Liver‐directed gene therapy for ornithine aminotransferase deficiency
Abstract 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...
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| Autors principals: | , , , , , , , , , , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
Springer Nature
2023-01-01
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| Col·lecció: | EMBO Molecular Medicine |
| Matèries: | |
| Accés en línia: | https://doi.org/10.15252/emmm.202217033 |
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