Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

@article{Ma2007ImprovementsIM,
  title={Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.},
  author={Xiucui Ma and Yuli Liu and Mindy Tittiger and Anne K. Hennig and Attila Zolt{\'a}n Kov{\'a}cs and Sarah Popelka and Baomei Wang and Ramin Sedaghat Herati and Mark Bigg and Katherine Parker Ponder},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2007},
  volume={15 5},
  pages={
          889-902
        }
}
Mucopolysaccharidosis I (MPS I) is caused by deficient alpha-L-iduronidase (IDUA) activity and results in the accumulation of glycosaminoglycans and multisystemic disease. Gene therapy could program cells to secrete mannose 6-phosphate-modified IDUA, and enzyme in blood could be taken up by other cells. Neonatal retroviral vector (RV)-mediated gene therapy has been shown to reduce the manifestations of murine MPS I; however, intravenous injection of RV into adults was ineffective owing to a… CONTINUE READING

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