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

Article ID	Journal	Published Year	Pages	File Type
8344115	Molecular Genetics and Metabolism	2012	9 Pages	PDF

Abstract

âº PPT1 knockout mice received intravenous weekly human PPT1 from birth or age 8Â weeks. âº The treatment was well tolerated without anaphylaxis or antibody formation. âº Survival increased and motor deterioration was delayed in mice treated from birth. âº Substantial clearance of autofluorescent storage from visceral organs was observed.

Keywords

PBS FBS NCL CLN1 PPT1 Palmitoyl-protein thioesterase-1 GROD neuronal ceroid lipofuscinosis enzyme replacement therapy Lysosomal storage disorder Batten disease granular osmiophilic deposits fetal bovine serum Infantile neuronal ceroid lipofuscinosis mannose 6-phosphate Phosphate-buffered saline

Related Topics

Life Sciences Biochemistry, Genetics and Molecular Biology Biochemistry

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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

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,