Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial (Q42228547)

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scientific article published on 5 May 2014
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Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial
scientific article published on 5 May 2014

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    Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial (English)
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    Marc Tardieu
    Michel Zérah
    Béatrice Husson
    Stéphanie de Bournonville
    Catherine Adamsbaum
    Fanny Vincent
    Michael Hocquemiller
    Christine Broissand
    Valérie Furlan
    Alessandro Fraldi
    Ronald G Crystal
    Thomas Baugnon
    Thomas Roujeau
    Jean-Michel Heard
    Olivier Danos
    506-516

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