A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

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作者： Giuseppe,Ronzitti ^[1] ; Giulia,Bortolussi ^[2] ; Remco,van Dijk ^[3] ; Fanny,Collaud ^[1] ; Severine,Charles ^[1] ; Christian,Leborgne ^[1] ; Patrice,Vidal ^[4] ; Samia,Martin ^[1] ; Bernard,Gjata ^[1] ; Marcelo Simon,Sola ^[1] ; Laetitia,van Wittenberghe ^[1] ; Alban,Vignaud ^[1] ; Philippe,Veron ^[1] ; Piter J,Bosma ^[3] ; Andres F,Muro ^[2] ; Federico,Mingozzi ^[5]

作者单位： Genethon , Evry, France. ^[1] International Centre for Genetic Engineering and Biotechnology , Trieste, Italy. ^[2] Tytgat Institute for Liver and Intestinal Research, Academic Medical Center , Amsterdam, the Netherlands. ^[3] Genethon, Evry, France; Universite' Pierre et Marie Curie - Paris 6, Paris, France. ^[4] Genethon, Evry, France; Universite' Pierre et Marie Curie - Paris 6, Paris, France; INSERM U951, Evry, France. ^[5]

DOI 10.1038/mtm.2016.49

PMID 27722180

发布时间 2022-01-29

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Molecular therapy. Methods & clinical development

2016年3卷

16049页

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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

相关期刊

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