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首页> 外文期刊>Experimental Animals >Comparative Effects of Recombinant Acid Sphingomyelinase Administration by Different Routes in Niemann-Pick Disease Mice
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Comparative Effects of Recombinant Acid Sphingomyelinase Administration by Different Routes in Niemann-Pick Disease Mice

机译：Niemann-Pick病小鼠不同途径重组酸性鞘磷脂酶给药的比较效果

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摘要

An inherited deficiency of acid sphingomyelinase (ASM) activity results in the Type A and B forms of Niemann-Pick disease (NPD). The aim of this study was to evaluate the effects of recombinant human ASM (rhASM) replacement therapy on the mouse model, by comparing different routes of administration. Eight NPD mice received rhASM via an intravenous injection (Ⅳ) administered at a dose of 1 mg/kg and another group of 8 NPD mice received the same dose by subcutaneous injection (SC). The plasma levels of ASM activity in intravenously administered mice were significantly elevated immediately after injection. In contrast, in the subcutaneously injected mice, the level of ASM activity was maximal 6 h after injection. The levels of ASM activity in both groups had declined substantially by 2 days after injection. It was concluded that rhASM administered by subcutaneous injection is completely absorbed, and offers a similar efficacy to intravenously administered recombinant enzyme.
机译：遗传的酸性鞘磷脂酶（ASM）活性缺陷导致A型和B型Niemann-Pick病（NPD）。这项研究的目的是通过比较不同的给药途径来评估重组人ASM（rhASM）替代疗法对小鼠模型的作用。八只NPD小鼠通过静脉注射（Ⅳ）以1 mg / kg的剂量接受rhASM，另一组八只NPD小鼠通过皮下注射（SC）接受相同剂量。注射后，静脉内给药的小鼠血浆中ASM活性水平明显升高。相反，在皮下注射的小鼠中，注射后6 h ASM活性最高。两组在注射后2天的ASM活性水平已大大下降。结论是通过皮下注射施用的rhASM被完全吸收，并提供与静脉内施用的重组酶相似的功效。

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