Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age.

@article{Matthes2012EfficacyOE,
  title={Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age.},
  author={Frank Matthes and Stijn Stroobants and Debora Gerlach and Claudia Wohlenberg and Carsten Wessig and Jens Olaf Svanholm Fogh and Volkmar Gieselmann and Matthias Eckhardt and Rudi D'hooge and Ulrich Matzner},
  journal={Human molecular genetics},
  year={2012},
  volume={21 11},
  pages={2599-609}
}
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by a functional deficiency of arylsulfatase A (ASA). Previous studies in ASA-knockout mice suggested enzyme replacement therapy (ERT) to be a promising treatment option. The mild phenotype of ASA-knockout mice did, however, not allow to examine therapeutic responses of the severe neurological symptoms that dominate MLD. We, therefore, generated an aggravated MLD mouse model displaying progressive demyelination and reduced… CONTINUE READING

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