Article
Identification of novel glucocerebrosidase chaperones by unexpected skeletal rearrangement reaction.
Bioorganic & medicinal chemistry letters - 15 Nov 2023
Takeda Kunitoshi, Watanabe Toru, Smith James R, Vesey David, Tiberghien Nathalie, Lewis Sian, Powney Ben, Schapira Anthony H V, Hoshikawa Tamaki, Takle Andrew K
Abstract excerpt
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 investigations revealed that compound 5 was slowly transformed into a regio-isomeric compound (6) in PBS buffer, plausibly via a...
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