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Items: 1 to 20 of 90

1.

Sustained normalization of neurological disease after intracranial gene therapy in a feline model.

McCurdy VJ, Johnson AK, Gray-Edwards HL, Randle AN, Brunson BL, Morrison NE, Salibi N, Johnson JA, Hwang M, Beyers RJ, Leroy SG, Maitland S, Denney TS, Cox NR, Baker HJ, Sena-Esteves M, Martin DR.

Sci Transl Med. 2014 Apr 9;6(231):231ra48. doi: 10.1126/scitranslmed.3007733.

2.

Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease.

Bradbury AM, Gray-Edwards HL, Shirley JL, McCurdy VJ, Colaco AN, Randle AN, Christopherson PW, Bird AC, Johnson AK, Wilson DU, Hudson JA, De Pompa NL, Sorjonen DC, Brunson BL, Jeyakumar M, Platt FM, Baker HJ, Cox NR, Sena-Esteves M, Martin DR.

Exp Neurol. 2015 Jan;263:102-12. doi: 10.1016/j.expneurol.2014.09.020. Epub 2014 Oct 5.

3.

Molecular consequences of the pathogenic mutation in feline GM1 gangliosidosis.

Martin DR, Rigat BA, Foureman P, Varadarajan GS, Hwang M, Krum BK, Smith BF, Callahan JW, Mahuran DJ, Baker HJ.

Mol Genet Metab. 2008 Jun;94(2):212-21. doi: 10.1016/j.ymgme.2008.02.004. Epub 2008 Mar 18.

4.

AAV-mediated gene delivery in a feline model of Sandhoff disease corrects lysosomal storage in the central nervous system.

Rockwell HE, McCurdy VJ, Eaton SC, Wilson DU, Johnson AK, Randle AN, Bradbury AM, Gray-Edwards HL, Baker HJ, Hudson JA, Cox NR, Sena-Esteves M, Seyfried TN, Martin DR.

ASN Neuro. 2015 Apr 13;7(2). pii: 1759091415569908. doi: 10.1177/1759091415569908. Print 2015 Mar-Apr.

5.
6.

Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.

McCurdy VJ, Rockwell HE, Arthur JR, Bradbury AM, Johnson AK, Randle AN, Brunson BL, Hwang M, Gray-Edwards HL, Morrison NE, Johnson JA, Baker HJ, Cox NR, Seyfried TN, Sena-Esteves M, Martin DR.

Gene Ther. 2015 Feb;22(2):181-9. doi: 10.1038/gt.2014.108. Epub 2014 Dec 4.

PMID:
25474439
7.

Development of lysosomal storage in mice with targeted disruption of the beta-galactosidase gene: a model of human G(M1)-gangliosidosis.

Itoh M, Matsuda J, Suzuki O, Ogura A, Oshima A, Tai T, Suzuki Y, Takashima S.

Brain Dev. 2001 Oct;23(6):379-84.

PMID:
11578847
8.

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.

9.

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.

10.

Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model.

Gray-Edwards HL, Regier DS, Shirley JL, Randle AN, Salibi N, Thomas SE, Latour YL, Johnston J, Golas G, Maguire AS, Taylor AR, Sorjonen DC, McCurdy VJ, Christopherson PW, Bradbury AM, Beyers RJ, Johnson AK, Brunson BL, Cox NR, Baker HJ, Denney TS, Sena-Esteves M, Tifft CJ, Martin DR.

Mol Ther. 2017 Apr 5;25(4):892-903. doi: 10.1016/j.ymthe.2017.01.009. Epub 2017 Feb 22.

11.

GM1-gangliosidosis in American black bears: clinical, pathological, biochemical and molecular genetic characterization.

Muthupalani S, Torres PA, Wang BC, Zeng BJ, Eaton S, Erdelyi I, Ducore R, Maganti R, Keating J, Perry BJ, Tseng FS, Waliszewski N, Pokras M, Causey R, Seger R, March P, Tidwell A, Pfannl R, Seyfried T, Kolodny EH, Alroy J.

Mol Genet Metab. 2014 Apr;111(4):513-21. doi: 10.1016/j.ymgme.2014.02.002. Epub 2014 Feb 13.

PMID:
24581871
12.

Insights into post-translational processing of beta-galactosidase in an animal model resembling late infantile human G-gangliosidosis.

Kreutzer R, Kreutzer M, Pröpsting MJ, Sewell AC, Leeb T, Naim HY, Baumgärtner W.

J Cell Mol Med. 2008 Sep-Oct;12(5A):1661-71. Epub 2007 Dec 14.

13.

Lysosomal accumulation of Trk protein in brain of GM₁ -gangliosidosis mouse and its restoration by chemical chaperone.

Takamura A, Higaki K, Ninomiya H, Takai T, Matsuda J, Iida M, Ohno K, Suzuki Y, Nanba E.

J Neurochem. 2011 Aug;118(3):399-406. doi: 10.1111/j.1471-4159.2011.07310.x. Epub 2011 Jun 17.

14.

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.

PMID:
9063740
15.

Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy.

Rigat BA, Tropak MB, Buttner J, Crushell E, Benedict D, Callahan JW, Martin DR, Mahuran DJ.

Mol Genet Metab. 2012 Sep;107(1-2):203-12. doi: 10.1016/j.ymgme.2012.06.007. Epub 2012 Jun 19.

16.

Hepatic beta galactosidase and feline GMI gangliosidosis.

Barnes IC, Kelly DF, Pennock CA, Randell JA.

Neuropathol Appl Neurobiol. 1981 Nov-Dec;7(6):463-76.

PMID:
6799849
17.

Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.

Hinderer C, Bell P, Gurda BL, Wang Q, Louboutin JP, Zhu Y, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Haskins ME, Wilson JM.

Mol Ther. 2014 Dec;22(12):2018-27. doi: 10.1038/mt.2014.135. Epub 2014 Jul 16.

18.

Diagnosis of feline GM1 gangliosidosis by enzyme assay of cultured conjunctival cells.

Nowakowski RW, Thompson JN, Baker HJ.

Invest Ophthalmol Vis Sci. 1988 Mar;29(3):487-90.

PMID:
3125124
19.

Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase:RTB lectin fusion.

Condori J, Acosta W, Ayala J, Katta V, Flory A, Martin R, Radin J, Cramer CL, Radin DN.

Mol Genet Metab. 2016 Feb;117(2):199-209. doi: 10.1016/j.ymgme.2015.12.002. Epub 2015 Dec 8.

PMID:
26766614
20.

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