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
Hu, Jie, Lu, Jui-Yun, Wong, Andrew M.S., Hynan, Linda S., Birnbaum, Shari G., Yilmaz, Denis S., Streit, Barbara M., Lenartowicz, Ewelina M., Thompson, Thomas C.M., Cooper, Jonathan D., Hofmann, Sandra L.
Published in Molecular genetics and metabolism (01.09.2012)
Published in Molecular genetics and metabolism (01.09.2012)
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