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Items: 1 to 20 of 115

1.

AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival.

Baek RC, Broekman ML, Leroy SG, Tierney LA, Sandberg MA, d'Azzo A, Seyfried TN, Sena-Esteves M.

PLoS One. 2010 Oct 18;5(10):e13468. doi: 10.1371/journal.pone.0013468.

2.

Complete correction of enzymatic deficiency and neurochemistry in the GM1-gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery.

Broekman ML, Baek RC, Comer LA, Fernandez JL, Seyfried TN, Sena-Esteves M.

Mol Ther. 2007 Jan;15(1):30-7.

PMID:
17164772
3.

Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan.

Weismann CM, Ferreira J, Keeler AM, Su Q, Qui L, Shaffer SA, Xu Z, Gao G, Sena-Esteves M.

Hum Mol Genet. 2015 Aug 1;24(15):4353-64. doi: 10.1093/hmg/ddv168. Epub 2015 May 10.

4.

Mechanisms of distribution of mouse beta-galactosidase in the adult GM1-gangliosidosis brain.

Broekman ML, Tierney LA, Benn C, Chawla P, Cha JH, Sena-Esteves M.

Gene Ther. 2009 Feb;16(2):303-8. doi: 10.1038/gt.2008.149. Epub 2008 Sep 25.

PMID:
18818671
5.

N-butyldeoxygalactonojirimycin reduces neonatal brain ganglioside content in a mouse model of GM1 gangliosidosis.

Kasperzyk JL, El-Abbadi MM, Hauser EC, D'Azzo A, Platt FM, Seyfried TN.

J Neurochem. 2004 May;89(3):645-53.

6.

Substrate reduction reduces gangliosides in postnatal cerebrum-brainstem and cerebellum in GM1 gangliosidosis mice.

Kasperzyk JL, d'Azzo A, Platt FM, Alroy J, Seyfried TN.

J Lipid Res. 2005 Apr;46(4):744-51. Epub 2005 Feb 1.

7.

Beta-galactosidase-deficient mouse as an animal model for GM1-gangliosidosis.

Matsuda J, Suzuki O, Oshima A, Ogura A, Noguchi Y, Yamamoto Y, Asano T, Takimoto K, Sukegawa K, Suzuki Y, Naiki M.

Glycoconj J. 1997 Sep;14(6):729-36.

PMID:
9337086
8.

Generalized CNS disease and massive GM1-ganglioside accumulation in mice defective in lysosomal acid beta-galactosidase.

Hahn CN, del Pilar Martin M, Schröder M, Vanier MT, Hara Y, Suzuki K, Suzuki K, d'Azzo A.

Hum Mol Genet. 1997 Feb;6(2):205-11.

9.
10.

Transient high-level expression of beta-galactosidase after transfection of fibroblasts from GM1 gangliosidosis patients with plasmid DNA.

Balestrin RC, Baldo G, Vieira MB, Sano R, Coelho JC, Giugliani R, Matte U.

Braz J Med Biol Res. 2008 Apr;41(4):283-8.

11.

Neurochemical, morphological, and neurophysiological abnormalities in retinas of Sandhoff and GM1 gangliosidosis mice.

Denny CA, Alroy J, Pawlyk BS, Sandberg MA, d'Azzo A, Seyfried TN.

J Neurochem. 2007 Jun;101(5):1294-302. Epub 2007 Apr 17.

12.

Generation and characterization of recombinant feline beta-galactosidase for preclinical enzyme replacement therapy studies in GM1 gangliosidosis.

Samoylova TI, Martin DR, Morrison NE, Hwang M, Cochran AM, Samoylov AM, Baker HJ, Cox NR.

Metab Brain Dis. 2008 Jun;23(2):161-73. doi: 10.1007/s11011-008-9086-5. Epub 2008 Apr 18.

PMID:
18421424
13.

Attenuation of ganglioside GM1 accumulation in the brain of GM1 gangliosidosis mice by neonatal intravenous gene transfer.

Takaura N, Yagi T, Maeda M, Nanba E, Oshima A, Suzuki Y, Yamano T, Tanaka A.

Gene Ther. 2003 Aug;10(17):1487-93.

PMID:
12900764
14.

Beta-galactosidase deficiency in a Korat cat: a new form of feline GM1-gangliosidosis.

De Maria R, Divari S, Bo S, Sonnio S, Lotti D, Capucchio MT, Castagnaro M.

Acta Neuropathol. 1998 Sep;96(3):307-14.

PMID:
9754965
15.
16.

Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease.

Dodge JC, Clarke J, Song A, Bu J, Yang W, Taksir TV, Griffiths D, Zhao MA, Schuchman EH, Cheng SH, O'Riordan CR, Shihabuddin LS, Passini MA, Stewart GR.

Proc Natl Acad Sci U S A. 2005 Dec 6;102(49):17822-7. Epub 2005 Nov 21.

17.

Cerebrospinal fluid biomarkers showing neurodegeneration in dogs with GM1 gangliosidosis: possible use for assessment of a therapeutic regimen.

Satoh H, Yamato O, Asano T, Yonemura M, Yamauchi T, Hasegawa D, Orima H, Arai T, Yamasaki M, Maede Y.

Brain Res. 2007 Feb 16;1133(1):200-8. Epub 2006 Dec 28.

PMID:
17196562
18.

Chaperone therapy update: Fabry disease, GM1-gangliosidosis and Gaucher disease.

Suzuki Y.

Brain Dev. 2013 Jun;35(6):515-23. doi: 10.1016/j.braindev.2012.12.002. Epub 2013 Jan 3. Review.

PMID:
23290321
20.

Integration of active human beta-galactosidase gene (100 kb) into genome using HSV/AAV amplicon vector.

Oehmig A, Cortés ML, Perry KF, Sena-Esteves M, Fraefel C, Breakefield XO.

Gene Ther. 2007 Jul;14(14):1078-91. Epub 2007 Apr 26.

PMID:
17460718
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