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Liver‐directed gene therapy for ornithine aminotransferase deficiency

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 p...

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Detalles Bibliográficos
Autores principales:	Boffa, Iolanda, Polishchuk, Elena, De Stefano, Lucia, Dell'Aquila, Fabio, Nusco, Edoardo, Marrocco, Elena, Audano, Matteo, Pedretti, Silvia, Caterino, Marianna, Bellezza, Ilaria, Ruoppolo, Margherita, Mitro, Nico, Cellini, Barbara, Auricchio, Alberto, Brunetti‐Pierri, Nicola
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2023
Materias:	Reports
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10086579/ https://www.ncbi.nlm.nih.gov/pubmed/36647689 http://dx.doi.org/10.15252/emmm.202217033

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