Liver‐directed gene therapy for ornithine aminotransferase deficiency

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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Main Authors: 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
Format: Article
Language:English
Published: Springer Nature 2023-01-01
Series:EMBO Molecular Medicine
Subjects:
AAV
gyrate atrophy of choroid and retina
inherited metabolic diseases
liver gene therapy
ornithine aminotransferase
Online Access:https://doi.org/10.15252/emmm.202217033
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https://doi.org/10.15252/emmm.202217033

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