Liver‐directed gene therapy for ornithine aminotransferase deficiency
Liver‐directed gene therapy for ornithine aminotransferase deficiency
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Publisher
London: Nature Publishing Group UK
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Language
English
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Publisher
London: Nature Publishing Group UK
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Contents
Gyrate atrophy of choroid and retina (GACR) is a chorioretinal degeneration caused by pathogenic variants in the gene encoding ornithine aminotransferase (OAT), an enzyme mainly expressed in liver. Affected patients have increased ornithine concentrations in blood and other body fluids and develop progressive constriction of vision fields leading t...
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Full title
Liver‐directed gene therapy for ornithine aminotransferase deficiency
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TN_cdi_doaj_primary_oai_doaj_org_article_e07eec5ef9114bf685675cb9215d0081
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_e07eec5ef9114bf685675cb9215d0081
Other Identifiers
ISSN
1757-4676,1757-4684
E-ISSN
1757-4684
DOI
10.15252/emmm.202217033
