http://purl.uniprot.org/citations/8348154 | http://www.w3.org/1999/02/22-rdf-syntax-ns#type | http://purl.uniprot.org/core/Journal_Citation |
http://purl.uniprot.org/citations/8348154 | http://www.w3.org/2000/01/rdf-schema#comment | "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."xsd:string |
http://purl.uniprot.org/citations/8348154 | http://purl.org/dc/terms/identifier | "doi:10.1038/ng0693-154"xsd:string |
http://purl.uniprot.org/citations/8348154 | http://purl.uniprot.org/core/author | "Danos O."xsd:string |
http://purl.uniprot.org/citations/8348154 | http://purl.uniprot.org/core/author | "Bohl D."xsd:string |
http://purl.uniprot.org/citations/8348154 | http://purl.uniprot.org/core/author | "Heard J.M."xsd:string |
http://purl.uniprot.org/citations/8348154 | http://purl.uniprot.org/core/author | "Moullier P."xsd:string |
http://purl.uniprot.org/citations/8348154 | http://purl.uniprot.org/core/date | "1993"xsd:gYear |
http://purl.uniprot.org/citations/8348154 | http://purl.uniprot.org/core/name | "Nat Genet"xsd:string |
http://purl.uniprot.org/citations/8348154 | http://purl.uniprot.org/core/pages | "154-159"xsd:string |
http://purl.uniprot.org/citations/8348154 | http://purl.uniprot.org/core/title | "Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts."xsd:string |
http://purl.uniprot.org/citations/8348154 | http://purl.uniprot.org/core/volume | "4"xsd:string |
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