Non-iminosugar glucocerebrosidase small molecule chaperones.

نویسندگان

  • Juan Jose Marugan
  • Wenwei Huang
  • Omid Motabar
  • Wei Zheng
  • Jingbo Xiao
  • Samarjit Patnaik
  • Noel Southall
  • Wendy Westbroek
  • Wendy A Lea
  • Anton Simeonov
  • Ehud Goldin
  • Maria A Debernardi
  • Ellen Sidransky
چکیده

Small molecule chaperones are a promising therapeutic approach for the Lysosomal Storage Disorders (LSDs). Here, we report the discovery of a new series of non-iminosugar glucocerebrosidase inhibitors with chaperone capacity, and describe their structure activity relationship (SAR), selectivity, cell activity phamacokinetics.

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عنوان ژورنال:
  • MedChemComm

دوره 3 1  شماره 

صفحات  -

تاریخ انتشار 2012