Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8344115 | Molecular Genetics and Metabolism | 2012 | 9 Pages |
Abstract
⺠PPT1 knockout mice received intravenous weekly human PPT1 from birth or age 8 weeks. ⺠The treatment was well tolerated without anaphylaxis or antibody formation. ⺠Survival increased and motor deterioration was delayed in mice treated from birth. ⺠Substantial clearance of autofluorescent storage from visceral organs was observed.
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Authors
Jie Hu, Jui-Yun Lu, Andrew M.S. Wong, Linda S. Hynan, Shari G. Birnbaum, Denis S. Yilmaz, Barbara M. Streit, Ewelina M. Lenartowicz, Thomas C.M. Thompson, Jonathan D. Cooper, Sandra L. Hofmann,