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Items: 1 to 20 of 288

1.

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.

2.

Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

Frisella WA, O'Connor LH, Vogler CA, Roberts M, Walkley S, Levy B, Daly TM, Sands MS.

Mol Ther. 2001 Mar;3(3):351-8.

3.

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.

PMID:
15233947
5.

Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse.

Sferra TJ, Backstrom K, Wang C, Rennard R, Miller M, Hu Y.

Mol Ther. 2004 Sep;10(3):478-91.

PMID:
15336648
7.

Engraftment of genetically engineered amniotic epithelial cells corrects lysosomal storage in multiple areas of the brain in mucopolysaccharidosis type VII mice.

Kosuga M, Sasaki K, Tanabe A, Li XK, Okawa H, Ogino I, Okuda O, Arai H, Sakuragawa N, Kamata Y, Azuma N, Suzuki S, Yamada M, Okuyama T.

Mol Ther. 2001 Feb;3(2):139-48.

8.

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.

9.
11.

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
12.

Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

Xu L, Mango RL, Sands MS, Haskins ME, Ellinwood NM, Ponder KP.

Mol Ther. 2002 Dec;6(6):745-58.

PMID:
12498771
13.

Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS.

Liu G, Chen YH, He X, Martins I, Heth JA, Chiorini JA, Davidson BL.

Mol Ther. 2007 Feb;15(2):242-7.

PMID:
17235300
14.

High level expression and export of beta-glucuronidase from murine mucopolysaccharidosis VII cells corrected by a double-copy retrovirus vector.

Wolfe JH, Kyle JW, Sands MS, Sly WS, Markowitz DG, Parente MK.

Gene Ther. 1995 Jan;2(1):70-8.

PMID:
7712336
15.

Brain transplantation of genetically modified bone marrow stromal cells corrects CNS pathology and cognitive function in MPS VII mice.

Sakurai K, Iizuka S, Shen JS, Meng XL, Mori T, Umezawa A, Ohashi T, Eto Y.

Gene Ther. 2004 Oct;11(19):1475-81.

PMID:
15295619
16.

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

Liu G, Martins I, Wemmie JA, Chiorini JA, Davidson BL.

J Neurosci. 2005 Oct 12;25(41):9321-7.

17.
18.

Encapsulation cell therapy for mucopolysaccharidosis type VII using genetically engineered immortalized human amniotic epithelial cells.

Nakama H, Ohsugi K, Otsuki T, Date I, Kosuga M, Okuyama T, Sakuragawa N.

Tohoku J Exp Med. 2006 May;209(1):23-32.

19.

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
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