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Liver-directed Gene Therapy for Ornithine Aminotransferase Deficiency

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

EMBO molecular medicine（2023）

引用 3|浏览57
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关键词
AAV,gyrate atrophy of choroid and retina,inherited metabolic diseases,liver gene therapy,ornithine aminotransferase
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