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. Hofmann

Research output: Contribution to journalArticlepeer-review

34 Scopus citations

Fingerprint

Dive into the research topics of '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'. Together they form a unique fingerprint.

Medicine & Life Sciences

Chemical Compounds