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. 1991 Dec 1;280(Pt 2):459–461. doi: 10.1042/bj2800459

Restoration of arylsulphatase A activity in human-metachromatic-leucodystrophy fibroblasts via retroviral-vector-mediated gene transfer.

W Rommerskirch 1, A L Fluharty 1, C Peters 1, K von Figura 1, V Gieselmann 1
PMCID: PMC1130570  PMID: 1684103

Abstract

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.

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Selected References

These references are in PubMed. This may not be the complete list of references from this article.

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