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

Published 2014 View Full Article

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Keywords

Journal

HUMAN GENE THERAPY

Volume 25, Issue 6, Pages 506-516

Publisher

Mary Ann Liebert Inc

Online

2014-02-14

DOI

10.1089/hum.2013.238

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