Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmal...
Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia
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Public Library of Science (PLoS)
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Language
English
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Public Library of Science (PLoS)
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Methylmalonic acidemia (MMA) is an inborn error of metabolism mostly caused by mutations in the mitochondrial methylmalonyl-CoA mutase gene (MMUT). MMA patients suffer from frequent episodes of metabolic decompensation, which can be life threatening. To mimic both the dietary restrictions and metabolic decompensation seen in MMA patients, we develo...
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Full title
Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia
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TN_cdi_doaj_primary_oai_doaj_org_article_b1d5a26130fa4ac4b29586136934a317
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_b1d5a26130fa4ac4b29586136934a317
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E-ISSN
1932-6203
DOI
10.1371/journal.pone.0274774
