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...
Topics
- Animals
- Cells, Cultured
- Disease Models, Animal
- Fibroblasts
- Genetic Therapy
- Genetic Vectors
- Glucuronidase
- Glycosaminoglycans
- Liver
- Lysosomes
- Mice
- Mice, Mutant Strains
- Mucopolysaccharidosis VII
- Organ Specificity
- Peritoneal Cavity
- Phenotype
- Prostheses and Implants
- Recombinant Fusion Proteins