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Items: 1 to 20 of 144

1.

Bone marrow transplantation prolongs life span and ameliorates neurologic manifestations in Sandhoff disease mice.

Norflus F, Tifft CJ, McDonald MP, Goldstein G, Crawley JN, Hoffmann A, Sandhoff K, Suzuki K, Proia RL.

J Clin Invest. 1998 May 1;101(9):1881-8.

2.

Biology and potential strategies for the treatment of GM2 gangliosidoses.

Chavany C, Jendoubi M.

Mol Med Today. 1998 Apr;4(4):158-65. Review.

PMID:
9572057
3.

[Molecular pathogenesis and therapeutic approach of GM2 gangliosidosis].

Tsuji D.

Yakugaku Zasshi. 2013;133(2):269-74. Review. Japanese.

4.
5.

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 MP, Crawley JN, Sandhoff K, Suzuki K, Proia RL.

Nat Genet. 1995 Oct;11(2):170-6.

PMID:
7550345
6.

Mouse model of GM2 activator deficiency manifests cerebellar pathology and motor impairment.

Liu Y, Hoffmann A, Grinberg A, Westphal H, McDonald MP, Miller KM, Crawley JN, Sandhoff K, Suzuki K, Proia RL.

Proc Natl Acad Sci U S A. 1997 Jul 22;94(15):8138-43.

7.

Apoptotic cell death in mouse models of GM2 gangliosidosis and observations on human Tay-Sachs and Sandhoff diseases.

Huang JQ, Trasler JM, Igdoura S, Michaud J, Hanal N, Gravel RA.

Hum Mol Genet. 1997 Oct;6(11):1879-85.

PMID:
9302266
8.

Reversibility of neuropathology in Tay-Sachs-related diseases.

Cachón-González MB, Wang SZ, Ziegler R, Cheng SH, Cox TM.

Hum Mol Genet. 2014 Feb 1;23(3):730-48. doi: 10.1093/hmg/ddt459. Epub 2013 Sep 20.

9.

Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation.

Jeyakumar M, Norflus F, Tifft CJ, Cortina-Borja M, Butters TD, Proia RL, Perry VH, Dwek RA, Platt FM.

Blood. 2001 Jan 1;97(1):327-9.

10.

Plasmid-based gene transfer ameliorates visceral storage in a mouse model of Sandhoff disease.

Yamaguchi A, Katsuyama K, Suzuki K, Kosaka K, Aoki I, Yamanaka S.

J Mol Med (Berl). 2003 Mar;81(3):185-93. Epub 2003 Feb 12.

PMID:
12682727
11.
12.
13.

Reversion of the biochemical defects in murine embryonic Sandhoff neurons using a bicistronic lentiviral vector encoding hexosaminidase alpha and beta.

Arfi A, Zisling R, Richard E, Batista L, Poenaru L, Futerman AH, Caillaud C.

J Neurochem. 2006 Mar;96(6):1572-9. Epub 2006 Jan 25.

14.

Therapeutic evaluation of GM2 gangliosidoses by ELISA using anti-GM2 ganglioside antibodies.

Tsuji D, Higashine Y, Matsuoka K, Sakuraba H, Itoh K.

Clin Chim Acta. 2007 Mar;378(1-2):38-41. Epub 2006 Oct 24.

PMID:
17196574
15.

Dramatically different phenotypes in mouse models of human Tay-Sachs and Sandhoff diseases.

Phaneuf D, Wakamatsu N, Huang JQ, Borowski A, Peterson AC, Fortunato SR, Ritter G, Igdoura SA, Morales CR, Benoit G, Akerman BR, Leclerc D, Hanai N, Marth JD, Trasler JM, Gravel RA.

Hum Mol Genet. 1996 Jan;5(1):1-14.

PMID:
8789434
16.

Mice lacking both subunits of lysosomal beta-hexosaminidase display gangliosidosis and mucopolysaccharidosis.

Sango K, McDonald MP, Crawley JN, Mack ML, Tifft CJ, Skop E, Starr CM, Hoffmann A, Sandhoff K, Suzuki K, Proia RL.

Nat Genet. 1996 Nov;14(3):348-52.

PMID:
8896570
17.

Late onset Tay-Sachs disease in mice with targeted disruption of the Hexa gene: behavioral changes and pathology of the central nervous system.

Miklyaeva EI, Dong W, Bureau A, Fattahie R, Xu Y, Su M, Fick GH, Huang JQ, Igdoura S, Hanai N, Gravel RA.

Brain Res. 2004 Mar 19;1001(1-2):37-50.

PMID:
14972652
18.

Effective gene therapy in an authentic model of Tay-Sachs-related diseases.

Cachón-González MB, Wang SZ, Lynch A, Ziegler R, Cheng SH, Cox TM.

Proc Natl Acad Sci U S A. 2006 Jul 5;103(27):10373-8. Epub 2006 Jun 26.

19.

Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin.

Jeyakumar M, Butters TD, Cortina-Borja M, Hunnam V, Proia RL, Perry VH, Dwek RA, Platt FM.

Proc Natl Acad Sci U S A. 1999 May 25;96(11):6388-93.

20.

Disruption of murine Hexa gene leads to enzymatic deficiency and to neuronal lysosomal storage, similar to that observed in Tay-Sachs disease.

Cohen-Tannoudji M, Marchand P, Akli S, Sheardown SA, Puech JP, Kress C, Gressens P, Nassogne MC, Beccari T, Muggleton-Harris AL, et al.

Mamm Genome. 1995 Dec;6(12):844-9.

PMID:
8747922

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