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Items: 1 to 20 of 137

1.

AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice.

Hennig AK, Ogilvie JM, Ohlemiller KK, Timmers AM, Hauswirth WW, Sands MS.

Mol Ther. 2004 Jul;10(1):106-16.

2.

Retinal function is improved in a murine model of a lysosomal storage disease following bone marrow transplantation.

Ohlemiller KK, Vogler CA, Roberts M, Galvin N, Sands MS.

Exp Eye Res. 2000 Nov;71(5):469-81.

PMID:
11040082
3.

Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII.

Donsante A, Levy B, Vogler C, Sands MS.

J Inherit Metab Dis. 2007 Apr;30(2):227-38.

PMID:
17308887
4.

Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse.

Sferra TJ, Qu G, McNeely D, Rennard R, Clark KR, Lo WD, Johnson PR.

Hum Gene Ther. 2000 Mar 1;11(4):507-19.

PMID:
10724030
5.

Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII.

Bielicki J, McIntyre C, Anson DS.

Mol Genet Metab. 2010 Dec;101(4):370-82. doi: 10.1016/j.ymgme.2010.08.013.

PMID:
20864369
7.

Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease.

Vogler C, Sands MS, Galvin N, Levy B, Thorpe C, Barker J, Sly WS.

J Inherit Metab Dis. 1998 Aug;21(5):575-86. Review.

PMID:
9728337
8.

Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer.

Daly TM, Ohlemiller KK, Roberts MS, Vogler CA, Sands MS.

Gene Ther. 2001 Sep;8(17):1291-8.

9.
10.

Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.

Gurda BL, De Guilhem De Lataillade A, Bell P, Zhu Y, Yu H, Wang P, Bagel J, Vite CH, Sikora T, Hinderer C, Calcedo R, Yox AD, Steet RA, Ruane T, O'Donnell P, Gao G, Wilson JM, Casal M, Ponder KP, Haskins ME.

Mol Ther. 2016 Feb;24(2):206-16. doi: 10.1038/mt.2015.189.

11.

Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus.

Watson GL, Sayles JN, Chen C, Elliger SS, Elliger CA, Raju NR, Kurtzman GJ, Podsakoff GM.

Gene Ther. 1998 Dec;5(12):1642-9.

12.

Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye.

Li W, Kong F, Li X, Dai X, Liu X, Zheng Q, Wu R, Zhou X, Lü F, Chang B, Li Q, Hauswirth WW, Qu J, Pang JJ.

Mol Vis. 2009;15:267-75.

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16.

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

Daly TM, Vogler C, Levy B, Haskins ME, Sands MS.

Proc Natl Acad Sci U S A. 1999 Mar 2;96(5):2296-300.

17.

Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs.

Mango RL, Xu L, Sands MS, Vogler C, Seiler G, Schwarz T, Haskins ME, Ponder KP.

Mol Genet Metab. 2004 May;82(1):4-19.

PMID:
15110316
18.

Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

Hennig AK, Levy B, Ogilvie JM, Vogler CA, Galvin N, Bassnett S, Sands MS.

J Neurosci. 2003 Apr 15;23(8):3302-7.

19.

Feline immunodeficiency virus vectors. Gene transfer to mouse retina following intravitreal injection.

Derksen TA, Sauter SL, Davidson BL.

J Gene Med. 2002 Sep-Oct;4(5):463-9.

PMID:
12221638
20.

Adenoviral vector-mediated beta-glucuronidase cDNA transfer to treat MPS VII RPE in vitro.

Verdugo ME, Scarpino V, Moullier P, Haskins ME, Aguirre GD, Ray J.

Curr Eye Res. 2001 Nov;23(5):357-67.

PMID:
11910525
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