title: Comparative effects of recombinant acid sphingomyelinase administration by different routes in niemann-pick disease mice.
abstract: 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.
authors.affiliation: Department of Laboratory Animal Medicine, College of Veterinary Medicine, Kyungpook National University, Daegu, Korea.
authors.forename: Jae-Sung
authors.initials: JS
authors.lastname: Bae
authors.forename: Kwang-Ho
authors.initials: KH
authors.lastname: Jang
authors.forename: Toru R
authors.initials: TR
authors.lastname: Schuchman
authors.forename: Hee Kyung
authors.initials: HK
authors.lastname: Jin
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D007275:Injections, Intravenous; D007279:Injections, Subcutaneous; D051379:Mice; D009542:Niemann-Pick Diseases / Q000188:drug therapy* / Q000209:etiology; D011994:Recombinant Proteins / Q000008:administration & dosage; D013108:Sphingomyelin Phosphodiesterase / Q000008:administration & dosage* / Q000172:deficiency / Q000493:pharmacokinetics; D013997:Time Factors; D014018:Tissue Distribution
chemical_list: D011994:Recombinant Proteins; D013108:Sphingomyelin Phosphodiesterase