(Q42228547)
Statements
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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
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Michel Zérah
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Béatrice Husson
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Stéphanie de Bournonville
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Catherine Adamsbaum
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Fanny Vincent
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Michael Hocquemiller
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Christine Broissand
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Valérie Furlan
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Alessandro Fraldi
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Ronald G Crystal
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Thomas Baugnon
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Thomas Roujeau
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Jean-Michel Heard
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Olivier Danos
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5 May 2014
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25
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6
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506-516
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Identifiers
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1 reference