Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy.

@article{Rigat2012EvaluationON,
  title={Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy.},
  author={Brigitte A Rigat and M. B. Tropak and Justin D Buttner and Ellen B Crushell and Daphne Benedict and John William Callahan and Douglas R. Martin and Don Joseph Mahuran},
  journal={Molecular genetics and metabolism},
  year={2012},
  volume={107 1-2},
  pages={203-12}
}
Deficiencies of lysosomal β-D-galactosidase can result in GM1 gangliosidosis, a severe neurodegenerative disease characterized by massive neuronal storage of GM1 ganglioside in the brain. Currently there are no available therapies that can even slow the progression of this disease. Enzyme enhancement therapy utilizes small molecules that can often cross the blood brain barrier, but are also often competitive inhibitors of their target enzyme. It is a promising new approach for treating diseases… CONTINUE READING

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