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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Publisher
San Francisco: Public Library of Science
Journal title
Language
English
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Publisher
San Francisco: Public Library of Science
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Contents
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_plos_journals_2716058971
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_2716058971
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ISSN
1932-6203
E-ISSN
1932-6203
DOI
10.1371/journal.pone.0274774
