Non-iminosugar glucocerebrosidase small molecule chaperones

Non-iminosugar glucocerebrosidase small molecule chaperones

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, cel...

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Veröffentlicht in:MedChemComm 2012-01, Vol.3 (1), p.56-60
Hauptverfasser: Marugan, Juan Jose, Huang, Wenwei, Motabar, Omid, Zheng, Wei, Xiao, Jingbo, Patnaik, Samarjit, Southall, Noel, Westbroek, Wendy, Lea, Wendy A, Simeonov, Anton, Goldin, Ehud, Debernardi, Maria A, Sidransky, Ellen
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Sprache:eng
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Zusammenfassung: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.
ISSN:2040-2503
2040-2511
DOI:10.1039/c1md00200g