Article
Phenotype correction in murine mucopolysaccharidosis type VII by transplantation of human amniotic epithelial cells after adenovirus-mediated gene transfer.
Cell transplantation - 1 Jan 2000
Kosuga M, Takahashi S, Sasaki K, Enosawa S, Li X K, Okuyama S, Fujino M, Suzuki S, Yamada M, Matsuo N, Sakuragawa N, Okuyama T
Abstract excerpt
Cell therapy with human amniotic epithelial (HAE) cells was developed as an alternative method for enzyme replacement therapy in congenital lysosomal storage disorders, but only limited therapeutic efficacy has been reported. A major drawback is insufficient production and secretion of lysosomal enzymes from HAE cells. In this study, we infected HAE cells with an E1-deleted adenoviral vector expressing human...
Topics
- Adenoviridae
- Amnion
- Animals
- Cells, Cultured
- Epithelial Cells
- Gene Expression
- Genetic Therapy
- Glucuronidase
- Humans
- Liver
- Lysosomes