Expression and processing of mature human frataxin after gene therapy in mice
Expression and processing of mature human frataxin after gene therapy in mice
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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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Friedreich’s ataxia is a degenerative and progressive multisystem disorder caused by mutations in the highly conserved frataxin (FXN) gene that results in FXN protein deficiency and mitochondrial dysfunction. While gene therapy approaches are promising, consistent induction of therapeutic FXN protein expression that is sub-toxic has proven challeng...
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Expression and processing of mature human frataxin after gene therapy in mice
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TN_cdi_doaj_primary_oai_doaj_org_article_86223bf35bfc4a128468812ab0bc1d9b
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_86223bf35bfc4a128468812ab0bc1d9b
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ISSN
2045-2322
E-ISSN
2045-2322
DOI
10.1038/s41598-024-59060-0
