Activation of human acid sphingomyelinase through modification or deletion of C-terminal cysteine.

@article{Qiu2003ActivationOH,
  title={Activation of human acid sphingomyelinase through modification or deletion of C-terminal cysteine.},
  author={Huawei Qiu and Tim Edmunds and Jennifer Baker-Malcolm and Kenneth P. Karey and Scott Estes and Cordula Schwarz and Heather J Hughes and Scott M Van Patten},
  journal={The Journal of biological chemistry},
  year={2003},
  volume={278 35},
  pages={32744-52}
}
One form of Niemann-Pick disease is caused by a deficiency in the enzymatic activity of acid sphingomyelinase. During efforts to develop an enzyme replacement therapy based on a recombinant form of human acid sphingomyelinase (rhASM), purified preparations of the recombinant enzyme were found to have substantially increased specific activity if cell harvest media were stored for several weeks at -20 degrees C prior to purification. This increase in activity was found to correlate with the loss… CONTINUE READING