Gene therapy for lysosomal storage disease: A no-brainer?

[1] Sly WS(1973)β-glucuronidase deficiency: Report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis J. Pediatr. 82 249-257

[2] Quinton BA(1997)Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fi broblasts secreting high levels of β-glucuronidase Nature Med. 3 771-774

[3] McAlister WH(1989)Murine mucopolysacchari dosis VII: Characterization of a mouse with β-glucuronidase deficiency J. Clin Invest. 83 1258-1266

[4] Rimoin DL(1991)Increased lifespan and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation Blood 78 3081-3092

[5] Taylor RM(1994)Enzyme replacement therapy for murine mucopolysaccharidosis type VII J. Clin. Invest. 93 2324-2331

[6] Wolfe JH(1997)Murine mucopolysaccharidosis type VII: Long-term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation J. Clin. Invest. 99 1596-1605

[7] Birkenmeier EH(1993)Gene therapy on the Sly Nature Genet. 4 105-106

[8] Birkenmeier EH(1995)Phenotypic correction in retinal pigment epithelium in murine mucopolysac charidosis VII by adenovirus-mediated gene transfer Proc Natl. Acad. Sci. U.S.A. 92 7700-7704

[9] Sands MS(1997)Adenovirus-mediated gene transfer and expression of human β-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice Proc. Natl. Acad. Sci. U.S.A. 94 1287-1292

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[1] Sly WS(1973)β-glucuronidase deficiency: Report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis J. Pediatr. 82 249-257

[2] Quinton BA(1997)Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fi broblasts secreting high levels of β-glucuronidase Nature Med. 3 771-774

[3] McAlister WH(1989)Murine mucopolysacchari dosis VII: Characterization of a mouse with β-glucuronidase deficiency J. Clin Invest. 83 1258-1266

[4] Rimoin DL(1991)Increased lifespan and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation Blood 78 3081-3092

[5] Taylor RM(1994)Enzyme replacement therapy for murine mucopolysaccharidosis type VII J. Clin. Invest. 93 2324-2331

[6] Wolfe JH(1997)Murine mucopolysaccharidosis type VII: Long-term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation J. Clin. Invest. 99 1596-1605

[7] Birkenmeier EH(1993)Gene therapy on the Sly Nature Genet. 4 105-106

[8] Birkenmeier EH(1995)Phenotypic correction in retinal pigment epithelium in murine mucopolysac charidosis VII by adenovirus-mediated gene transfer Proc Natl. Acad. Sci. U.S.A. 92 7700-7704

[9] Sands MS(1997)Adenovirus-mediated gene transfer and expression of human β-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice Proc. Natl. Acad. Sci. U.S.A. 94 1287-1292

[10] Sands MS(undefined)undefined undefined undefined undefined-undefined