| First Author | Moullier P | Year | 1993 |
| Journal | Nat Genet | Volume | 4 |
| Issue | 2 | Pages | 154-9 |
| PubMed ID | 8348154 | Mgi Jnum | J:11889 |
| Mgi Id | MGI:60159 | Doi | 10.1038/ng0693-154 |
| Citation | Moullier P, et al. (1993) Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts [see comments]. Nat Genet 4(2):154-9 |
| abstractText | 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 beta-glucuronidase cDNA was introduced with a retroviral vector into mutant mice skin fibroblasts grown in primary culture. Fourteen mutant mice were implanted intraperitoneally with these modified cells embedded into collagen lattices. All animals expressed beta-glucuronidase from the vascularized neo-organs that developed after implantation and accumulated the enzyme in their tissues. A complete disappearance of the lysosomal storage lesions was observed in their liver and spleen. |
