Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type...
Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type 9 AAV vector encoding arylsulfatase A
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London: Nature Publishing Group UK
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English
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London: Nature Publishing Group UK
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Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by an arylsulfatase A (ARSA) deficiency and characterized by severe neurological symptoms resulting from demyelination within the central and peripheral nervous systems. We investigated the feasibility and efficacy of intrathecal administration of a type 9 adeno-associated vir...
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Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type 9 AAV vector encoding arylsulfatase A
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TN_cdi_doaj_primary_oai_doaj_org_article_4dd8f9e3c6f542a4b4d9938ecef5e221
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_4dd8f9e3c6f542a4b4d9938ecef5e221
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ISSN
2045-2322
E-ISSN
2045-2322
DOI
10.1038/s41598-021-99979-2
