363. Improvement of Sandhoff Phenotype Following Intravenous Injection of Adeno-Associated Viral Vector Expressing a Hexosaminidase Isoenzyme in Adult Sandhoff Mice: Preclinical Safety and Efficacy Study

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Bibliographic Details
Published in:Molecular therapy 2016-05, Vol.24, p.S145-S146
Main Authors: Osmon, Karlaina J.L., Woodley, Evan, Thompson, Patrick, Karumuthil-Melethil, Subha, Gray, Steven, Walia, Jagdeep
Format: Article
Language:English
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ISSN:1525-0016
1525-0024
DOI:10.1016/S1525-0016(16)33172-0