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Items: 1 to 20 of 251

1.
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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.

4.

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.

6.

Lentiviral-transduced human mesenchymal stem cells persistently express therapeutic levels of enzyme in a xenotransplantation model of human disease.

Meyerrose TE, Roberts M, Ohlemiller KK, Vogler CA, Wirthlin L, Nolta JA, Sands MS.

Stem Cells. 2008 Jul;26(7):1713-22. doi: 10.1634/stemcells.2008-0008. Epub 2008 Apr 24.

7.
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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
9.

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
10.

Phenotype correction in murine mucopolysaccharidosis type VII by transplantation of human amniotic epithelial cells after adenovirus-mediated gene transfer.

Kosuga M, Takahashi S, Sasaki K, Enosawa S, Li XK, Okuyama S, Fujino M, Suzuki S, Yamada M, Matsuo N, Sakuragawa N, Okuyama T.

Cell Transplant. 2000 Sep-Oct;9(5):687-92.

PMID:
11144966
11.

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
12.

In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors.

Stein CS, Kang Y, Sauter SL, Townsend K, Staber P, Derksen TA, Martins I, Qian J, Davidson BL, McCray PB Jr.

Mol Ther. 2001 Jun;3(6):850-6.

13.

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
14.
15.

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.

16.

Donor cell expansion is delayed following nonablative in utero transplantation to treat murine mucopolysaccharidosis type VII.

Barker JE, Schuldt AJ, Lessard ML, Jude CD, Vogler CA, Soper BW.

Exp Hematol. 2003 Nov;31(11):1112-8.

PMID:
14585377
17.

In utero fetal liver cell transplantation without toxic irradiation alleviates lysosomal storage in mice with mucopolysaccharidosis type VII.

Barker JE, Deveau S, Lessard M, Hamblen N, Vogler C, Levy B.

Blood Cells Mol Dis. 2001 Sep-Oct;27(5):861-73.

PMID:
11783949
18.

Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells.

Yoshimitsu M, Higuchi K, Ramsubir S, Nonaka T, Rasaiah VI, Siatskas C, Liang SB, Murray GJ, Brady RO, Medin JA.

Gene Ther. 2007 Feb;14(3):256-65. Epub 2006 Aug 24.

PMID:
16929352
19.

Gene therapy for murine mucopolysaccharidosis type VII.

Sands MS, Wolfe JH, Birkenmeier EH, Barker JE, Vogler C, Sly WS, Okuyama T, Freeman B, Nicholes A, Muzyczka N, Chang PL, Axelrod HR.

Neuromuscul Disord. 1997 Jul;7(5):352-60. Review.

PMID:
9267850
20.

Donor cell replacement in mice transplanted in utero is limited by immune-independent mechanisms.

Barker JE, Schuldt AJ, Lessard MD, Jude CD, Vogler CA, Soper BW.

Blood Cells Mol Dis. 2003 Sep-Oct;31(2):291-7.

PMID:
12972038
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