Dual-function AAV gene therapy reverses late-stage Canavan disease pathology in mice
Dual-function AAV gene therapy reverses late-stage Canavan disease pathology in mice
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Publisher
Switzerland: Frontiers Media S.A
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Language
English
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Publisher
Switzerland: Frontiers Media S.A
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Contents
The leukodystrophy Canavan disease is a fatal white matter disorder caused by loss-of-function mutations of the aspartoacylase-encoding
gene. There are no effective treatments available and experimental gene therapy trials have failed to provide sufficient amelioration from Canavan disease symptoms. Preclinical studies suggest that Canavan disea...
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Full title
Dual-function AAV gene therapy reverses late-stage Canavan disease pathology in mice
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TN_cdi_doaj_primary_oai_doaj_org_article_537c8c0bb7154f5697810c52b8705019
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_537c8c0bb7154f5697810c52b8705019
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ISSN
1662-5099
E-ISSN
1662-5099
DOI
10.3389/fnmol.2022.1061257
