Article
Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells.
Human gene therapy - 10 Oct 2000
Ross C J, Bastedo L, Maier S A, Sands M S, Chang P L
Abstract excerpt
Most lysosomal enzyme deficiencies are catastrophic illnesses with no generally available treatments. We have used the beta-glucuronidase-deficient mouse model of mucopolysaccharidosis type VII (MPS VII) to develop an alternative approach to therapy. A "universal" cell line engineered to secrete the missing enzyme is implanted in all recipients requiring the same enzyme replacement. The cells, although...
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