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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
Osmon, Karlaina J.L., Woodley, Evan, Thompson, Patrick, Karumuthil-Melethil, Subha, Gray, Steven, Walia, JagdeepVolume:
24
Language:
english
Journal:
Molecular Therapy
DOI:
10.1016/s1525-0016(16)33172-0
Date:
May, 2016
File:
PDF, 549 KB
english, 2016