TY - JOUR
T1 - Comparative effects of recombinant acid sphingomyelinase administration by different routes in Niemann-Pick disease mice
AU - Bae, Jae Sung
AU - Jang, Kwang Ho
AU - Schuchman, Edward H.
AU - Jin, Hee Kyung
PY - 2004
Y1 - 2004
N2 - 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.
AB - 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.
KW - Acid sphingomyelinase
KW - Enzyme replacement therapy
KW - Lysosomal storage disorder
UR - http://www.scopus.com/inward/record.url?scp=16644383960&partnerID=8YFLogxK
U2 - 10.1538/expanim.53.417
DO - 10.1538/expanim.53.417
M3 - Article
C2 - 15516789
AN - SCOPUS:16644383960
SN - 1341-1357
VL - 53
SP - 417
EP - 421
JO - Experimental Animals
JF - Experimental Animals
IS - 5
ER -