Gene therapy using a liver-targeted AAV vector restores...

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Molecular Therapy 2014 / 01

Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE

Torres-Torronteras, Javier, Viscomi, Carlo, Cabrera-Pérez, Raquel, Cámara, Yolanda, Di Meo, Ivano, Barquinero, Jordi, Auricchio, Alberto, Pizzorno, Giuseppe, Hirano, Michio, Zeviani, Massimo, Martí, R
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Language:
english
Journal:
Molecular Therapy
DOI:
10.1038/mt.2014.6
Date:
January, 2014
File:
PDF, 1003 KB
english, 2014
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