GT-02216 is an allosteric ligand for Glucosylceramidase (GCase) that enhances its enzymatic activity. This compound not only promotes the activity of GCase in primary human fibroblasts in a dose-dependent manner but also effectively reduces the accumulation of hexosylsphingosine (HexCer), a substrate linked to various pathologies. Additionally, GT-02216 has demonstrated the ability to decrease Tau accumulation in mutant GBA1 fibroblasts, making it a valuable tool in the study of Parkinson’s disease and related neurodegenerative conditions.
GT-02216 is an allosteric ligand for Glucosylceramidase (GCase) that enhances its enzymatic activity. This compound not only promotes the activity of GCase in primary human fibroblasts in a dose-dependent manner but also effectively reduces the accumulation of hexosylsphingosine (HexCer), a substrate linked to various pathologies. Additionally, GT-02216 has demonstrated the ability to decrease Tau accumulation in mutant GBA1 fibroblasts, making it a valuable tool in the study of Parkinson’s disease and related neurodegenerative conditions.
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