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Article

Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts.

Nature genetics - 1 Jun 1993

Moullier P, Bohl D, Heard J M, Danos O

Abstract excerpt

Genetic defects of lysosomal hydrolases result in severe storage diseases and treatments based on enzyme replacement have been proposed. In mice lacking beta-glucuronidase, which develop a disease homologous to human mucopolysaccharidosis type VII (Sly syndrome), we have used autologous implants of genetically-modified skin fibroblasts for the continuous in vivo production of the enzyme. The human...

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PMID: 8348154
DOI: 10.1038/ng0693-154

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