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Daly demonstrated that a single treatment at birth could completely prevent the development of clinical disease in a murine model of mucopolysaccharidosis type VII, a lysosomal storage disorder.
With the exception of the formation of liver tumors in some mice, the results of the gene therapy experiments in mice engineered to mimic a human disease called mucopolysaccharidosis type VII were encouraging.
The mice were born with mucopolysaccharidosis Type VII, a disfiguring and eventually fatal disease caused by mutations in the genes that code for beta-glucuronidase.