Global-scale control of gene expression in the adult mouse...

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Molecular Therapy 2017 / 8

Global-scale control of gene expression in the adult mouse nervous system by a single AAV-PHP.B systemic injection enables GBA1 gene therapy for complete protection from synucleinopathy

Morabito, Giuseppe, Giannelli, Serena, Ordazzo, Gabriele, Bido, Simone, Castoldi, Valerio, Indrigo, Marzia, Cabassi, Tommaso, Cattaneo, Stefano, Luoni, Mirko, Cancellieri, Cinzia, Sessa, Alessandro, B
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Language:
english
Journal:
Molecular Therapy
DOI:
10.1016/j.ymthe.2017.08.004
Date:
August, 2017
File:
PDF, 10.47 MB
english, 2017
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