Restoration of arylsulphatase A activity in human-metachromatic-leucodystrophy fibroblasts via retroviral-vector-mediated gene transfer
| Autor: | K von Figura, Christoph Peters, W Rommerskirch, Volkmar Gieselmann, A L Fluharty |
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| Rok vydání: | 1991 |
| Předmět: |
Arylsulfatase A
viruses Cell Fractionation Transfection Biochemistry Viral vector Cell Line 03 medical and health sciences 0302 clinical medicine Retrovirus Cerebrosides Lysosomal storage disease medicine Humans Molecular Biology Cerebroside-Sulfatase 030304 developmental biology 0303 health sciences biology Hydrolysis Genetic transfer Cell Biology DNA Leukodystrophy Metachromatic Provirus Fibroblasts biology.organism_classification medicine.disease Molecular biology Precipitin Tests 3. Good health Metachromatic leukodystrophy Retroviridae 030220 oncology & carcinogenesis Research Article |
| Zdroj: | The Biochemical journal. 280 |
| ISSN: | 0264-6021 |
| Popis: | Metachromatic leukodystrophy is a lysosomal storage disease caused by the deficiency of arylsulphatase A (ASA). A human ASA cDNA was subcloned into the retroviral vector pXT1. Replication-defective retrovirus was generated by transfection of the vector into the amphotropic packaging cell line PA317. Human fibroblasts from a patient suffering from metachromatic leucodystrophy was infected with the recombinant retrovirus. Infected fibroblasts expressed ten times more ASA compared with control fibroblasts from a normal individual. The ASA encoded by the integrated provirus was shown to be correctly transported into the lysosomes and to normalize the impaired degradation of cerebroside sulphate. |
| Databáze: | OpenAIRE |
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