Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease

@inproceedings{Ashe2011IminosugarBasedIO,
  title={Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease},
  author={Karen M. Ashe and Dinesh S. Bangari and Lingyun Li and Mario A. Cabrera-Salazar and Scott D. Bercury and Jennifer B. Nietupski and Christopher G. F. Cooper and Johannes M. F. G. Aerts and Edward R. Lee and Diane P. Copeland and Seng H. Cheng and Ronald K. Scheule and John Marshall},
  booktitle={PloS one},
  year={2011}
}
The neuropathic glycosphingolipidoses are a subgroup of lysosomal storage disorders for which there are no effective therapies. A potential approach is substrate reduction therapy using inhibitors of glucosylceramide synthase (GCS) to decrease the synthesis of glucosylceramide and related glycosphingolipids that accumulate in the lysosomes. Genz-529468, a blood-brain barrier-permeant iminosugar-based GCS inhibitor, was used to evaluate this concept in a mouse model of Sandhoff disease, which… CONTINUE READING

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