Infusion of recombinant human acid sphingomyelinase into niemann-pick disease mice leads to visceral, but not neurological, correction of the pathophysiology.

@article{Miranda2000InfusionOR,
  title={Infusion of recombinant human acid sphingomyelinase into niemann-pick disease mice leads to visceral, but not neurological, correction of the pathophysiology.},
  author={Sandra Regina Miranda and Xin He and Calogera M. Simonaro and Shimon Gatt and A. Dagan and Robert J Desnick and Edward H Schuchman},
  journal={FASEB journal : official publication of the Federation of American Societies for Experimental Biology},
  year={2000},
  volume={14 13},
  pages={1988-95}
}
An inherited deficiency of acid sphingomyelinase (ASM) activity results in the Type A and B forms of Niemann-Pick disease (NPD). Using the ASM-deficient mouse model (ASMKO) of NPD, we evaluated the efficacy of enzyme replacement therapy (ERT) for the treatment of this disorder. Recombinant human ASM (rhASM) was purified from the media of overexpressing Chinese Hamster ovary cells and i.v. injected into 16 five-month-old ASMKO mice at doses of 0.3, 1, 3, or 10 mg/kg every other day for 14 days… CONTINUE READING
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