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pubmed-article:1684103pubmed:abstractTextMetachromatic 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.lld:pubmed
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pubmed-article:1684103pubmed:articleTitleRestoration of arylsulphatase A activity in human-metachromatic-leucodystrophy fibroblasts via retroviral-vector-mediated gene transfer.lld:pubmed
pubmed-article:1684103pubmed:affiliationUniversität Göttingen, Abteilung Biochemie II, Federal Republic of Germany.lld:pubmed
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pubmed-article:1684103pubmed:publicationTypeResearch Support, U.S. Gov't, P.H.S.lld:pubmed
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