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Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl–protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis
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. HofmannVolume:
107
Year:
2012
Language:
english
DOI:
10.1016/j.ymgme.2012.05.009
File:
PDF, 1.62 MB
english, 2012