Identification of pyrimidinyl piperazines as non-iminosugar glucocerebrosidase (GCase) pharmacological chaperones

已完结 10 由 Holly 发布于 2024/12/27 13:56:52

DOI:10.1016/j.bmcl.2023.129130
作者：Tamaki Hoshikawa ,?Toru Watanabe ,?Makoto Kotake ,?Nathalie Tiberghien ,?Chi-kit Woo ,?Sian Lewis ,?Thomas Briston ,?Mumta Koglin ,?James M. Staddon ,?Ben Powney ,?Anthony H.V. Schapira ,?Andrew K. Ta
文献类型：期刊论文
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Elsevier

Abstract：Glucocerebrosidase (GCase) is a lysosomal enzyme encoded by the GBA1 gene, loss of function variants of which cause an autosomal recessive lysosomal storage disorder, Gaucher disease (GD). H

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