Nature Medicine
3, 719 - 720 (1997)
doi:10.1038/nm0797-719
Gene therapy for lysosomal storage disease: A no-brainer?William S. Sly1
& Carole Vogler2
1Edward A. Doisy Department of Biochemistry and Molecular Biology, St. Louis, MO 63104, USA
2Department of Pathology St. Louis University School of Medicine, St. Louis, MO 63104, USA Transplants of fibroblasts secreting high levels of  -glucuronidase decrease lesions in the brains of mice with Sly syndrome, a lysososmal storage disease (771−774).
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