Comparative Effects of Recombinant Acid Sphingomyelinase Administration by Different Routes in Niemann-Pick Disease Mice

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概要

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• 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.

• 社団法人　日本実験動物学会の論文

著者

• JANG Kwang-Ho

  Department of Veterinary Surgery, College of Veterinary Medicine, Kyungpook National University

• Bae Jae-sung

  College Of Veterinary Medicine Kyungpook National University

• Jang K‐h

  Kyungpook National Univ. Daegu Kor

• Jang Kwang-ho

  College Of Veterinary Medicine Kyungpook National University

• Jang Kwang-ho

  Department Of Veterinary Surgery College Of Veterinary Medicine Kyungpook National University

• SCHUCHMAN EDWARD

  Department of Human Genetics, Mount Sinai School of Medicine

• Jin Hee

  College Of Veterinary Medicine Kyungpook National University

• BAE Jae-Sung

  Department of Laboratory Animal Medicine and Department of Surgery, College of Veterinary Medicine,

• JIN Hee

  Department of Laboratory Animal Medicine and Department of Surgery, College of Veterinary Medicine,

• Jin Hee-kyung

  College Of Veterinary Medicine Kyungpook National University

• Schuchman Edward

  Department Of Human Genetics Mount Sinai School Of Medicine

• Schuchman Edward

  Department Of Genetics And Genomic Sciences Mount Sinai School Of Medicine

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