Chang H.H., Kawasaki K., Yasuda K., Wu H.L., Garman S.C., and Fan J.Q. Mutant alpha-galactosidase A enzymes identified in Fabry disease patients with residual enzyme activity: biochemical characterization and restoration of normal intracellular processing by 1-deoxygalactonojirimycin. Biochem. J. 406 (2007) 285-295
-
(2007)
Biochem. J.
, vol.406
, pp. 285-295
-
-
Ishii, S.1
Chang, H.H.2
Kawasaki, K.3
Yasuda, K.4
Wu, H.L.5
Garman, S.C.6
Fan, J.Q.7
-
28
-
-
26444609722
-
ER retention and degradation as the molecular basis underlying Gaucher disease heterogeneity
-
Ron I., Horowitz M., Department of Cell R., and Immunology T.A.U.R.A.I. ER retention and degradation as the molecular basis underlying Gaucher disease heterogeneity. Hum. Mol. Genet. 14 (2005) 2387-2398
-
(2005)
Hum. Mol. Genet.
, vol.14
, pp. 2387-2398
-
-
Ron, I.1
Horowitz, M.2
Department of Cell, R.3
-
29
-
-
33744741034
-
Endoplasmic reticulum stress-induced caspase-4 activation mediates apoptosis and neurodegeneration in INCL
-
Kim S.J., Zhang Z., Hitomi E., Lee Y.C., and Mukherjee A.B. Endoplasmic reticulum stress-induced caspase-4 activation mediates apoptosis and neurodegeneration in INCL. Hum. Mol. Genet. 15 (2006) 1826-1834
-
(2006)
Hum. Mol. Genet.
, vol.15
, pp. 1826-1834
-
-
Kim, S.J.1
Zhang, Z.2
Hitomi, E.3
Lee, Y.C.4
Mukherjee, A.B.5
-
30
-
-
33744761561
-
Palmitoyl-protein thioesterase-1 deficiency leads to the activation of caspase-9 and contributes to rapid neurodegeneration in INCL
-
Kim S.J., Zhang Z., Lee Y.C., and Mukherjee A.B. Palmitoyl-protein thioesterase-1 deficiency leads to the activation of caspase-9 and contributes to rapid neurodegeneration in INCL. Hum. Mol. Genet. 15 (2006) 1580-1586
-
(2006)
Hum. Mol. Genet.
, vol.15
, pp. 1580-1586
-
-
Kim, S.J.1
Zhang, Z.2
Lee, Y.C.3
Mukherjee, A.B.4
-
31
-
-
31144462635
-
Palmitoyl-protein thioesterase-1 deficiency mediates the activation of the unfolded protein response and neuronal apoptosis in INCL
-
Zhang Z., Lee Y.C., Kim S.J., Choi M.S., Tsai P.C., Xu Y., Xiao Y.J., Zhang P., Heffer A., and Mukherjee A.B. Palmitoyl-protein thioesterase-1 deficiency mediates the activation of the unfolded protein response and neuronal apoptosis in INCL. Hum. Mol. Genet. 15 (2006) 337-346
-
(2006)
Hum. Mol. Genet.
, vol.15
, pp. 337-346
-
-
Zhang, Z.1
Lee, Y.C.2
Kim, S.J.3
Choi, M.S.4
Tsai, P.C.5
Xu, Y.6
Xiao, Y.J.7
Zhang, P.8
Heffer, A.9
Mukherjee, A.B.10
-
32
-
-
61449148380
-
Comparative analysis of brain lipids in mice, cats, and humans with Sandhoff Disease
-
in press, doi:10.1007/s11745.008.3268
-
R.C. Baek, D.R. Martin, N.R. Cox, T.N. Seyfried, Comparative analysis of brain lipids in mice, cats, and humans with Sandhoff Disease, Lipids, in press, doi:10.1007/s11745.008.3268.
-
Lipids
-
-
Baek, R.C.1
Martin, D.R.2
Cox, N.R.3
Seyfried, T.N.4
-
33
-
-
0348023267
-
Domestic cats as laboratory animals
-
Fox J.G., Anderson L.C., Lowe F.M., and Quimby F.W. (Eds), Academic Press, New York, NY
-
Griffin B., and Baker H.J. Domestic cats as laboratory animals. In: Fox J.G., Anderson L.C., Lowe F.M., and Quimby F.W. (Eds). Laboratory Animal Medicine (2002), Academic Press, New York, NY 460-482
-
(2002)
Laboratory Animal Medicine
, pp. 460-482
-
-
Griffin, B.1
Baker, H.J.2
-
34
-
-
0008816796
-
Transplantation in animal model systems
-
Desnick R.J. (Ed), Churchill Livingstone, New York
-
Haskins M., Baker H.J., Birkenmeier E., Hoogerbrugge P.M., Poorthuis B.J.H.M., Sakiyama T., Shull R.M., Taylor R.M., Thrall M.A., and Walkley S.U. Transplantation in animal model systems. In: Desnick R.J. (Ed). Treatment of Genetic Diseases (1991), Churchill Livingstone, New York 183-201
-
(1991)
Treatment of Genetic Diseases
, pp. 183-201
-
-
Haskins, M.1
Baker, H.J.2
Birkenmeier, E.3
Hoogerbrugge, P.M.4
Poorthuis, B.J.H.M.5
Sakiyama, T.6
Shull, R.M.7
Taylor, R.M.8
Thrall, M.A.9
Walkley, S.U.10
-
35
-
-
0029634358
-
Bone marrow transplantation for lysosomal diseases
-
Walkley S.U., and Dobrenis K. Bone marrow transplantation for lysosomal diseases. Lancet 345 (1995) 1382-1383
-
(1995)
Lancet
, vol.345
, pp. 1382-1383
-
-
Walkley, S.U.1
Dobrenis, K.2
-
36
-
-
0141996932
-
Adeno-associated virus vector-mediated transduction in the cat brain
-
Vite C.H., Passini M.A., Haskins M.E., and Wolfe J.H. Adeno-associated virus vector-mediated transduction in the cat brain. Gene Ther. 10 (2003) 1874-1881
-
(2003)
Gene Ther.
, vol.10
, pp. 1874-1881
-
-
Vite, C.H.1
Passini, M.A.2
Haskins, M.E.3
Wolfe, J.H.4
-
37
-
-
0031263554
-
Lysosomal storage diseases of animals: an essay in comparative pathology
-
Jolly R.D., and Walkley S.U. Lysosomal storage diseases of animals: an essay in comparative pathology. Vet. Pathol. 34 (1997) 527-548
-
(1997)
Vet. Pathol.
, vol.34
, pp. 527-548
-
-
Jolly, R.D.1
Walkley, S.U.2
-
38
-
-
0042385114
-
Prevalence of mucopolysaccharidosis type VI mutations in Siamese cats
-
Crawley A.C., Muntz F.H., Haskins M.E., Jones B.R., and Hopwood J.J. Prevalence of mucopolysaccharidosis type VI mutations in Siamese cats. J. Vet. Int. Med. 17 (2003) 495-498
-
(2003)
J. Vet. Int. Med.
, vol.17
, pp. 495-498
-
-
Crawley, A.C.1
Muntz, F.H.2
Haskins, M.E.3
Jones, B.R.4
Hopwood, J.J.5
-
39
-
-
0031985352
-
Two mutations within a feline mucopolysaccharidosis type VI colony cause three different clinical phenotypes
-
Crawley A.C., Yogalingam G., Muller V.J., and Hopwood J.J. Two mutations within a feline mucopolysaccharidosis type VI colony cause three different clinical phenotypes. J. Clin. Invest. 101 (1998) 109-119
-
(1998)
J. Clin. Invest.
, vol.101
, pp. 109-119
-
-
Crawley, A.C.1
Yogalingam, G.2
Muller, V.J.3
Hopwood, J.J.4
-
40
-
-
34247859067
-
Pyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 gangliosidosis
-
Maegawa G.H., Tropak M., Buttner J., Stockley T., Kok F., Clarke J.T., and Mahuran D.J. Pyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 gangliosidosis. J. Biol. Chem. 282 (2007) 9150-9161
-
(2007)
J. Biol. Chem.
, vol.282
, pp. 9150-9161
-
-
Maegawa, G.H.1
Tropak, M.2
Buttner, J.3
Stockley, T.4
Kok, F.5
Clarke, J.T.6
Mahuran, D.J.7
-
41
-
-
34147160607
-
Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease
-
Lee J.P., Jeyakumar M., Gonzalez R., Takahashi H., Lee P.J., Baek R.C., Clark D., Rose H., Fu G., Clarke J., McKercher S., Meerloo J., Muller F.J., Park K.I., Butters T.D., Dwek R.A., Schwartz P., Tong G., Wenger D., Lipton S.A., Seyfried T.N., Platt F.M., and Snyder E.Y. Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease. Nat. Med. 13 (2007) 439-447
-
(2007)
Nat. Med.
, vol.13
, pp. 439-447
-
-
Lee, J.P.1
Jeyakumar, M.2
Gonzalez, R.3
Takahashi, H.4
Lee, P.J.5
Baek, R.C.6
Clark, D.7
Rose, H.8
Fu, G.9
Clarke, J.10
McKercher, S.11
Meerloo, J.12
Muller, F.J.13
Park, K.I.14
Butters, T.D.15
Dwek, R.A.16
Schwartz, P.17
Tong, G.18
Wenger, D.19
Lipton, S.A.20
Seyfried, T.N.21
Platt, F.M.22
Snyder, E.Y.23
more..
-
42
-
-
33745925879
-
Effective gene therapy in an authentic model of Tay-Sachs related diseases
-
Cachon-Gonzalez M.B., Wang S.Z., Lynch A., Ziegler R., Cheng S.H., and Cox T.M. Effective gene therapy in an authentic model of Tay-Sachs related diseases. Proc. Natl. Acad. Sci. USA 103 (2006) 10373-10378
-
(2006)
Proc. Natl. Acad. Sci. USA
, vol.103
, pp. 10373-10378
-
-
Cachon-Gonzalez, M.B.1
Wang, S.Z.2
Lynch, A.3
Ziegler, R.4
Cheng, S.H.5
Cox, T.M.6
-
43
-
-
33845970263
-
Complete correction of enzymatic deficiency and neurochemistry in the GM1 gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery
-
Broekman M.L., Baek R.C., Comer L.A., Fernandez J.L., Seyfried T.N., and Sena-Esteves M. Complete correction of enzymatic deficiency and neurochemistry in the GM1 gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery. Mol. Ther. 15 (2007) 30-37
-
(2007)
Mol. Ther.
, vol.15
, pp. 30-37
-
-
Broekman, M.L.1
Baek, R.C.2
Comer, L.A.3
Fernandez, J.L.4
Seyfried, T.N.5
Sena-Esteves, M.6
|