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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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Autori principali: Iolanda Boffa, Elena Polishchuk, Lucia De Stefano, Fabio Dell'Aquila, Edoardo Nusco, Elena Marrocco, Matteo Audano, Silvia Pedretti, Marianna Caterino, Ilaria Bellezza, Margherita Ruoppolo, Nico Mitro, Barbara Cellini, Alberto Auricchio, Nicola Brunetti‐Pierri
Natura: Artigo
Lingua:Inglês
Pubblicazione: Springer Nature 2023-01-01
Serie:EMBO Molecular Medicine
Soggetti:
AAV
gyrate atrophy of choroid and retina
inherited metabolic diseases
liver gene therapy
ornithine aminotransferase
Accesso online:https://doi.org/10.15252/emmm.202217033
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