α-Galactosidase A expressed in the salivary glands partially corrects organ biochemical deficits in the fabry mouse through endocrine trafficking.

@article{Passineau2011GalactosidaseAE,
  title={α-Galactosidase A expressed in the salivary glands partially corrects organ biochemical deficits in the fabry mouse through endocrine trafficking.},
  author={Michael J. Passineau and Timothy Fahrenholz and Laurie Machen and Lee Zourelias and Katherine Nega and Rachel Paul and Mary Macdougall and Olga A. Mamaeva and Richard A Steet and Jarrod W Barnes and H M 'Skip' Kingston and Raymond L. Benza},
  journal={Human gene therapy},
  year={2011},
  volume={22 3},
  pages={293-301}
}
Fabry disease is caused by an X-linked deficiency of the lysosomal enzyme α-galactosidase A (GLA) and has been treated successfully with enzyme replacement therapy (ERT). Gene therapy has been proposed as an alternative to ERT due to the presumed advantages of continuous, endogenous production of the therapeutic enzyme. GLA production in the liver and its therapeutic efficacy in the Fabry mouse have been demonstrated previously with various viral vector systems. In consideration of the… CONTINUE READING