Identification of novel glucocerebrosidase chaperones by unexpected skeletal rearrangement reaction

[Display omitted] Compound 5 was identified from a high-throughput screening campaign as a small molecule pharmacological chaperone of glucocerebrocidase (GCase), a lysosomal hydrolase encoded by the GBA1 gene, variants of which are associated with Gaucher disease and Parkinson’s disease. Further in...

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Veröffentlicht in:	Bioorganic & medicinal chemistry letters 2023-11, Vol.96, p.129531-129531, Article 129531
Hauptverfasser:	Takeda, Kunitoshi, Watanabe, Toru, Smith, James R., Vesey, David, Tiberghien, Nathalie, Lewis, Sian, Powney, Ben, Schapira, Anthony H.V., Hoshikawa, Tamaki, Takle, Andrew K.
Format:	Artikel
Sprache:	eng
Schlagworte:	Gaucher disease Gaucher Disease - drug therapy Glucocerebrosidase Glucosylceramidase - genetics Humans Molecular Chaperones Mutation Parkinson Disease Pharmacological chaperone Rearrangement reaction
Online-Zugang:	Volltext
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