Enzyme augmentation therapy enhances the therapeutic efficacy of bone marrow transplantation in mucopolysaccharidosis type II mice.

Abstract

Before the availability of an enzyme replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II), patients were treated by bone marrow transplantation (BMT). However, the effectiveness of BMT for MPS II was equivocal, particularly at addressing the CNS manifestations. To study this further, we subjected a murine model of MPS II to BMT and evaluated… (More)
DOI: 10.1016/j.ymgme.2013.09.013

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@article{Akiyama2014EnzymeAT, title={Enzyme augmentation therapy enhances the therapeutic efficacy of bone marrow transplantation in mucopolysaccharidosis type II mice.}, author={Kazumasa Akiyama and Yohta Shimada and Takashi Higuchi and Makoto Ohtsu and Hiromitsu Nakauchi and Hiroshi Kobayashi and Takahiro Fukuda and Hiroyuki Ida and Yoshikatsu Eto and Brett E. Crawford and Jillian R.M. Brown and Toya Ohashi}, journal={Molecular genetics and metabolism}, year={2014}, volume={111 2}, pages={139-46} }