首页> 外文OA文献 >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

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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

机译：在婴儿神经元类脑脂质脂褐变的临床前小鼠模型中，使用重组棕榈酰蛋白硫酯酶的静脉内大剂量酶替代疗法可减少内脏溶酶体贮藏并适度延长生存期

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Hu, Jie; Lu, Jui-Yun; Wong, Andrew M. S.; Hynan, Linda S.; Birnbaum, Shari G.; Yilmaz, Denis S.; Streit, Barbara M.; Lenartowicz, Ewelina; Thompson, Thomas C. M.; Cooper, Jonathan D.; Hofmann, Sandra L.;
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年度 2012
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1. 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 [J] . HuJ., LuJ.-Y., WongA.M.S., Molecular genetics and metabolism . 2012,第1a2期

机译：在婴儿神经元类脑脂质脂褐变的临床前小鼠模型中，重组棕榈酰蛋白硫酯酶的静脉内大剂量酶替代疗法减少了内脏溶酶体的储存并适度延长了生存期
2. Human recombinant palmitoyl-protein thioesterase-1 (PPT1) for preclinical evaluation of enzyme replacement therapy for infantile neuronal ceroid lipofuscinosis. [J] . Lu JY, Hu J, Hofmann SL Molecular genetics and metabolism . 2010,第4期

机译：人类重组棕榈酰蛋白硫酯酶-1（PPT1）用于婴幼儿神经元类固醇脂褐变病的酶替代治疗的临床前评估。
3. Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis [J] . Lu Jui-Yun, Nelvagal Hemanth R., Wang Lingling, Molecular genetics and metabolism . 2015,第1a2期

机译：鞘内酶替代疗法可改善小儿神经元类固醇脂褐变的临床前小鼠模型的运动功能和存活率
4. Gene therapy and enzyme replacement in a mouse model of late infantile neuronal ceroid lipofuscinosis. [D] . Chang, Michael Chia Hsiu. 2009

机译：晚期婴儿神经元类固醇脂褐藻病小鼠模型中的基因治疗和酶替代。
5. 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 [O] . Jie Hu, Jui-Yun Lu, Andrew M.S. Wong, -1

机译：具有重组棕榈酰蛋白硫代酯酶的静脉注射高剂量酶替代疗法减少了内脏溶酶体储存在婴儿神经元曲线胰管型唇脑病的临床前小鼠模型中适度延长存活
6. Human recombinant palmitoyl-protein thioesterase-1 (PPT1) for preclinical evaluation of enzyme replacement therapy for infantile neuronal ceroid lipofuscinosis [O] . Jui-Yun Lu, Jie Hu, Sandra L. Hofmann 2010

机译：人类重组棕榈酰蛋白硫代酯酶-1（PPT1）用于婴儿神经元曲线胰管型酶替代治疗的临床前评价

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

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