Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

Author: Dominguez Elisa   Marais Thibaut   Chatauret Nicolas   Benkhelifa-Ziyyat Sofia   Duque Sandra   Ravassard Philippe   Carcenac Romain   Astord Stphanie   de Moura Aurlie Pereira   Voit Thomas   Barkats Martine  

Publisher: Oxford University Press

ISSN: 1460-2083

Source: Human Molecular Genetics, Vol.20, Iss.4, 2011-02, pp. : 681-693

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